Neurology & Neurological Sciences Clinical Trials
How to Participate
Sign up here to be added to a registry for clinical trials. This survey will ask you questions about yourself and contact information that takes about 10 minutes.
Who We Are
Learn More
For more details on all of our clinical trials visit the SOM Clinical Trial Database or ClinicalTrials.gov
What is a clinical trial?
- Clinical trials are research studies that explore whether a medical strategy, treatment or device is safe and effective for people. Before people are given a new intervention (drug, device, or treatment), it is carefully studied in the laboratory or in animals. Studies with the most promising results are then moved into clinical trials with people. Clinical trials are used to evaluate new and better ways to treat, prevent, detect, diagnose, and manage symptoms of diseases.
Why Participate?
- By participating in clinical research, you help medical science by providing valuable information about treatments and prevention of disease. Most treatments we use today are the result of past clinical trials. One way to gain access to promising new interventions is to participate in a clinical trial while also helping science.
Eligibility
- Researchers follow clinical trials guidelines when deciding who can participate in a study. These guidelines are called Inclusion/Exclusion Criteria. These criteria are based on factors such as age, gender, the type and stage of a disease, treatment history, and other medical conditions. Trial coordinators can provide additional information about individual trials.
What are the benefits and risks of participating in a clinical trial?
- By being in a clinical trial you help advance medicine and help future patients. You may also have access to promising treatments by joining a clinical trial. However, all clinical trials have risks. There may be side effects or other risks, which may or may not be worse than those from regular care. The intervention (drug, device, treatment) may not work for you, even if it helps others and you may get a placebo. Joining a research trial may be more burdensome due to more frequent visits. However, more visits may give you more attention and monitoring which you would not normally get by your regular doctor.
The Experimental Subject's Bill of Rights include the basic required elements of informed consent and the Experimental Subject's Bill of Rights (California Law).
These documents do not contain study specific information, but state what will be explained to the participant about the specific study by the interpreter (e.g. key information, purpose of the research, expected duration, procedures, risks, benefits, alternatives [if any], confidentiality of information, compensation [if any] for research-related injuries, whom to contact for questions about the research and research subjects' rights, that participation is voluntary, future use of the participants' information or biological specimens, and whether information about the research will be submitted for inclusion in a clinical trial registry).
Clinical Trials
Central Neuroscience Trials Group
Research Studies affiliated with the Stanford ADRC
Healthy Brain Aging Study
Goal: The Healthy Brain Aging Study refers to research undertaken by the Stanford Alzheimer’s Disease Research Center (ADRC). The Stanford ADRC is part of a nationwide network of congressionally mandated Centers of Excellence supported by the National Institutes of Health (NIH). We study people with Alzheimer’s disease, mild cognitive impairment, Lewy body disease, Parkinson’s disease, and normal cognition (healthy brain aging). The goal is to obtain information on factors that affect the risk of Alzheimer’s disease and related disorders and to find out how healthy brain aging differs from disorders like Alzheimer’s disease and Parkinson’s disease. The study collects information on medical history and family history. Data collection often includes questionnaires, memory testing, a blood sample, a brain scan, a stool sample, a very small (3 millimeter) skin biopsy, and spinal fluid examination. Not everyone can do all these studies, but we ask participants to consider these when possible. The Healthy Brain Study follows volunteers over time, and many participants consider brain donation at the time of death. Brain donation provides a definitive diagnosis and provides tissues for scientific research.
Sponsor: The NIH National Institute on Aging (grant no. P30 AG066515)
PI: Victor Henderson, MD, MS
Study status: Actively recruiting
Contact: Veronica Ramirez
vramirez1@stanford.edu
(650) 721-2409
Mind & Memory Changes Study (LB-SPARK)
Goal: The aim is to understand mind and memory changes in Parkinson’s disease through this longitudinal study over time
Sponsor: The Lewy Body Scientific Partnership for Advancing Research and Knowledge
PI: Kathleen Poston, MD, MS
Study status: actively recruiting individuals with Parkinson’s disease, Dementia with Lewy bodies, and healthy volunteers
Coordinator: Alena Smith (lbsparkstudy@stanford.edu)
Alzheimer Gut Microbiome Project (AGMP)
Goal: To understand the role of the gut microbiome derived from stool samples in relation to Alzheimer disease.
Sponsor: The NIH National Institute on Aging (grant no. U19 AG063744) and Duke University
PI: Ami Bhatt, MD, PhD and Victor Henderson, MD, MS
Study status: Recruit occurs through the Stanford ADRC
Asian Cohort for Alzheimer’s Disease (ACAD)
Goal: To study the genetics of Alzheimer’s disease in people of Chinese ancestry (Mandarin- or English-speaking). Eligible ACAD participants are encouraged to co-enroll in the Stanford ADRC.
Sponsor: The NIH National Institute on Aging (grant no. R56 AG069130) and the University of Pennsylvania
Website: https://med.stanford.edu/acad
PI: Victor Henderson, MD, MS
Study status: Actively recruiting
Research coordinator: Veronica Ramirez
vramirez1@stanford.edu
(650) 721-2409
Study of Tau Aggregation in Neurodegeneration and Drinking (STAND)
Goal: The STAND project will be the first to investigate tau pathology and its relationship to cognitive impairment in living humans with Alcohol Use Disorder (AUD) using tau-PET imaging.
PI: Claudia Padula, PhD
Sponsor: The Stanford Department of Psychiatry and Behavioral Sciences
Study Status: Actively Recruiting
Research Coordinator: Sophia Y. Li, syuyeli@stanford.edu
Development of a cost-effective and neurobiologically valid VR assessment tool for early detection of AD
Goal: To assess virtual reality measures in relation to MRI brain scan images to detect very early changes of Alzheimer’s disease.
Sponsor: The NIH National Institute on Aging (grant no. R21 AG073973)
PI: Hadi Hosseini, PhD
Study status: Recruiting
Research coordinator: Samantha Reitmaier
mcik_study@stanford.edu
or samreit@stanford.edu
(650) 724-2939
Early Onset AD Consortium - the LEAD Study (LEADS)
Goal: To study the natural history of people with early-onset Alzheimer’s disease in order to improve diagnosis and assess biomarker progression. LEADS participants are eligible to co-enroll in the Stanford ADRC.
Sponsor: The NIH National Institute on Aging (grant no. U01 AGA057195) and Indiana University/Purdue University
PI: Sharon Sha, MD, MS
Study status: Recruiting
Research Coordinator: Stephanie Tran
Email: trans@stanford.edu
Phone: 650-521-7287
Iron as an imaging biomarker for inflammation in Alzheimer’s disease
Goal: To learn how iron may be involved in Alzheimer’s disease through MRI and PET-MRI brain imaging.
Sponsor: NIH National Institute on Aging (grant no. R01 AG061120) and GE Healthcare
Status: Actively recruiting
PI: Michael Zeineh, MD
Research Coordinator: Meghan Bell
mbell11@stanford.edu
(650) 736-1584
Neighborhoods Study: Contextual disadvantage and Alzheimer's disease and related dementias
Goal: To evaluate how neighborhood disadvantage across the life span affects the risk of dementia (limited to Stanford ADRC participants).
Sponsor: The NIH National Institute on Aging (grant no. R01AG070883) and the University of Wisconsin
PI: Victor Henderson, MD, MS and Patricia Rodriguez Espinosa, PhD
Study status: Not recruiting
Research coordinator: Nicole Caceres
Speaker-listener coupling and brain dynamics during naturalistic verbal communication in Alzheimer's disease
Goal: To study natural speech communication in Alzheimer’s disease using functional MRI brain imaging.
Sponsor: The NIH National Institute on Aging (grant no. R21 DC017950-02S1)
PI: Vinod Menon, PhD and Daniel Abrams, PhD
Study status: Actively recruiting
Research coordinator: Delaney Ubellacker, braindevelopment@stanford.edu
Tool to identify Parkinson’s disease and Dementia with Lewy Bodies using digital facial expression biomarkers
Goal: To create a digital image of the face in people with early stages of Parkinson’s disease to help predict the severity and progression of Parkinson symptoms.
Sponsor: The Michael J. Fox Foundation for Parkinson’s Disease Research and Global Brain Health Institute
PI: Kathleen Poston, MD, MS
Study status: Actively recruiting
Research coordinator: Alena Smith
alenaa@stanford.edu
(650) 269-0484
North American Prodromal Synucleinopathy Stage 2 (NAPS2) Consortium
PI: Mitchell Miglis, MD
Study Status: Closed to enrollment
Research Coordinator: Adele Viviani
Contact: autonomicresearch@stanford.edu
Open-label prospective study for immune therapies in autoimmune gastrointestinal dysmotility (AGID)
PI: Dong-In Sinn, MD
Study Status: Open to enrollment
Research Coordinator: Barbara Hung
Contact: autonomicresearch@stanford.edu
Syn-Sleep Study
PI: Mitchell Miglis, MD
Study Status: Closed to enrollment
Research Coordinator: Adele Viviani
Contact: autonomicresearch@stanford.edu
A Phase 2, double-blind, placebo-controlled, parallel-group study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and potential efficacy of multiple doses of ONO-2808 in patients with Multiple System Atrophy (MSA)
PI: Mitchell Miglis, MD
Study Status: Closed to enrollment
Research Coordinator: Jordan Seliger or Barbara Hung
Contact: autonomicresearch@stanford.edu
A Phase 2 Double-Blind Placebo-Controlled Single-Dose Study of Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of REGN7544, an NPR1 Antagonist Monoclonal Antibody, in Patients with Postural Orthostatic Tachycardia Syndrome
PI: Mitchell Miglis, MD
Study Status: Open to enrollment
Research Coordinator: Barbara Hung
Contact: autonomicresearch@stanford.edu
A Phase 3, Randomized, Multicenter, Double-Blind, PlaceboControlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)
PI: Safwan Jaradeh, MD (Cardiovascular Medicine PI: Kevin Alexander, MD)
Study Status: Open to enrollment
Research Coordinator: Adele Viviani
Contact: autonomicresearch@stanford.edu
Effect of low sodium oxybate (LXB) on autonomic symptom burden in idiopathic hypersomnia patients with postural tachycardia syndrome: a placebo-controlled, double-blind, randomized withdrawal study
PI: Mitchell Miglis, MD
Study Status: Enrolling soon
Research Coordinator: Jordan Seliger or Barbara Hung
Contact: autonomicresearch@stanford.edu
Ultrasound Guided Stellate Ganglion Block in Postural Tachycardia Syndrome: A randomized double blind sham placebo-controlled pilot study
PI: Mitchell Miglis, MD (Anesthesia PI: Anna Maria Bombardieri, MD, PhD)
Study Status: Open to enrollment
Research Coordinator: Barbara Hung
Contact: autonomicresearch@stanford.edu
A First-in-human Study of Inhibitory Interneurons (NRTX-1001) in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (MTLE)
PI: Kevin Graber, MD
NCT05135091
Study Status: Enrolling
Research Coordinator: Adele Viviani
Contact: epilepsyresearch@stanford.edu
A Study of Inhibitory Interneurons (NRTX1001) for the Treatment of Drug-resistant Bilateral Temporal Lobe Focal Seizures
PI: Kevin Graber, MD
NCT06422923
Study Status: Closed to enrollment
Research Coordinator: Adele Viviani
Contact: epilepsyresearch@stanford.edu
Electrophysiological Biomarkers of Consciousness in Patients with Epilepsy and Patients with Psychogenic Non-Epileptic Spells
PI: Kimford Meador, MD
Study Status: Enrolling
Research Coordinator: Adele Viviani
Contact: epilepsyresearch@stanford.edu
Electroencephalographic Biomarkers of Neuro-Cognitive Function
PI: Kimford Meador, MD
Study Status: Enrolling
Research Coordinator: Adele Viviani
Contact: epilepsyresearch@stanford.edu
Using Artificial Intelligence to Choose the Optimal Antiepileptic Drug
Protocol ID: 46059
PI: Robert S. Fisher, MD, PhD
Status: Enrolling
Contact: epilepsyresearch@stanford.edu
A Prospective, Open-Label, Single-Arm, Multi-Center, Pilot Study To Evaluate The Safety, Tolerability, And Preliminary Efficacy Of Low- Intensity Focused Ultrasound Neuromodulation In Patients With Drug Resistant Unilateral Or Bilateral Temporal Lobe Epilepsy
Protocol ID: 73191
PI: Robert S. Fisher, MD, PhD
Status: Enrolling
Research Coordinator: Adele Viviani
Contact: epilepsyresearch@stanford.edu
Transcranial Direct Current Stimulation to Treat Epilepsy
Protocol ID: 47711
PI: Robert S. Fisher, MD, PhD
Status: Enrolling
Research Coordinator: Adele Viviani
Contact: epilepsyresearch@stanford.edu
AI-Guided 3D Video Analysis for Seizure Detection
Protocol ID: 53035
PI: Robert S. Fisher, MD, PhD
Status: Enrolling
Contact: epilepsyresearch@stanford.edu
Cognitive Effects of Vinpocetine in Patients with Epilepsy
PI: Kimford Meador, MD
NCT02011971
Study Status: Enrolling soon
Research Coordinator: Adele Viviani
Contact: epilepsyresearch@stanford.edu
Personal Impact of Epilepsy Scale (PIES): Determining the Minimally Important Change
Protocol ID : 43107
PI: Robert S. Fisher, MD, PhD
Status: Closed, in data analysis phase
Contact: epilepsyresearch@stanford.edu
A Phase 2/3 Multicenter, Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy, Safety and Tolerability of BHV-7000 in Subjects with Refractory Focal Onset Epilepsy
PI: Yi Li
NCT06132893
Study Status: Enrolling
Research Coordinator: Adele Viviani
Contact: epilepsyresearch@stanford.edu
A Phase 2, Global, Multicenter, Long-term Safety Study Designed to Assess the Safety and Tolerability of BHV-7000 in Subjects with Refractory Focal Onset Epilepsy
PI: Yi Li
Study Status: Enrolling
Research Coordinator: Adele Viviani
Contact: epilepsyresearch@stanford.edu
A prospective study to evaluate the use of the Minder device to aid in developing a treatment plan after inconclusive prolonged EEG in patients with epilepsy.
PI: Zarha Sadat-Hossieny
NCT07110337
Study Status: Enrolling
Research Coordinator: Adele Viviani
Contact: epilepsyresearch@stanford.edu
A prospective long-term follow-up study to evaluate the use of the Minder device to aid in treatment after actionable event identification in patients diagnosed with epilepsy.
PI: Jessica Falco-Walter
NCT07110454
Study Status: Enrolling
Research Coordinator: Adele Viviani
Contact: epilepsyresearch@stanford.edu
The Epilepsy Genomics Research Project
PI: Yi Li
Study Status: Enrolling
Research Coordinator: Adele Viviani
Contact: epilepsyresearch@stanford.edu
POWER Project: Pregnancy Outcomes in Women with Epilepsy Research Project
PI: Yi Li
Study Status: Enrolling
Research Coordinator: Adele Viviani
Contact: epilepsyresearch@stanford.edu
Focal Cerebral Arteriopathy Steroid Trial (FOCAS)
NCT06040255
PI: Sarah Lee
Research Coordinator: Jeilo Gauna childneuroresearch@stanford.edu
Status: Active
A Double-Blind, Randomized, Placebo-Controlled, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of SPN-817 in Adults with Focal Onset Seizures
PI: Niyatee Samudra
Study Status: Enrolling
Research Coordinator: Adele Viviani
Contact: epilepsyresearch@stanford.edu
Bluewing: An Open-Label Study of Zorevunersen in Adult Zorevunersen-Naïve Patients with Dravet Syndrome
PI: Yi Li
Study Status: Enrolling
Research Coordinator: Adele Viviani
Contact: epilepsyresearch@stanford.edu
Characterizing the Gut Microbiome of Patients with Migraine: A Case Control Study at a Tertiary Care Center
PI: Niushen Zhang, MD
Study Status: Active, not recruiting
Contact: Stephanie Tran trans@stanford.edu
Stanford Memory Disorders Clinical Trials
For patient referrals, please reach out to:
Kaila Sevilla @ kailas44@stanford.edu and Pragya Tripathi @ pragyat@stanford.edu
Active, Open for Enrollment
Alzheimer's Disease Trials
MINDSET 2-BMS Trial:
Brief Summary: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of KarXT+KarX-EC for the Treatment of Cognitive Impairment associated with Mild to Moderate Alzheimer’s Disease (MINDSET 2)
This is a clinical research trial to understand whether an investigational drug, KarXT+KarX-EC can improve cognition and overall function in participants with mild to moderate Alzheimer’s disease compared to placebo.
The study treatment consists of two pills: KarXT and KarX-EC.
- KarXT contains two drugs: xanomeline tartrate and trospium chloride.
- KarX-EC contains only xanomeline tartrate.
Xanomeline tartrate stimulates muscarinic receptors (cell parts that receive and respond to a signal in the nervous system) that are involved in thinking and behavioral functions. Trospium chloride is a muscarinic receptor inhibitor that is expected to reduce the negative peripheral (outside the brain) side effects of xanomeline tartrate without diminishing its intended beneficial effects in the brain.
Study drug is an oral drug. If selected, participants will get a drug assigned based on chance. Like the flip of a coin, you will have a 50% chance of receiving either the study drug or placebo.
The time commitment for this study is for about 34 weeks. There would be total of 12-14 onsite visits, which includes
- Screening period (up to 6 weeks),
- Treatment period (approximately 24 weeks) and
- Safety follow up will occur for 4 weeks after treatment discontinuation.
Who can Participate:
- Ages 50-85 years.
- Diagnosis of Mild to moderate Alzheimer’s Disease
- Ability to swallow capsules.
- Participants must have study partner who maintains adequate contact (around 10 hours per week or more) and is willing to attend all study visits.
- BMI within 18 to 40 kg/m2.
- MMSE score of 12 through 22.
If you are interested, please contact Olivia Lu; olivialu@stanford.edu; 650-374-9286
Sponsor: Bristol-Myers Squibb Company
Intervention: KarXT + KarX-EC
Indication: Mild to moderate dementia due to Alzheimer’s disease
Age:60-85Years
Clinicaltrials.gov identifier: https://clinicaltrials.gov/study/NCT06976203
PI: Dr Sharon Sha, MD
Learn more: https://www.bmsstudyconnect.com/it/en/clinical-trials/NCT06976203.html
Bumetanide AD Trial:
Brief Summary: A Phase IIa, Randomized, Double-Blind, Active Placebo-Controlled, Parallel Group study to Evaluate the Safety and Tolerability of Bumetanide in patients with Alzheimer’s Disease
This is clinical research trial using Bumetanide to determine whether it can help slow the development of symptoms in people with early dementia due to Alzheimer’s Disease. Bumetanide is approved by the U.S. Food and Drug Administration (FDA) to treat swelling (edema) and high blood pressure but its use for slowing the symptoms in people with Alzheimer’s Disease symptoms is investigational.
This is an oral study drug. If selected, participants will be randomly assigned to 1 of 3 study groups to receive either:
- 0.25 mg bumetanide daily,
- 0.5 mg bumetanide daily, or
- a placebo (non-active drug for comparison)
Time commitment for this study is approximately 9 months which involves 7 study site visits and up to 4 follow-up phone calls.
The study will have 3 periods:
- Screening period (up to 12 weeks)
- Treatment period (6 months)
- Follow-up period (1 month)
Who can participate:
- Male or female participants aged 50 to 85 years of age at the time of signing the informed consent form
- Diagnosis of either mild cognitive impairment or mild dementia due to Alzheimer`s disease
- Gradual and progressive change in memory function for ≥ 6 months reported by subjects or study partner.
- Evidence of Alzheimer’s Disease pathological process, as confirmed on amyloid PET, plasma ptau-181, or cerebrospinal fluid amyloid-beta/phosphorylated tau index.
- Ability to swallow bumetanide tablet.
- Neuroimaging (MRI) obtained during screening or historic within a year of enrolment consistent with the clinical diagnosis of Alzheimer’s disease.
- Resides at home or in the community (assisted living acceptable).
- Participants must have a study partner.
If you are interested, please contact: Mina Kmiecik; mina.kmiecik@stanford.edu; 650-387-1559
Sponsor: Knight Initiative Brain Resilience Initiative Translational Grant, Stanford University
Intervention: Bumetanide
Indication: Mild Cognitive impairment or mild dementia due to Alzheimer’s disease
Clinicaltrials.gov identifier: https://classic.clinicaltrials.gov/ct2/show/NCT06052163
PI: Kyan Younes, MD
Neuro/PET Study (healthy controls, MS, AD, PD and ALS)
Ashvattha Trial:
Brief Summary: A Phase 1/2 Study to Evaluate the Safety, Pharmacokinetics and Biodistribution of an Imaging agent, 18F-OP-801 (18F Hydroxyl Dendrimer), After Intravenous Administration to Patients with Amyotrophic Lateral Sclerosis (ALS), Alzheimer’s Disease (AD), Multiple Sclerosis (MS), Parkinson’s Disease (PD), and Healthy Volunteers (HV)
The primary objective is to evaluate the safety and tolerability of 18F-OP-801 (18F Hydroxyl Dendrimer [OP-801]), a PET imaging tracer, for imaging in healthy volunteers (HV) subjects and people with amyotrophic lateral sclerosis (ALS), Alzheimer’s disease (AD), multiple sclerosis (MS), or Parkinson’s disease (PD).
For patients with Alzheimer’s Disease, the treatment period is a minimum of 4 days and a maximum of 15 days for PET treatment (18F-OP-801) with a follow-up visit 14 days (Days 18-29) after administration of the second dose.
Including the 60-day screening period, approximate length of study participation will be up to 89 days.
Who can participate:
Inclusion Criteria Specific to AD Subjects:
Adult (Age 40 to 80, inclusive) at the Screening Visit
Clinical diagnosis of early-stage Alzheimer disease.
MMSE score >20 at Screening
If you are interested, please contact: Olivia Lu; olivialu@stanford.edu; 650-374-9286
Sponsor: Ashvattha Therapeutics,Inc
Intervention: 18F-OP-801 (18F Hydroxyl Dendrimer),
Indication: Amyotrophic Lateral Sclerosis (ALS), Alzheimer’s Disease (AD), Multiple Sclerosis (MS), Parkinson’s Disease (PD), and Healthy Volunteers (HV)
Age: 40-60 years
Clinicaltrials.gov identifier: https://clinicaltrials.gov/study/NCT05395624
PI: Farshaad Moradi, MD
Observational Research Study
LEADS (Longitudinal Early Onset Alzheimer's Disease Study)
Brief Summary: A natural history, non-treatment study designed to look at disease progression in individuals with early onset cognitive impairment (less than 65 years old).
This is a non-treatment study for adults diagnosed with early-onset Alzheimer’s disease (EOAD) and Early onset non-Alzheimer's Disease (EO-nonAD). Clinical/cognitive, imaging, biomarker, and genetic characteristics will be collected over 3 years.
Who can participate:
Cognitively impaired (EOAD and EOnonAD) Cohorts Only:
- Meets criteria for Mild Cognitive Impairment due to Alzheimer’s disease or dementia due to Alzheimer’s disease.
- Age between 40-64 years (inclusive) at the time of consent
- Must have a study partner who spends a minimum average of 10 hours per week with the participant.
- Must be fluent in English or Spanish
If you are interested, please contact: Stephanie Tran; trans@stanford.edu; 650-521-7287
Sponsor: Indiana University and NIA (LEADS)
Intervention: N/A - Observational
Indication: Early onset Alzheimer's Disease (EOAD), Early onset non-Alzheimer's Disease (EO-nonAD), and cognitively normal (CN)
Clinicaltrials.gov identifier: https://clinicaltrials.gov/study/NCT03507257
PI: Sharon Sha, MD
Learn more: https://www.alzheimers.gov/clinical-trials/longitudinal-early-onset-alzheimers-disease-study-leads
Observational Trials for Neurodegenerative Disease
Huntington's Disease Project
Brief Summary: Neurodegenerative Disease Research Platform - Novel Remote Monitoring and Deep Phenotyping.
This is an observation study, and the goal of the study is to develop novel markers of disease onset and progression in people with neurodegenerative disease. This observational study will connect remote monitoring with biomarkers of neurodegenerative disease. Mobile app-based cognitive testing and video-based computer vision and machine learning algorithms will identify markers of neurodegenerative disease progression and link them to changes in plasma, skin biopsy, and cerebrospinal fluid with the goal of developing novel biomarkers and therapeutics.
This study consists of one in-person visit per year. The remaining assessments are remote (using video and mobile app assessments) once every three months for at least one year, and participants are invited to continue for as long as they wish for many years to come with the same schedule of events annually (i.e., one in-person visit and three remote evaluations per year)
Who can participate:
1. Individuals who have a neurodegenerative disease diagnosis including but not limited to Huntington`s Disease, Alzheimer`s disease, Parkinson`s disease, Lewy Body Dementia, Frontotemporal Dementia, or Normal Pressure Hydrocephalous.
This group will be subdivided into two groups:
a. Clinical: Clinical features diagnostic of one of the neurodegenerative diseases.
b. Pre-clinical: Biomarker positive without clinical features diagnostic of neurodegenerative disease
2. Healthy aging controls.
3. Non-carrier family members of genetic mutations carriers.
If you are interested, please contact:
Minhtrang Chu; mtchu@stanford.edu; 650-250-3160
Sponsor: Schwab Charitable
Intervention: N/A observational trial
Indication: Neurodegenerative disease diagnosis including but not limited to Huntington`s Disease, Alzheimer`s disease, Parkinson`s disease, Lewy Body Dementia, Frontotemporal Dementia, or Normal Pressure Hydrocephalous.
Age: ≥18 years
Clinicaltrials.gov identifier: N/A
PI: Kyan Younes, MD
Registry Study
ALZ-NET Registry study
Brief Summary: Patient registry for Alzheimer's Network for Treatment and Diagnostics (ALZ-NET)
This study is an observational study including people who are evaluated for or receive treatment with novel (or new) FDA-approved therapies for Alzheimer’s disease. ALZ-NET will follow and record information about people’s health, safety, brain scans, and long-term health outcomes during their regular memory care. ALZ-NET aims to be a resource for evidence gathering, information sharing, and education across clinical and research communities to support and improve care of individuals living with Alzheimer’s disease.
Patients will be monitored if they are evaluated for treatment, throughout treatment duration, and following treatment completion for as long as they are willing. Patients may stop using any novel FDA-approved therapy for Alzheimer’s Disease, or any other commercially available non-investigational Alzheimer’s Disease therapy, during their ALZ-NET participation period. Patients who discontinue the FDA-approved therapy for Alzheimer’s Disease will continue to be followed for the duration of ALZ-NET with all clinical evaluations.
Who can participate:
- Patient or patient’s legally authorized representative (LAR) or proxy (e.g., spouse or legal guardian) has the ability to understand the purpose and risks of ALZ-NET and provide signed and dated informed consent and authorization.
- Patient is at least 18 years of age at the time of informed consent.
- Patient has memory concerns and/or may have diagnosis of Alzheimer’s disease and has been identified by an approved site investigator (as defined by protocol) to be appropriate for treatment with novel FDA-approved AD therapies
- Patient’s treating clinician has made the decision to treat the patient with novel FDA-approved therapy for AD independent of the purpose of ALZ-NET and has already or will be initiating treatment.
If you are interested, please contact: Stephanie Tran; trans@stanford.edu; 650-521-7287
Sponsor: Alzheimer’s Association
Intervention: N/A observational trial. ALZ-NET will collect longitudinal clinical and safety data for enrolled patients treated with novel FDA approved AD therapies.
Indication: Alzheimer’s Dementia or other dementia
Clinicaltrials.gov identifier: N/A
PI:Kyan Younes, MD
Learn more: https://www.alz-net.org/
Active, not recruiting
Sponsor: Ono Pharmaceutical Co., Ltd.
Intervention: ONO-2020
Indication: Mild to moderate Alzheimer’s Dementia
Age: 55-85 years
Clinicaltrials.gov identifier: https://clinicaltrials.gov/study/NCT06881836
PI: Sharon Sha, MD
Learn more: https://www.trialx.com/clinical-trials/listings/297730/a-study-of-ono-2020-in-participants-with-mild-to-moderate-alzheimers-disease/
Brief Summary : A Phase II, 26-week, double-blind, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, and efficacy of ONO-2020 in patients with mild to moderate Alzheimer’s Disease.
This is a clinical research trial to understand whether an investigational drug, ONO-2020 modify the disease progression in patient with mild to moderate Alzheimer’s disease by causing a delay in cognitive decline in AD patients and improves the symptomatic cognition. ONO-2020 is an oral drug. If selected, participants will be randomly assigned to one of three study groups :20 mg of ONO-2020(active study drug) or 60 mg of ONO-2020(active study drug) or Placebo (non-active drug for comparison). The time commitment for this study is for about 8 months which includes screening period (up to 42 days), treatment period (approximately 26 weeks) and safety follow up will occur for 4 weeks after treatment discontinuation. There would be total of 11 onsite visits.
Contact: Kaila Sevilla;kailas44@stanford.edu 650-454-5458
Sponsor: Cognition Therapeutics, Inc and National Institute on Aging (NIA)(START Study)
Intervention: CT1812
Indication: Early Alzheimer’s Dementia
Age: ≥18 years
Clinicaltrials.gov identifier: https://classic.clinicaltrials.gov/ct2/show/NCT05531656
PI: Sharon Sha, MD
Learn more: https://start-study.org/
Brief Summary: Synaptic Therapy Alzheimer’s Research Trial (START): Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Trial to Evaluate the Safety and Efficacy of CT1812 in Early Alzheimer’s Disease over 18 Months.
This is a clinical research trial to understand whether an investigational drug, CT1812 safely slows down the memory loss caused by the Alzheimer’s disease.
CT1812 is an oral drug. If selected, particpants will be randomly assigned to one of three study groups :200 mg of CT1812 (active study drug) or 100 mg of CT1812 (active study drug) or Placebo(non-active drug for comparison).
Contact: Lanja Sinjary; lsinjary@stanford.edu; 650-507-7394
Sponsor: Brigham’s and Women’s Hospital (APEX-AHEAD Plasma Extension study)
Intervention: N/A Observation
Indication: Healthy individuals with normal cognition at risk for Alzheimer’s disease
Age:55-80 years old
PI: Sharon Sha, MD
Learn more: https://www.actcinfo.org/alzheimers-plasma-extension-apex-study/
Brief Summary: This is an observational research study. The APEX study is designed to follow individuals who do not meet all the requirements to be in the AHEAD 3-45 Study. This study is to help researchers understand the changes in blood tests over time that may predict future risk of amyloid build-up, and to understand the decline in thinking and memory that may occur in older individuals who are at risk for developing Alzheimer’s Disease. The APEX study will compare people who did not have intermediate or amyloid levels in the brain (APEX participants) to individuals who did (AHEAD 3-45 Study). Researchers also hope to learn about the factors that may preserve memory and thinking in older individuals.
Contact: Mina Kmiecik; mina.kmiecik@stanford.edu; 650-387-1559
Sponsor: AriBio Co., Ltd.(AriBio Study-Polaris AD )
Intervention: AR1001
Indication: Early Alzheimer’s Disease
Age: ≥55 years
PI: Sharon Sha, MD
Learn more: https://www.earlyalzheimersdiseasestudy.com/
Brief Summary: A Phase 3 Double-blind, Randomized, Placebo-controlled Trial to Evaluate the Efficacy and Safety of AR1001 in Participants with Early Alzheimer’s Disease.
Clinicaltrials.gov identifier: https://classic.clinicaltrials.gov/ct2/show/NCT05531526
Contact: Kaila Sevilla; kailas44@stanford.edu; 650- 454-5458
Sponsor: Biogen MA, Inc.(CELIA Study)
Intervention: BIIB080
Indication: MCI and Alzheimer’s Disease
Age: 50-80 Years old
PI: Irina Skylar-Scott, MD
Learn more: https://www.biogentriallink.com/en-us/home/find-a-clinical-trial/clinical-trials-study-details.html?nctId=NCT05399888
Brief Summary: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Efficacy, Safety, and Tolerability of BIIB080 in Subjects with Mild Cognitive Impairment Due to Alzheimer’s Disease or Mild Alzheimer’s Disease Dementia
Clinicaltrials.gov identifier: https://classic.clinicaltrials.gov/ct2/show/NCT05399888
Contact: Olivia Lu; Olivialu@stanford.edu; 650-374-9286
Sponsor: Eisai and NIH (AHEAD 3-45 Study)
Intervention: BAN2401 (monoclonal antibody binding to amyloid)
Indication: Healthy individuals with normal cognition at risk for Alzheimer’s disease
Age: 55-80 years old
Brief Summary: A Placebo-Controlled, Double-Blind, Parallel-Treatment Arm, 216 Week Study to Evaluate Efficacy and Safety of Treatment With BAN2401 in Subjects With Preclinical Alzheimer’s Disease and Elevated Amyloid (A45 Trial) and in Subjects With Early Preclinical Alzheimer’s Disease and Intermediate Amyloid (A3 Trial)
Clinicaltrials.gov identifier: https://clinicaltrials.gov/study/NCT04468659
PI: Sharon Sha, MD
Contact: Mina Kmiecik; mina.kmiecik@stanford.edu; 650-387-1559
Lewy Bodies Dementia Trials
Sponsor: Cognition Therapeutics, Inc.(Cognition EAP)
Intervention: CT1812(Zervimesine)
Indication: Mild to moderate dementia with Lewy Bodies
Age: 50-86 years
Clinicaltrials.gov identifier: https://clinicaltrials.gov/study/NCT06961760
PI: Sharon Sha, MD
Learn more: https://ir.cogrx.com/press_releases/philanthropic-donor-funds-cognition-therapeutics-expanded-access-program-for-zervimesine-ct1812-in-dementia-with-lewy-bodies/
Brief Summary: A Single Arm, Open Label, Expanded Access Program to Provide 100 mg CT1812 (zervimesine) and to Collect Long Term Safety and Efficacy Data in Participants with Mild to Moderate Dementia with Lewy Bodies (DLB).
This is a multi-center, open label, expanded access program (EAP) that will provide 100 mg CT1812 for up to one year to participants with mild-to-moderate DLB.
Participants will be screened for eligibility by physical, laboratory, and select psychometric and neurologic assessments. Participants, after having met all inclusion criteria, and none of the exclusion criteria, will be enrolled. The first dose will be taken in the clinic. For days where there are no clinic visits, participants will ingest study drug each morning at home. Participants and their caregivers/study partners will have a study visit for safety procedures, and repeat select psychometric/neurologic testing, at Day 180 and Day 360. Participants will have laboratory samples taken for safety testing approximately every 2 weeks after their baseline visit until Day 60, then every 30 Days until Day 360. For each participant, a follow-up call will occur approximately 30 days after the last study drug intake.
Contact: Stephanie Tran; trans@stanford.edu; 650-521-7287
PARKINSON'S DISEASE
Blood biomarkers study in REM sleep behavior disorder (RBD)
Purpose: studying neuroinflammation and immune protective factors in patients with RBD
PI: Emmanuel H. During, MD
Co-PI: Emmanuel Mignot, MD, PhD
Study status: Open, enrollment ongoing
Research Coordinator: Ana Cahuas
acahuas@stanford.edu 650 721 5489
Home diagnosis of REM sleep behavior disorder (RBD) using wearable sleep trackers
Purpose: developing a machine learning method to diagnose RBD in the home environment with wrist-worn actigraphy
PI: Emmanuel H. During, MD
Study status: Open, enrollment ongoing
Research Coordinator: Ana Cahuas
acahuas@stanford.edu 650 721 5489
U19 North American Prodromal Synucleinopathy (NAPS) consortium
Purpose: Neuroprotection trial planning in REM sleep behavior disorder (RBD)
PI: Emmanuel H. During, MD
Study status: Open, enrollment ongoing
Research Coordinator: Vincent Nguyen
vnguyen9@stanford.edu
Sodium Oxybate in Refractory REM Sleep Behavior Disorder: A Randomized Controlled Study
Purpose: This study evaluates the efficacy of sodium oxybate in individuals with and without Parkinson’s disease who act out their dreams (REM Sleep Behavior Disorder, known as “RBD”) and do not respond to usual therapies.
PI: Mitchell Miglis, MD
Study status: Open, enrollment ongoing
Research Coordinator: Ana Cahuas
acahuas@stanford.edu 650 721 5489
Mind & Memory Changes Study (LB-SPARK)
PI: Kathleen Poston, MD, MS
Study status: Open, Enrollment ongoing
Research coordinators: Larissa Gomez and Ava Belzer
LBSparkStudy@stanford.edu
Healthy Brain Aging Study
Sponsor: NIH/NIA
PI:Victor Henderson, MD and Tony Wyss-Coray, PhD
Study Status: Open, enrollment ongoing
Research coordinator: Christina Wyss-Coray
ADRCstanford@stanford.edu (650) 721-2409
Bilateral Closed Loop Deep Brain Stimulation for Freezing of Gait using Neural and Kinematic Feedback (NCT04043403)
Sponsor(s): NIH/NINDS, BRAIN Initiative
Purpose: This study aims to investigate the safety and feasibility of adaptive deep brain stimulation for impaired gait and freezing of gait in Parkinson’s disease, driven by subject-specific neural or behavioral control variables, and in response to medication.
PI: Helen Bronte-Stewart, MD, MS
Study status: Open, enrollment ongoing
Research coordinator: Sudeep Aditham
sudeepa@stanford.edu (650) 723-6709
The Natural History of Synucleinopathies
PI: Mitchell Miglis, MD
Study status: Open, enrollment by invitation only
Research coordinator: Jordan Seliger
jseliger@gmail.com
ATYPICAL PARKINSONISM (MSA, PSP, CBD, DLB)
M-STAR
Sponsor: Biohaven
Site PI: Mitchell Miglis, MD
Study status: Closed for enrollment
Research coordinator: Ruba Shaik
rubas@stanford.edu 650-546-1436
HUNTINGTON’S DISEASE
Generation HD
Clinical Trials Protocol#: NCT03761849
Sponsor: Roche
Site PI: Jacinda Sampson, MD, PhD
Study status: Enrollment Closed, trial ongoing
Research coordinator: Stephanie Tran
trans@stanford.edu
Upcoming Movement Clinical Trials
PARKINSON’S DISEASE
ADX48621 for the Treatment of Levodopa Induced Dyskinesia in Patients With Parkinson's Disease
Clinical Trials Protocol#: NCT01336088
Sponsor: Addex Pharmaceuticals
Study status: Pending, open to enrollment Fall/Winter 2020
Research coordinator: Lila Perrone
perronel@stanford.edu
Open-Label Study With Pimavanserin on Activities of Daily Living in Subjects With Parkinson's Disease Psychosis
Clinical Trials Protocol#: NCT04292223
Sponsor: Acadia
Site PI: Sharon Sha, MD, MS
Study status: Pending, open to enrollment Fall/Winter 2020
Research coordinator: Lila Perrone
perronel@stanford.edu
Project BIG: The Stanford Brain Immune Gut Research Initiative
PI: Jeffrey Dunn, MD
Research Coordinators: Kristen Gee, and Taya Salman neuroimmunologyresearch@stanford.edu
Status: Enrolling
A Phase 1, Open-Label, Single Center Study of KYV-101, an Autologous Fully-Human Anti-CD19 Chimeric Antigen Receptor T-Cell (CD19 CAR T) Therapy, in Subjects with Non-relapsing and Progressive Forms of Multiple Sclerosis (Kyverna/IIT)
NCT06138132
PI: Jeffrey Dunn, MD
Research Coordinator: Emma Martinez, neuroimmunologyresearch@stanford.edu
Status: Enrolling
A Phase 1 Open-label, Multiregional, Multicenter, Basket Study Evaluating the Safety and Efficacy of KITE-363, an Autologous Anti-CD19/CD20 CAR T-cell Therapy in Participants with Relapsed/Refractory Autoimmune Neurologic Diseases (KITE)
NCT07304154
PI: Kristin Galetta, MD
Research Coordinators: Jennifer Cuevas, and Kristen Gee, neuroimmunologyresearch@stanford.edu
Status: Enrolling
A Multicenter Randomized Controlled Trial of Best Available Therapy versus Autologous Hematopoietic Stem Cell Transplant for Treatment-Resistant Relapsing Multiple Sclerosis (BEAT-MS)
NCT04047628
PI: Jeffrey Dunn, MD
Research Coordinators: Taya Salman, and Emma Martinez, neuroimmunologyresearch@stanford.edu
Status: Enrolling
A Single-arm, Open-label, Phase I Study to Determine the Safety, Tolerability, and Preliminary Efficacy of Obe-cel in Participants with Refractory Progressive Forms of Multiple Sclerosis (BOBCAT/Autolus).
NCT07139743
PI: Jeffrey Dunn, MD
Research Coordinators: Bella Nguyen, and Emma Martinez neuroimmunologyresearch@stanford.edu
Status: Enrolling
A Long-Term Follow-Up Study for Participants Previously Treated with KYV-101 (KYSA-4) (LTFU)
PI: Jeffrey Dunn, MD
Research Coordinator: Emma Martinez, neuroimmunologyresearch@stanford.edu
Status: Enrolling
R21 - Immune-mediated Cognitive Impairment in adult participants with NMOSD
PI: May Han, MD
Research Coordinators: Kristen Gee, and Jennifer Cuevas, neuroimmunologyresearch@stanford.edu
Status: Enrolling
Retinal Biomarkers of Inflammation and Degeneration in Multiple Sclerosis
PI: Heather Moss, MD
Research Coordinator: Kristen Gee, neuroimmunologyresearch@stanford.edu
Status: Enrolling
CorEvitas SPHERES (Synergy of Prospective Health & Experimental Research for Emerging Solutions) Registry for Neuromyelitis Optica Spectrum Disorder (NMOSD)
NCT04886492
PI: May Han, MD
Research Coordinator: Kristen Gee, neuroimmunologyresearch@stanford.edu
Status: Enrolling
A Phase 3 randomized, double-blind, efficacy and safety study comparing frexalimab (SAR441344) to placebo in adult participants with Nonrelapsing Secondary Progressive Multiple Sclerosis (FREVIVA)
NCT06141486
PI: Christopher Lock, MD
Research Coordinator: Jennifer Cuevas, neuroimmunologyresearch@stanford.edu
Status: Enrollment closed
A Randomized, Double-blind, Placebo-Controlled, Multicenter, Phase 3, Pivotal Study with an Open-Label Extension Period to Evaluate the Efficacy and Safety of Rozanlixizumab in Adult Participants with Myelin Oligodentrocyte Glycoprotein (MOG) antibody-associated disease (MOG-AD) (cosMOG)
NCT05063162
PI: May Han, MD
Research Coordinator: Kristen Gee, neuroimmunologyresearch@stanford.edu
Status: Enrollment Closed
A Multicenter, Single-arm, Open-label, Extension, Rollover Study To Evaluate The Long-term Safety And Efficacy Of Ocrelizumab In Patients With Multiple Sclerosis (OLERO)
NCT05269004
PI: Jeffrey Dunn, MD
Research Coordinator: Jennifer Cuevas, neuroimmunologyresearch@stanford.edu
Status: Enrollment closed
A Phase IIIB Multicenter, Randomized, Double-Blind, Controlled Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of a Higher Dose of Ocrelizumab in Adults with Primary Progressive Multiple Sclerosis (BN42083/GAVOTTE)
NCT04548999
PI: Lucas Kipp, MD
Research Coordinator: Jennifer Cuevas, neuroimmunologyresearch@stanford.edu
Status: Enrollment closed
Neuromuscular Clinical Research Group
Adult studies
All Neuromuscular Conditions
Subject Database and Specimen Repository for Neuromuscular and Neurodegenerative Disorders
Protocol ID: 23888
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Veronica Schnyer, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: This is a study that involves collecting clinical information of subjects (patients with any neurological condition or their close family member) and tissue samples to develop a recruitment database and tissue bank, which will be of great value in helping the investigators learn more about various related neurological conditions.
Status: Recruiting
Determination of Standards for Maximal and Submaximal Exercise testing for Individuals with Neuromuscular Disease (CPET)
Protocol ID: 54078
NCT: N/A
PI: Dr. Tina Duong
Study Coordinator: Sabrina Salvatore, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Understanding exercise testing which will assess how heart and lungs work together to get oxygen to muscles in children and adults with muscular dystrophies
Status: Recruiting
Recruitment Notes: All clinic patients ages 9+ with diagnosed neuromuscular disorders who are able to perform testing on cycle ergometer as well as controls
A6MCT Testing the feasibility and efficacy of the 6-minute assisted cycle test in non-ambulatory neuromuscular patients
Protocol ID: N/A
NCT: N/A
PI: Dr. Tina Duong
Study Coordinator: Audrey Chun, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Status: Recruiting
Development and Validation for the Adult Test of Neuromuscular Disorders (ATEND), a Functional Motor Outcome Measure
Protocol ID: 58208
NCT: N/A
PI: Dr. Tina Duong
Study Coordinator: Tia Lum, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Development and evaluation of psychometric properties of the ATEND for weaker individuals with neuromuscular disorders
Status: Recruiting
Recruiting Notes: Non-ambulatory individual in a wheelchair with a neuromuscular diagnosis
OpenCAP Video technology to assess timed function tests and gait quality
Protocol ID: N/A
NCT: N/A
PI: Dr. Tina Duong
Study Coordinator: Sarah Ismail, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Status: Recruiting
Addressing Immunotoxicities to AAV Gene Therapy for DMD and Other Disorders
Protocol ID: 79348
NCT: N/A
PI: Dr. John W. Day and Dr. Bradley Hamilton
Study Coordinator: TBD, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose:
Status: Recruiting
Amyotrophic Lateral Sclerosis (ALS)
Clinical Procedures to Support Research in ALS (CAPTURE-ALS)
Protocol ID: 54467
NCT03489278
PI: Dr. Maxwell Greene
Study Coordinator: Raiye Hailu, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: NINDS/NIH, MDA
Purpose: Natural History Study of ALS and PLS
Status: Recruiting in clinic
Refining the Patient-Ranked Order of Function in People Living with ALS (PROOF)
Protocol ID: 75570
NCT: N/A
PI: Dr. Maxwell Greene
Study Coordinator: Raiye Hailu, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: DOD and Dutch Research Council
Purpose: Natural History Study of ALS, interview-based study
Status: Recruiting
A Phase 1-3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION363 in Amyotrophic Lateral Sclerosis Patients with Fused in Sarcoma Mutations (FUSION)
Protocol ID: 59619
NCT04768972
PI: Dr. John W. Day
Study Coordinator: Habib Mofakham Fini, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: IONIS Pharmaceuticals
Purpose: Assessing the safety and efficacy of ION363 in children and adults with ALS
Status: Active, no longer recruiting
Recruitment Notes: Confirmed genetic mutation in FUS, ages 16-65
Expanded Access Program to ION363 FUS ALS treatment
Protocol ID: N/A
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Habib Mofakham Fini, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Expanded Access Program
Status: Recruiting on case-by-case basis
Briefer Cognitive Behavioral Screen in ALS
Protocol ID: 77489
NCT: N/A
PI: Drs. Maxwell Greene and Ying Lee
Study Coordinator: Raiye Hailu, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Status: Recruiting
Becker and Duchenne muscular dystrophy (BMD/DMD)
A Phase 2 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Effect of EDG-5506 on Safety, Biomarkers, Pharmacokinetics, and Functional Measures in Adults and Adolescents with Becker Muscular Dystrophy (GRAND CANYON)
Protocol ID: 72069
NCT05291091
PI: Dr. John W. Day
Study Coordinator: Sonia Pathak, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Edgewise Therapeutics
Purpose: Assessing the safety and efficacy of EDG-5506-201 in adults with BMD
Status: Active, no longer recruiting
Recruitment Notes: Ambulatory adults with BMD ages 18-50
A Phase 2 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Effect of EDG-5506 on Safety, Biomarkers, Pharmacokinetics, and Functional Measures in Adults and Adolescents with Becker Muscular Dystrophy (GRAND CANYON OLE)
Protocol ID: 81418
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Sonia Pathak, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Edgewise Therapeutics
Purpose: Assessing the safety and efficacy of EDG-5506-201 in adults with BMD
Status: Recruiting by invitation only
A Gene Delivery Study to Evaluate the Safety of and Expression From SRP-9001 in Duchenne Muscular Dystrophy (DMD) (ENDEAVOR)
Protocol ID: 59448
NCT04626674
PI: Dr. John W. Day
Study Coordinator: Raiye Hailu, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Sarepta Therapeutics, Inc
Purpose: Assessing the safety and efficacy of SRP-9001 gene therapy in individuals with DMD
Status: Recruiting
Long-term follow-up study for all SRP-9001 studies (EXPEDITION)
Protocol ID: 71344
NCT05967351
PI: Dr. Carolina Tesi-Rocha
Study Coordinator: Sonia Pathak, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Sarepta Therapeutics, Inc
Purpose: Long term follow-up study of safety and efficacy of SRP-9001
Status: Recruiting by invitation only
Open label extension study of AOC 1044 in Healthy Adult Volunteers and Participants with Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping (EXPLORE44 OLE)
Protocol ID: 72283
NCT06244082
PI: Dr. Carolina Tesi-Rocha
Study Coordinator: Natalia Wieczorek, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avidity Biosciences
Purpose: Assessing the safety and efficacy of AOC1044 in DMD
Status: Active, no longer recruiting
Wearable Technology to Assess Gait Function in SMA and DMD
NCT: 74765
PI: Dr. John Day
Study Coordinator: Rabia Farooquee, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Assess gait function using AI technology
Status: Recruiting in clinic
Summit Vamorolone Registry
Protocol ID: 78275
NCT: N/A
PI: Dr. Jenna Klotz
Study Coordinator: Michelle Jepsen, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Observational study of boys ages 2 and older with DMD taking vamorolone
Status: Recruiting soon
Charcot-Marie Tooth Disease (CMT)
Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Type (CMT1B), 2A (CMT2A), 4A (CMT4A), 4C (CMT4C), and Others
Protocol ID: 50330
NCT01193075
PI: Dr. Maxwell Greene
Study Coordinator: Michelle Jepsen, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: NIH and MDA
Purpose: Natural history study of CMT
Status: Recruiting in clinic
A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J (CMT4J)
Protocol ID: 74511
NCT06151600
PI: Dr. John W. Day
Study Coordinator: Lin Karman, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Elpida Therapeutics
Purpose: Natural history study of CMT4J
Status: Recruiting
Facioscapulohumeral Muscular Dystrophy (FSHD)
Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)
Protocol ID: 57072
NCT04635891
PI: Dr. John W. Day
Study Coordinator: Sabrina Salvatore, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: NIH and MDA
Purpose: Motor Outcomes to Validate Evaluations in FSHD
Status: Recruiting
A Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, and Pharmacokinetics of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FORTITUDE OLE)
Protocol ID: 74674
PI: Dr. John W. Day
Study Coordinator: Nikita Sehgal, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avidity Biosciences
Purpose: Open label extension study of safety and efficacy of AOC-1020 in adults with FSHD
Status: Recruiting by invitation only
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FORWARD, or FORTITUDE-3)
Protocol ID: 80414
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Nikita Sehgal, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avidity Biosciences
Purpose: Assessing the safety and efficacy of AOC-1020 in adults with FSHD
Status: Recruiting
Recruitment Notes: Ages 16-70 with diagnosis of FSHD
A Phase 1, Double-blinded, Randomized, Dose-repeating, Placebo-controlled, Cross-over Study to Assess the Safety and Preliminary Efficacy of Allogeneic ULSC on Disease Severity in Facioscapulohumeral Muscular Dystrophy (Restem)
Protocol ID: 77579
NCT07086521
PI: Dr. John W. Day
Study Coordinator: Sarah Ismail, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Restem and Solve FSHD
Purpose: Assessing safety and efficacy of stem cell treatment in FSHD
Status: Recruiting
Recruitment Notes: Participants 15 years or older with genetically confirmed FSHD1 or FSHD2
GNE myopathy
Observational study of adults with GNE myopathy
Protocol ID: 77271
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Sabrina Salvatore, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Natural history study and development of improved measures of muscle involvement of GNE myopathy in adults
Status: Recruiting
Recruitment Notes: Adults with GNE ages 18-65
IBM
A Study to Evaluate the Efficacy and Safety of ABC008 for Inclusion Body Myositis
Protocol ID: 68761
NCT05721573
PI: Dr. Neelam Goyal
Study Coordinator: Nikita Sehgal, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Abcuro, Inc.
Purpose: Assessing the safety and efficacy of ABC008 in adults with IBM
Status: Active, no longer recruiting
An Open-label, Multicenter Study to Evaluate the Long-term Safety and Efficacy of Ulviprubart (ABC008) in Subjects Who Have Completed a Trial of Ulviprubart for the Treatment of Inclusion Body Myositis
Protocol ID: 78504
NCT: N/A
PI: Dr. Neelam Goyal
Study Coordinator: Raiye Hailu, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Abcuro, Inc.
Purpose: Open label extension trial assessing the safety and efficacy of ABC008 in adults with IBM
Status: Recruiting by invitation only
Multifocal Motor Neuropathy (MMN)
A Study to Assess the Efficacy and Safety of Empasiprubart Versus IVIg in Adults with Multifocal Motor Neuropathy (EMPASSION)
Protocol ID: 79238
NCT: N/A
PI: Dr. Srikanth Muppidi
Study Coordinator: Sonia Pathak, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Argenx INC
Purpose: Assessing the safety and efficacy of Empasiprubart versus IVIg in adults with MMN
Status: Recruiting
Myasthenia Gravis (MG)
A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell Therapy, in Subjects with Refractory Generalized Myasthenia Gravis
Protocol ID: 74139
NCT06193889
PI: Dr. Srikanth Muppidi
Study Coordinator: Susan Thomas, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Kyverna Therapeutics Inc.
Purpose: Assessing safety and efficacy of CAR-T treatment in gMG
Status: Active, no longer recruiting
A Phase 1 Study of Anitocabtagene Autoleucel for the Treatment of Subjects with Non-Oncology Plasma Cell-related Diseases
Protocol ID: 78298
NCT06626919
PI: Dr. Srikanth Muppidi
Study Coordinator: Susan Thomas, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: ArcellX Inc.
Purpose: Assessing safety and efficacy of anitocabtagene autoleucel in MG
Status: Recruiting
Myotonic dystrophy (DM)
A natural history investigating clinical outcomes and quality of life in children with Myotonic Dystrophy (ANIQ)
Protocol ID: 78905
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Sarah Ismail, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Natural history study of congenital and childhood DM1 in participants ages 0-24
Status: Recruiting soon
Stanford outcome validation with functional measures in myotonic dystrophy (MYOCAP/Actimyo)
Protocol ID: 62522
NCT: N/A
PI: Dr. Tina Duong
Study Coordinator: Sarah Ismail, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Outcome validation with functional measures in myotonic dystrophy
Status: Recruiting
Stanford study of central nervous system involvement in DM1 (DDC)
Protocol ID: 74320
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Lin Karman, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Characterization of neurological symptoms and biomarkers of DM1
Status: Recruiting
Recruitment Notes: Adults with DM1 and their unaffected family members
Global Study of Del-desiran for the Treatment of DM1 (HARBOR)
Protocol ID: 74759
NCT06411288
PI: Dr. John W. Day
Study Coordinator: Rabia Farooquee, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avidity Biosciences
Purpose: Assessing the safety and efficacy of Delpacibart etedesiran in people with DM1
Status: Active, no longer recruiting
A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1 (HARBOR OLE)
Protocol ID: 79538
NCT07008469
PI: Dr. John W. Day
Study Coordinator: Natalia Wieczorek, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avidity Biosciences
Purpose: Assessing the long-term safety and efficacy of Delpacibart etedesiran in people with DM1
Status: Recruiting by invitation only
Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
Protocol ID: 65435
NCT: NCT03981575
PI: Dr. Jacinda Sampson
Study Coordinator: Sarah Ismail, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Status: Active, no longer recruiting
A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple-Ascending Dose Study of PGN-EDODM1 in Adult Participants with Myotonic Dystrophy Type 1 (FREEDOM2-DM1)
Protocol ID: 75989
NCT06667453
PI: Dr. Jacinda Sampson
Study Coordinator: Wendy Ao, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: PepGen Inc.
Purpose: Assessing the safety and efficacy of PGN-EDODM1 in adults with DM1
Status: Recruiting
Recruitment Notes: Participants must have a diagnosis of DM1 and be 16-60 years old
A Phase 1/2, Randomized, Double-blind, Placebo‑controlled Single- and Multiple‑dose Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VX‑670 in Adult Subjects with Myotonic Dystrophy Type 1 (GALILEO)
Protocol ID: 73893
NCT06185764
PI: Dr. Jacinda Sampson
Study Coordinator: Sonia Pathak, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Vertex Pharmaceuticals
Purpose: Evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses
Status: Recruiting
Recruitment Notes: Participants must have a diagnosis of DM1 and be 18-60 years old
An Open-label Extension Study Evaluating the Long-term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects with Myotonic Dystrophy Type I (GALILEO OLE)
Protocol ID: 81692
NCT: TBD
PI: Dr. Jacinda Sampson
Study Coordinator: Sonia Pathak, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Vertex Pharmaceuticals
Purpose: Evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses
Status: Recruiting soon by invitation only
A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents with Congenital Myotonic Dystrophy (REACH CDM)
Protocol ID: 61610
NCT03692312
PI: Dr. John W. Day
Study Coordinator: Rabia Farooquee, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: AMO Pharmaceuticals
Purpose: Long term follow up study determining safety and efficacy of Tideglusib in congenital DM1
Status: Active, no longer recruiting
Pompe disease (PD)
Pompe Disease Registry
Protocol ID: 12372
NCT00231400
PI: Dr. John W. Day
Study Coordinator: Meng Yao, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Genzyme/Sanofi
Purpose: To collect information on the subjects with rare diseases like Pompe Disease and other lysosomal storage disorders longitudinally
Status: Recruiting in clinic
Recruitment Notes: All Stanford patients diagnosed with Pompe disease
A Phase 1/2, Open-Label, Ascending-Dose Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT845, an AAV8-Delivered Gene Transfer Therapy in Patients with Late Onset Pompe Disease (FORTIS)
Protocol ID: 53753
NCT04174105
PI: Dr. John W. Day
Study Coordinator: Habib Mofakham Fini, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Astellas Pharma Inc
Purpose: Determine the safety and efficacy of AT845 in adults with Pompe disease
Status: Active, no longer recruiting
Spinal muscular atrophy (SMA)
A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants with Spinal Muscular Atrophy Who Previously Participated in an Investigational Study with Nusinersen (ONWARD)
Protocol ID: 59527
NCT04729907
PI: Dr. John W. Day
Study Coordinator: Raiye Hailu, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen
Purpose: Assessing the long-term safety and efficacy of higher doses of Spinraza in SMA
Status: Recruiting by invitation only
A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants with Spinal Muscular Atrophy Previously Treated with Risdiplam (ASCEND)
Protocol ID: 62798
NCT05067790
PI: Dr. John W. Day
Study Coordinator: Lidia Choniawko, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen
Purpose: Assessing safety and efficacy of higher dose of Spinraza in SMA
Status: Active, no longer recruiting
Clinical Study of Spinal Muscular Atrophy (iSMAC/APNCR)
Protocol ID: 31140
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Shelby Vogt-Domke, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: SMA Foundation, CureSMA, Biogen
Purpose: To study the natural history of SMA, and response to disease-modifying treatments
Status: Recruiting in clinic
SMA pregnancy outcomes and chart review study
Protocol ID: 77364
NCT: N/A
PI: Dr. Jacinda Sampson
Study Coordinator: Shelby Vogt-Domke, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: CureSMA, Biogen
Purpose: To study pregnancy health and outcomes for women with SMA
Status: Recruiting
Recruitment Notes: Women with SMA who are pregnant or were previously pregnant
Long-Term Safety & Efficacy of Apitegromab in Patients with SMA Who Completed Previous Trials of Apitegromab (ONYX)
Protocol ID: 68228
NCT05626855
PI: Dr. Carolina Tesi Rocha
Study Coordinator: Natalia Wieczorek, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Scholar Rock
Purpose: Long term safety and efficacy of Apitegromab
Status: Recruiting by invitation only
A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
Protocol ID: 52085
NCT05626855
PI: Dr. John W Day
Study Coordinator: Wendy Ao, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avexis/Novartis
Purpose: Assessing the long-term effects of AVXS-101
Status: Active, no longer recruiting
Adult SMA Exploratory study (ASE)
Protocol ID: 55518
NCT: N/A
PI: Dr. Tina Duong
Study Coordinator: Shelby Vogt-Domke, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen
Purpose: Observational study and MRI of adults with SMA clinically treated with nusinersen or risdiplam
Status: Recruiting
Safety and Performance of the ThecaFlex DRx™ System Port and Catheter for Chronic Intrathecal Access, Cerebrospinal Fluid (CSF) Aspiration, and Delivery of Nusinersen in Spinal Muscular Atrophy (SMA) Patients Resistant to Lumbar Puncture (PIERRE) Trial
Protocol ID: N/A
NCT05866419
PI: Dr. John W. Day
Study Coordinator: Habib Mofakham Fini, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Alcyone Therapeutics
Purpose: Testing an implantable device for delivery of nusinersen
Status: Recruiting
Recruitment Notes: Patients with SMA, ages 3+ who are resistant to lumbar puncture
An Open Label, Single Cohort Study to Assess the Pharmacokinetic Profile of Nusinersen (BIIB058) Administered via the ThecaFlex DRx™ System (PIERRE-PK)
Protocol ID: N/A
NCT06555419
PI: Dr. John W. Day
Study Coordinator: Habib Mofakham Fini, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen
Purpose: Determining how the body processes nusinersen treatment when delivered through an implantable device
Status: Recruiting
Recruitment Notes: Participants must be enrolled in the PIERRE trial
A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old with Spinal Muscular Atrophy (SMA) (OPAL)
Protocol ID: 80599
NCT07047144
PI: Dr. Carolina Tesi Rocha
Study Coordinator: Wendy Ao, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Scholar Rock Inc
Purpose: Assessing safety and efficacy of apitegromab in children <2 with SMA
Status: Recruiting
Recruitment Notes: Age <2 with SMA diagnosis, previously received SMA gene therapy or continuing to receive nusinersen or risdiplam
A Randomized, Double-blind, Placebo-controlled, Multinational, Multicenter study with open-label treatment extension to assess the effect of Min-102 on the progression of adrenomyeloneuropathy in male patients with x-linked adrenoleukodystrophy
PI: Jacinda Sampson, MD
NCT03231878
Study Status: Closed to Enrollment
Research Coordinator: Lila Perrone
Contact: Perronel@stanford.edu
Stroke and Neurocritical Care Clinical Research Group
BOOST3 – Brain Oxygen Optimization in Severe TBI Phase 3
BOOST3 is a study to learn if either of two strategies for monitoring and treating patients with traumatic brain injury (TBI) in the intensive care unit (ICU) is more likely to help them get better. More information: https://med.stanford.edu/neurology/divisions/neurocriticalcare/clinical-trials/boost3.html
Protocol ID: 49130
PI: Karen G. Hirsch, MD
Study Coordinator: Arely Campos-Melendez
Status: RECRUITING
POSTICECAP: Patterns of Survivors’ Recovery Trajectories in the ICECAP Trial
This study will describe the extent of improvement or deterioration in functional, cognitive, and health related quality of life outcomes within months after an out of hospital cardiac arrest (OHCA) and will estimate the prospective associations of clinical interventions, rehabilitation, and social determinants with those dimensions of recovery in a large, well characterized, racially ethnically diverse, US representative cohort of OHCA patients.
Protocol ID: 75020
PI: Karen Hirsch, MD
Study Coordinator: Arely Campos-Melendez
Status: RECRUITING
PRECISE - Perfusion imaging to identify posterior circulation candidates for thrombectomy
The purpose of this research is to find out if advanced brain imaging can help identify which patients who have suffered an ischemic stroke (a blockage of blood flow) to the back of their brain are most likely to benefit from a procedure to remove the blood clot to restore the blood flow to the brain.
Protocol ID: Pro00053806
PI: Gregory Albers, MD
Study Coordinator: Irina Eyngorn
Status: Recruitment closed
CRISP 2 - CT Perfusion to Predict Response to Recanalization in Ischemic Stroke Project
The purpose of this research is to analyze brain imaging data and clinical data to understand how stroke progresses through the brain.
Protocol ID: 56227
PI: Maarten Lansberg, MD, PhD
Study coordinator: Camila Krause
Status: RECRUITING
SATURN - Statins Use in Intracerebral Hemorrhage Patients
This research is being done to find out if it is better to continue or discontinue statin drugs (a type of cholesterol-lowering medication) in people who had a brain hemorrhage (bleeding in the brain) while taking such a statin drug. It will also examine if having certain genes (apolipoproteine E) influences the likelihood of getting a brain hemorrhage while taking a statin drug.
Protocol ID: 55536
PI: Chitra Venkatasubramanian, MD
Study coordinator: Nhi Nguyen
Status: RECRUITING
ASPIRE: Anticoagulation in ICH (intracerebral hemorrhage) Survivors for Stroke Prevention and Recovery
The purpose of this study is to compare the effects of a drug called apixaban with aspirin in patients with atrial fibrillation (irregular heart beating) and a recent brain hemorrhage (bleeding in the brain) to see which is better in preventing strokes and death.
NCT03907046
IRB #: 52983
PI: Chitra Venkatasubramanian, MD
Study coordinator: Nhi Nguyen
Status: RECRUITING
CLUTCH: Clevidipine for the Antihypertensive Treatment of Acute Intracerebral Hemorrhage
The purpose of this study is to compare the rate of hypertensive subjects with intracerebral hemorrhage (ICH) who reach systolic blood pressure (SBP) target with stability within 60 minutes of enrollment, among patients treated with IV clevidipine with those treated with alternate IV antihypertensive regimen. The study is designed to observe the effects of medications commonly used to reduce blood pressure as per national guidelines for stroke care. It aims to compare different hospital protocols for lowering blood pressure.
IRB #: 84247
PI: Chitra Venkatasubramanian, MD
Study coordinator: Camila Krause
Status: RECRUITING
Librexia Stroke Study: Efficacy and Safety of Milvexian, an Oral Factor XIa Inhibitor, for Stroke Prevention after an Acute Ischemic Stroke or High-Risk Transient Ischemic Attack
The purpose of this research study is to see if an experimental drug, called milvexian in addition to standard of care, is safe and useful in reducing the risk of future ischemic stroke in participants after ischemic stroke or transient ischemic attack compared to placebo (tablet with no active drug) in addition to standard of care.
IRB #: 70999
PI: Nirali Vora, MD
Study coordinator: Riddhi Shah
Status: Recruitment closed
Clarity: Cilostazol for Prevention of Recurrent Stroke
The primary goal of this trial is to determine if the addition of cilostazol to standard of care treatment with aspirin or clopidogrel reduces the risk of major adverse cardiovascular events in patients with a history of a non-cardioembolic ischemic stroke or high-risk TIA.
IRB #: 86070
PI: Nirali Vora, MD
Study coordinator: Riddhi Shah
Status: RECRUITING SOON
CIRCA Registry: The CIRCA Chronotype and Stroke Registry: A Multicenter Study of the Association of Chronotype with Presentation, Course, and Outcome of Acute Cerebral Ischemia
The goal of this study is to figure out how natural sleep patterns, known as "chronotype," affect the course of stroke and stroke treatment.
IRB #: 72264
PI: Gregory Albers, MD
Study Coordinator: Diana Gonzalez
Status: RECRUITING
Sodium imaging Study: Ischemic Brain Damage & Triple/Single Quantum Sodium MRI
The objective of this study is to use a new method of Magnetic Resonance Imaging (MRI) to identify the sodium concentration in brain tissue in patients with a stroke. We hope that this new method may be helpful in assessing the outcomes of potential therapies for acute stroke.
IRB #: 64720
PI: Fernando Boada, PhD
Study coordinator: Irina Eyngorn
Status: Recruitment closed
StrokeCog
The purpose of this observational study is to understand the long-term effects of stroke on a person’s memory and thinking. To determine this, we will schedule stroke patients to come in on a yearly basis for memory testing and collection of a small amount of blood.
Protocol ID: 42089
PI: Marion Buckwalter, MD, PhD
Study Coordinator: Chastin Chung
Status: ACTIVE, NOT RECRUITING
StrokeCog-BBB
The purpose of this observational study is to learn about cognitive outcomes in stroke patients understand the long-term effects of stroke on a person’s memory and thinking, and how this may relate to the blood brain barrier. To determine this, participants will undergo yearly cognitive testing and MRIs two years apart. Researchers will compare cognitive outcomes in non-stroke patients who have cardiovascular risk factors to understand the effects of stroke on these outcomes.
Protocol ID: 65450
PI: Marion Buckwalter, MD, PhD
Study Coordinator: Chastin Chung
STATUS: RECRUITING CONTROLS
OUTER LIMITS 24: Endovascular Therapy for Large Core Patients with Uncertain Response to Thrombectomy
The purpose of this study is to assess the safety and efficacy of thrombectomy in both an early and extended time window to patients with a very large ischemic stroke.
IRB #: 81016
PI: Gregory Albers, MD
Study coordinator: Irina Eyngorn
Status: RECRUITING
STEP: StrokeNet Thrombectomy Endovascular Platform
STEP Platform Domain A EVT Indication Expansion: LVO Mild Deficit/Low NIH Stroke Scale Score Strata
This study is being done to learn whether adding an immediate clot removal procedure, along with standard medical treatment, is better for stroke patients that have a blockage of a large sized blood vessel (artery) in the brain, but with mild stroke symptoms.
STEP Platform Domain A EVT Indication Expansion: Medium Vessel Occlusion Strata
This study is being done to learn whether adding a clot removal procedure, along with standard medical treatment, is better for stroke patients that have a blockage of a medium sized blood vessel (artery) in the brain.
IRB #: 76081
PI: Maarten Lansberg, MD, PhD
Study coordinator: Nhi Nguyen
Status: RECRUITING
TESTED: Treatment with Endovascular Intervention for Stroke Patients with Existing Disability
This is a prospective observational study in which the purpose is to compare the risks and benefits of endovascular thrombectomy (EVT) to medical management (no EVT) in ischemic stroke patients who have a blockage in one of the large blood vessels in the brain and have a moderate-to-severe disability prior to their stroke. Using several assessment tools, the study team will gather information on the participants' daily functioning ability before and after the stroke.
IRB #: 82468
PI: Maarten Lansberg, MD, PhD
Study coordinator: Samantha Rhodes
Status: RECRUITING
ReMEDy2: Phase 2/3 Adaptive Design, Randomized Double-blind Placebo-controlled Study to Evaluate the Safety and Efficacy of DM199 for the Treatment of Acute Ischemic Stroke
The purpose of this study is to see if an experimental drug called DM199 (rinvecalinase alfa) is safe and effective in treating patients with moderate stroke severity, who present within 24 hours of Acute Ischemic Stroke (AIS) onset due to small and medium vessel occlusions. This study focuses on participants who have limited treatment options.
IRB #: 79072
PI: Christina Mijalski, MD
Study coordinator: Nhi Nguyen
Status: RECRUITING
cAPPricorn-1
The purpose of this study is to evaluate the efficacy, safety, tolerability, and pharmacodynamics (PD) of intrathecally (IT) administered ALN-APP in adult patients with Cerebral Amyloid Angiopathy (CAA).
IRB #: 76787
PI: Chitra Venkatasubramanian, MD
Study coordinator: Camila Krause
Status: ACTIVE, NOT RECRUITING
Neuro-Oncology Clinical Research Group
Newly Diagnosed Glioma
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Optune® (TTFields, 200 kHz) concomitant with Maintenance Temozolomide and Pembrolizumab Versus Optune® Concomitant with Maintenance Temozolomide and Placebo for the Treatment of Newly Diagnosed Glioblastoma (EF-41/KEYNOTE D58)
PI: Lawrence Recht, MD
Research Coordinator: Sahara Rout
Contact: ssrout@stanford.edu
ClinicalTrials.gov#: NCT06556563
Phase II Safusidenib Erbumine in Patients With Isocitrate Dehydrogenase 1 (IDH1) Mutant Glioma
PI: Seema Nagpal, MD
Research Coordinator: Lewis Naya
Contact: lnaya@stanford.edu
ClinicalTrials.gov#: NCT05303519
Recurrent High Grade Glioma
Phase I Clinical Trial of Locoregionally (LR) Delivered Autologous B7-H3 Chimeric Antigen Receptor T Cells (B7-H3CART) in Adults with Recurrent Glioblastoma Multiforme (GBM)
PI: Reena Thomas, MD PhD
ClinicalTrials.gov#: NCT05474378
A Phase I Study of Multiple Doses of Neural Stem Cell-Based Oncolytic Virotherapy (NSC-CRAd-S-pk7) Administered Intracerebrally to Patients with Recurrent High Grade Gliomas
PI: Melanie Hayden Gephart, MD
ClinicalTrials.gov#: NCT05139056
H3K27M Mutant Glioma
Phase 1 Clinical Trial of Autologous GD2 Chimeric Antigen Receptor (CAR) T Cells (GD2CART) for Diffuse Intrinsic Pontine Gliomas (DIPG) and Spinal Diffuse Midline Glioma (DMG)
PI: Michelle Monje, MD PhD
ClinicalTrials.gov #: NCT04196413
Brain and Spine Metastases
A Randomized Phase III TriaL Comparing SingLe- Versus Multi-Fraction Spine STereotActic Radiosurgery for Patients with Spinal Metastases (ALL-STAR)
PI: Erqi Pollum, MD
ClinicalTrials.gov#: NCT06173401
For questions, please contact: merkel@stanford.edu or jmaala@stanford.edu
LGG
Newly Diagnosed
ACNS1833: A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine Versus Selumetinib (NSC# 748727, IND# 77782) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) Not Associated with BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)
Diagnosis: Low-Grade Glioma (LGG)
Recurrent/Refractory/Progressive
There are currently no open trials
DIPG
Newly Diagnosed
GD2 CAR-T: Phase 1 Clinical Trial of Autologous GD2 Chimeric Antigen Receptor (CAR) T cells (GD2CART) for Diffuse Intrinsic Pontine Gliomas (DIPG) and Spinal Diffuse Midline Glioma (DMG)
Diagnosis: Diffuse Intrinsic Pontine Glioma (DIPG) and Spinal Cord Diffuse Midline Glioma (DMG) that is H3K27 mutated
Recurrent/Refractory/Progressive
PEPN2111: A Phase 1/2 Trial of CBL0137 (NSC# 825802, IND# 155843) in Patients with Relapsed or Refractory Solid Tumors including CNS Tumors and Lymphoma
Diagnosis: Progressive or Recurrent DIPG
HGG
Newly Diagnosed
ACNS1821: A Phase 1/2 Trial of Selinexor (KPT-330) and Radiation Therapy in Newly-Diagnosed Pediatric Diffuse Intrinsic Pontine Glioma (DIPG) and High-Grade Glioma (HGG)
Diagnosis: High-Grade Glioma (HGG) / ONLY HGG Stratum Open
Recurrent/Refractory/Progressive
There are currently no open trials
Ependymoma
Newly Diagnosed
There are currently no open trials
Recurrent/Refractory/Progressive
There are currently no open trials
MEDULLOBLASTOMA
Newly Diagnosed
SJiMB21: Phase 2 Study of Molecular and Clinical Risk-Directed Therapy for Infants and Young Children with Newly Diagnosed Medulloblastoma
Diagnosis: Medulloblastoma
Recurrent/Refractory/Progressive
BCC016: DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed Medulloblastoma
Diagnosis: Relapsed Medulloblastoma
ATRT
Newly Diagnosed
There are currently no open trials
Recurrent/Refractory/Progressive
There are currently no open trials
Neurofibromatosis-1 (NF1)
Newly Diagnosed
There are currently no open trials
Recurrent/Refractory/Progressive
Natural History of Neurofibromatosis Type 1-Associated Non-Optic Pathway Glioma to Support the Development of Evidence-Based Criteria for Surveillance and Initiation of Treatment
Diagnosis: NF1 NOT associated with Optic Pathway Glioma
Other: Non-Treatment
NF1-OPG Natural History Study: Developing Evidence-Based Criteria for Initiating Treatment for Neurofibromatosis Type 1 Associated Optic Pathway Glioma/CTF 004: The Collection of Blood Samples from Patients with NF1 for Research Purposes
Diagnosis: NF1 associated with Optic Pathway Glioma
NF1 Patient Registry
Diagnosis: NF
OTHER STUDIES & MOLECULAR BASED THERAPIES
ACNS2321: A Phase II Trial Evaluating Chemotherapy followed by Response-Based Reduced Radiation Therapy for Patients with Central Nervous System Germinomas
Diagnosis: Newly diagnosed germinoma
ACNS2021: A Phase 2 Trial of Chemotherapy followed by Response-Based Whole Ventricular & Spinal Canal Irradiation (WVSCI) for Patients with Localized Non-Germinomatous Central Nervous System Germ Cell Tumor
Diagnosis: Newly diagnosed with localized primary CNS NGGCT of the suprasellar and/or pineal region
G042286: A Phase I/II, Open-Label, Multicenter, Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Alectinib in Pediatric Participants With ALK Fusion-Positive Solid or CNS Tumors for Whom Prior Treatment Has Proven to be Ineffective or for Whom There is No Satisfactory Treatment Available
Diagnosis: CNS or solid tumors harboring ALK gene fusions
APEC14B1: The Project: Everychild Protocol: A Registry, Eligibility, Screening, Biology, and Outcome Study
Diagnosis: Registry or eligibility arm
FURTHER INFORMATION AND SUMMARIES FOR HEALTH CARE PROFESSIONALS
Pediatric Clinical Research Group
For questions, please contact: merkel@stanford.edu or jmaala@stanford.edu
LGG
Newly Diagnosed
ACNS1833: A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine Versus Selumetinib (NSC# 748727, IND# 77782) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) Not Associated with BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)
Diagnosis: Low-Grade Glioma (LGG)
Recurrent/Refractory/Progressive
There are currently no open trials
DIPG
Newly Diagnosed
GD2 CAR-T: Phase 1 Clinical Trial of Autologous GD2 Chimeric Antigen Receptor (CAR) T cells (GD2CART) for Diffuse Intrinsic Pontine Gliomas (DIPG) and Spinal Diffuse Midline Glioma (DMG)
Diagnosis: Diffuse Intrinsic Pontine Glioma (DIPG) and Spinal Cord Diffuse Midline Glioma (DMG) that is H3K27 mutated
Recurrent/Refractory/Progressive
PEPN2111: A Phase 1/2 Trial of CBL0137 (NSC# 825802, IND# 155843) in Patients with Relapsed or Refractory Solid Tumors including CNS Tumors and Lymphoma
Diagnosis: Progressive or Recurrent DIPG
HGG
Newly Diagnosed
ACNS1821: A Phase 1/2 Trial of Selinexor (KPT-330) and Radiation Therapy in Newly-Diagnosed Pediatric Diffuse Intrinsic Pontine Glioma (DIPG) and High-Grade Glioma (HGG)
Diagnosis: High-Grade Glioma (HGG) / ONLY HGG Stratum Open
Recurrent/Refractory/Progressive
There are currently no open trials
Ependymoma
Newly Diagnosed
There are currently no open trials
Recurrent/Refractory/Progressive
There are currently no open trials
MEDULLOBLASTOMA
Newly Diagnosed
SJiMB21: Phase 2 Study of Molecular and Clinical Risk-Directed Therapy for Infants and Young Children with Newly Diagnosed Medulloblastoma
Diagnosis: Medulloblastoma
Recurrent/Refractory/Progressive
BCC016: DFMO as Maintenance Therapy for Molecular High/Very High Risk and Relapsed Medulloblastoma
Diagnosis: Relapsed Medulloblastoma
ATRT
Newly Diagnosed
There are currently no open trials
Recurrent/Refractory/Progressive
There are currently no open trials
Neurofibromatosis-1 (NF1)
Newly Diagnosed
There are currently no open trials
Recurrent/Refractory/Progressive
Natural History of Neurofibromatosis Type 1-Associated Non-Optic Pathway Glioma to Support the Development of Evidence-Based Criteria for Surveillance and Initiation of Treatment
Diagnosis: NF1 NOT associated with Optic Pathway Glioma
Other: Non-Treatment
NF1-OPG Natural History Study: Developing Evidence-Based Criteria for Initiating Treatment for Neurofibromatosis Type 1 Associated Optic Pathway Glioma/CTF 004: The Collection of Blood Samples from Patients with NF1 for Research Purposes
Diagnosis: NF1 associated with Optic Pathway Glioma
NF1 Patient Registry
Diagnosis: NF
OTHER STUDIES & MOLECULAR BASED THERAPIES
ACNS2321: A Phase II Trial Evaluating Chemotherapy followed by Response-Based Reduced Radiation Therapy for Patients with Central Nervous System Germinomas
Diagnosis: Newly diagnosed germinoma
ACNS2021: A Phase 2 Trial of Chemotherapy followed by Response-Based Whole Ventricular & Spinal Canal Irradiation (WVSCI) for Patients with Localized Non-Germinomatous Central Nervous System Germ Cell Tumor
Diagnosis: Newly diagnosed with localized primary CNS NGGCT of the suprasellar and/or pineal region
G042286: A Phase I/II, Open-Label, Multicenter, Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Alectinib in Pediatric Participants With ALK Fusion-Positive Solid or CNS Tumors for Whom Prior Treatment Has Proven to be Ineffective or for Whom There is No Satisfactory Treatment Available
Diagnosis: CNS or solid tumors harboring ALK gene fusions
APEC14B1: The Project: Everychild Protocol: A Registry, Eligibility, Screening, Biology, and Outcome Study
Diagnosis: Registry or eligibility arm
FURTHER INFORMATION AND SUMMARIES FOR HEALTH CARE PROFESSIONALS
Pediatric studies
All Neuromuscular Conditions
Subject Database and Specimen Repository for Neuromuscular and Neurodegenerative Disorders
Protocol ID: 23888
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Veronica Schnyer, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: This is a study that involves collecting clinical information of subjects (patients with any neurological condition or their close family member) and tissue samples to develop a recruitment database and tissue bank, which will be of great value in helping the investigators learn more about various related neurological conditions.
Status: Recruiting
Determination of Standards for Maximal and Submaximal Exercise testing for Individuals with Neuromuscular Disease (CPET)
Protocol ID: 54078
NCT: N/A
PI: Dr. Tina Duong
Study Coordinator: Sabrina Salvatore, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Understanding exercise testing which will assess how heart and lungs work together to get oxygen to muscles in children and adults with muscular dystrophies
Status: Recruiting
Recruitment Notes: All clinic patients ages 9+ with diagnosed neuromuscular disorders who are able to perform testing on cycle ergometer as well as controls
A6MCT Testing the feasibility and efficacy of the 6-minute assisted cycle test in non-ambulatory neuromuscular patients
Protocol ID: N/A
NCT: N/A
PI: Dr. Tina Duong
Study Coordinator: Audrey Chun, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Status: Recruiting
OpenCAP Video technology to assess timed function tests and gait quality
Protocol ID: N/A
NCT: N/A
PI: Dr. Tina Duong
Study Coordinator: Sarah Ismail, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Status: Recruiting
Addressing Immunotoxicities to AAV Gene Therapy for DMD and Other Disorders
Protocol ID: 79348
NCT: N/A
PI: Dr. John W. Day and Dr. Bradley Hamilton
Study Coordinator: TBD, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose:
Status: Recruiting
Amyotrophic Lateral Sclerosis (ALS)
Clinical Procedures to Support Research in ALS (CAPTURE-ALS)
Protocol ID: 54467
NCT03489278
PI: Dr. Maxwell Greene
Study Coordinator: Raiye Hailu, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: NINDS/NIH, MDA
Purpose: Natural History Study of ALS and PLS
Status: Recruiting in clinic
Refining the Patient-Ranked Order of Function in People Living with ALS (PROOF)
Protocol ID: 75570
NCT: N/A
PI: Dr. Maxwell Greene
Study Coordinator: Raiye Hailu, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: DOD and Dutch Research Council
Purpose: Natural History Study of ALS, interview-based study
Status: Recruiting
A Phase 1-3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION363 in Amyotrophic Lateral Sclerosis Patients with Fused in Sarcoma Mutations (FUSION)
Protocol ID: 59619
NCT04768972
PI: Dr. John W. Day
Study Coordinator: Habib Mofakham Fini, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: IONIS Pharmaceuticals
Purpose: Assessing the safety and efficacy of ION363 in children and adults with ALS
Status: Active, no longer recruiting
Recruitment Notes: Confirmed genetic mutation in FUS, ages 16-65
Expanded Access Program to ION363 FUS ALS treatment
Protocol ID: N/A
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Habib Mofakham Fini, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Expanded Access Program
Status: Recruiting on case-by-case basis
Briefer Cognitive Behavioral Screen in ALS
Protocol ID: 77489
NCT: N/A
PI: Drs. Maxwell Greene and Ying Lee
Study Coordinator: Raiye Hailu, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Status: Recruiting
Becker and Duchenne muscular dystrophy (BMD/DMD)
A Gene Delivery Study to Evaluate the Safety of and Expression From SRP-9001 in Duchenne Muscular Dystrophy (DMD) (ENDEAVOR)
Protocol ID: 59448
NCT04626674
PI: Dr. John W. Day
Study Coordinator: Raiye Hailu, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Sarepta Therapeutics, Inc
Purpose: Assessing the safety and efficacy of SRP-9001 gene therapy in individuals with DMD
Status: Recruiting
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants with Duchenne Muscular Dystrophy (DMD) (ENVISION)
Protocol ID: 64248
NCT05881408
PI: Dr. Carolina Tesi-Rocha
Study Coordinator: Raiye Hailu, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Sarepta Therapeutics, Inc
Purpose: Assessing the safety and efficacy of SRP-9001 gene therapy in individuals with DMD
Status: Active, no longer recruiting
Long-term follow-up study for all SRP-9001 studies (EXPEDITION)
Protocol ID: 71344
NCT05967351
PI: Dr. Carolina Tesi-Rocha
Study Coordinator: Sonia Pathak, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Sarepta Therapeutics, Inc
Purpose: Long term follow-up study of safety and efficacy of SRP-9001
Status: Recruiting by invitation only
Open label extension study of AOC 1044 in Healthy Adult Volunteers and Participants with Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping (EXPLORE44 OLE)
Protocol ID: 72283
NCT06244082
PI: Dr. Carolina Tesi-Rocha
Study Coordinator: Natalia Wieczorek, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avidity Biosciences
Purpose: Assessing the safety and efficacy of AOC1044 in DMD
Status: Active, no longer recruiting
A Phase 1/2 Open-label, Dose Escalation and Dose Expansion Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Intravenous RGX-202 Gene Therapy in Males with Duchenne Muscular Dystrophy (DMD) (AFFINITY DUCHENNE)
Protocol ID: 52155
NCT05693142
PI: Dr. Carolina Tesi-Rocha
Study Coordinator: Lidia Choniawko, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: REGENXBIO
Purpose: Assessing the safety and efficacy of RGX-202 gene therapy in children with DMD
Status: Recruiting
Recruitment Notes: Boy ages 4-12, ambulatory, DMD mutations in exons 18 or higher
A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-202 Gene Therapy in Males with Duchenne Muscular Dystrophy (AFFINITY LTFU)
Protocol ID: 81214
NCT: N/A
PI: Dr. Carolina Tesi-Rocha
Study Coordinator: Natalia Wieczorek, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: REGENXBIO
Purpose: Assessing the safety and efficacy of RGX-202 gene therapy in children with DMD
Status: Recruiting by invitation only
Wearable Technology to Assess Gait Function in SMA and DMD
NCT: 74765
PI: Dr. John Day
Study Coordinator: Rabia Farooquee, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Assess gait function using AI technology
Status: Recruiting in clinic
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal Injection of INS1201 in Ambulatory Males with Duchenne Muscular Dystrophy (Insmed ASCEND)
Protocol ID: 78652
NCT06817382
PI: Dr. John W. Day
Study Coordinator: Rabia Farooquee, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Insmed Pharmaceuticals Inc.
Purpose: Assessing the safety and efficacy of INS1201 in boys with DMD
Status: Recruiting
Recruitment Notes: Boys with DMD, age 2 years
Summit Vamorolone Registry
Protocol ID: 78275
NCT: N/A
PI: Dr. Jenna Klotz
Study Coordinator: Michelle Jepsen, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Observational study of boys ages 2 and older with DMD taking vamorolone
Status: Recruiting soon
Congenital muscular dystrophy (CMD)
Clinical Trial Readiness for Children 0-5 years with Congenital Muscular Dystrophy Secondary to LAMA2 Mutations (READY CMD)
Protocol ID: 75022
NCT: N/A
PI: Dr. Carolina Tesi Rocha
Study Coordinator: Lin Karman, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Observational study in children ages 0-5 with LAMA2-related CMD
Status: Recruiting
Charcot-Marie Tooth Disease (CMT)
Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Type (CMT1B), 2A (CMT2A), 4A (CMT4A), 4C (CMT4C), and Others
Protocol ID: 50330
NCT01193075
PI: Dr. Maxwell Greene
Study Coordinator: Michelle Jepsen, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: NIH and MDA
Purpose: Natural history study of CMT
Status: Recruiting in clinic
A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J (CMT4J)
Protocol ID: 74511
NCT06151600
PI: Dr. John W. Day
Study Coordinator: Lin Karman, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Elpida Therapeutics
Purpose: Natural history study of CMT4J
Status: Recruiting
Facioscapulohumeral Muscular Dystrophy (FSHD)
Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)
Protocol ID: 57072
NCT04635891
PI: Dr. John W. Day
Study Coordinator: Sabrina Salvatore, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: NIH and MDA
Purpose: Motor Outcomes to Validate Evaluations in FSHD
Status: Recruiting
Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE PEDS)
Protocol ID: 80456
PI: Dr. Carolina Tesi Rocha
Study Coordinator: Lin Karman, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: NIH
Purpose: Motor Outcomes to Validate Evaluations in FSHD
Status: Recruiting soon
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FORWARD, or FORTITUDE-3)
Protocol ID: 80414
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Nikita Sehgal, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avidity Biosciences
Purpose: Assessing the safety and efficacy of AOC-1020 in adults with FSHD
Status: Recruiting
Recruitment Notes: Ages 16-70 with diagnosis of FSHD
A Phase 1, Double-blinded, Randomized, Dose-repeating, Placebo-controlled, Cross-over Study to Assess the Safety and Preliminary Efficacy of Allogeneic ULSC on Disease Severity in Facioscapulohumeral Muscular Dystrophy (Restem)
Protocol ID: 77579
NCT07086521
PI: Dr. John W. Day
Study Coordinator: Sarah Ismail, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Restem and Solve FSHD
Purpose: Assessing safety and efficacy of stem cell treatment in FSHD
Status: Recruiting
Recruitment Notes: Participants 15 years or older with genetically confirmed FSHD1 or FSHD2
Myasthenia Gravis (MG)
An Open-Label Uncontrolled Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Activity of Nipocalimab in Children Aged 2 to less than 18 years with Generalized Myasthenia Gravis
Protocol ID: 64037
NCT05265273
Dr. Carolina Tesi-Rocha
Study Coordinator: TBD, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Janssen
Purpose: Assessing the safety and efficacy of Nipocalimab in children with gMG
Status: Recruiting
Recruitment Notes: Children with gMG ages 2-18 with suboptimal response to current stable therapy
Myotonic dystrophy (DM)
A natural history investigating clinical outcomes and quality of life in children with Myotonic Dystrophy (ANIQ)
Protocol ID: 78905
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Sarah Ismail, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Natural history study of congenital and childhood DM1 in participants ages 0-24
Status: Recruiting soon
Stanford outcome validation with functional measures in myotonic dystrophy (MYOCAP/Actimyo)
Protocol ID: 62522
NCT: N/A
PI: Dr. Tina Duong
Study Coordinator: Sarah Ismail, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Outcome validation with functional measures in myotonic dystrophy
Status: Recruiting
Global Study of Del-desiran for the Treatment of DM1 (HARBOR)
Protocol ID: 74759
NCT06411288
PI: Dr. John W. Day
Study Coordinator: Rabia Farooquee, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avidity Biosciences
Purpose: Assessing the safety and efficacy of Delpacibart etedesiran in people with DM1
Status: Active, no longer recruiting
A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1 (HARBOR OLE)
Protocol ID: 79538
NCT07008469
PI: Dr. John W. Day
Study Coordinator: Natalia Wieczorek, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avidity Biosciences
Purpose: Assessing the long-term safety and efficacy of Delpacibart etedesiran in people with DM1
Status: Recruiting by invitation only
A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple-Ascending Dose Study of PGN-EDODM1 in Adult Participants with Myotonic Dystrophy Type 1 (FREEDOM2-DM1)
Protocol ID: 75989
NCT06667453
PI: Dr. Jacinda Sampson
Study Coordinator: Wendy Ao, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: PepGen Inc.
Purpose: Assessing the safety and efficacy of PGN-EDODM1 in adults with DM1
Status: Recruiting
Recruitment Notes: Participants must have a diagnosis of DM1 and be 16-60 years old
A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents with Congenital Myotonic Dystrophy (REACH CDM)
Protocol ID: 61610
NCT03692312
PI: Dr. John W. Day
Study Coordinator: Rabia Farooquee, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: AMO Pharmaceuticals
Purpose: Long term follow up study determining safety and efficacy of Tideglusib in congenital DM1
Status: Active, no longer recruiting
Nemaline Myopathy
Nemaline Myopathy Natural History Study (NM-CTRN)
Protocol ID: 72180
PI: Dr. Carolina Tesi Rocha
Study Coordinator: Sarah Ismail, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: A Foundation Building Strength
Purpose: Natural history study of Nemaline Myopathy
Status: Recruiting soon
Recruitment Notes: Children with Nemaline Myopathy
Pompe disease (PD)
Pompe Disease Registry
Protocol ID: 12372
NCT00231400
PI: Dr. John W. Day
Study Coordinator: Meng Yao, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Genzyme/Sanofi
Purpose: To collect information on the subjects with rare diseases like Pompe Disease and other lysosomal storage disorders longitudinally
Status: Recruiting in clinic
Recruitment Notes: All Stanford patients diagnosed with Pompe disease
Spinal muscular atrophy (SMA)
A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants with Spinal Muscular Atrophy Who Previously Participated in an Investigational Study with Nusinersen (ONWARD)
Protocol ID: 59527
NCT04729907
PI: Dr. John W. Day
Study Coordinator: Raiye Hailu, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen
Purpose: Assessing the long-term safety and efficacy of higher doses of Spinraza in SMA
Status: Recruiting by invitation only
A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants with Spinal Muscular Atrophy Previously Treated with Risdiplam (ASCEND)
Protocol ID: 62798
NCT05067790
PI: Dr. John W. Day
Study Coordinator: Lidia Choniawko, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen
Purpose: Assessing safety and efficacy of higher dose of Spinraza in SMA
Status: Active, no longer recruiting
Clinical Study of Spinal Muscular Atrophy (iSMAC/APNCR)
Protocol ID: 31140
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Shelby Vogt-Domke, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: SMA Foundation, CureSMA, Biogen
Purpose: To study the natural history of SMA, and response to disease-modifying treatments
Status: Recruiting in clinic
SMA pregnancy outcomes and chart review study
Protocol ID: 77364
NCT: N/A
PI: Dr. Jacinda Sampson
Study Coordinator: Shelby Vogt-Domke, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: CureSMA, Biogen
Purpose: To study pregnancy health and outcomes for women with SMA
Status: Recruiting
Recruitment Notes: Women with SMA who are pregnant or were previously pregnant
Long-Term Safety & Efficacy of Apitegromab in Patients with SMA Who Completed Previous Trials of Apitegromab (ONYX)
Protocol ID: 68228
NCT05626855
PI: Dr. Carolina Tesi Rocha
Study Coordinator: Natalia Wieczorek, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Scholar Rock
Purpose: Long term safety and efficacy of Apitegromab
Status: Recruiting by invitation only
A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
Protocol ID: 52085
NCT05626855
PI: Dr. John W Day
Study Coordinator: Wendy Ao, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avexis/Novartis
Purpose: Assessing the long-term effects of AVXS-101
Status: Active, no longer recruiting
Safety and Performance of the ThecaFlex DRx™ System Port and Catheter for Chronic Intrathecal Access, Cerebrospinal Fluid (CSF) Aspiration, and Delivery of Nusinersen in Spinal Muscular Atrophy (SMA) Patients Resistant to Lumbar Puncture (PIERRE) Trial
Protocol ID: N/A
NCT05866419
PI: Dr. John W. Day
Study Coordinator: Habib Mofakham Fini, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Alcyone Therapeutics
Purpose: Testing an implantable device for delivery of nusinersen
Status: Recruiting
Recruitment Notes: Patients with SMA, ages 3+ who are resistant to lumbar puncture
An Open Label, Single Cohort Study to Assess the Pharmacokinetic Profile of Nusinersen (BIIB058) Administered via the ThecaFlex DRx™ System (PIERRE-PK)
Protocol ID: N/A
NCT06555419
PI: Dr. John W. Day
Study Coordinator: Habib Mofakham Fini, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen
Purpose: Determining how the body processes nusinersen treatment when delivered through an implantable device
Status: Recruiting
Recruitment Notes: Participants must be enrolled in the PIERRE trial
A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients with Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy (HINALEA 2)
Protocol ID: 74740
NCT05861999
PI: Dr. John W. Day
Study Coordinator: TBD, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Roche
Purpose: Assessing safety and efficacy of risdiplam treatment after SMA gene therapy
Status: Recruiting
Recruitment Notes: Age <2 with previous SMA gene therapy treatment with plateau or decline
A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old with Spinal Muscular Atrophy (SMA) (OPAL)
Protocol ID: 80599
NCT07047144
PI: Dr. Carolina Tesi Rocha
Study Coordinator: Wendy Ao, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Scholar Rock Inc
Purpose: Assessing safety and efficacy of apitegromab in children <2 with SMA
Status: Recruiting
Recruitment Notes: Age <2 with SMA diagnosis, previously received SMA gene therapy or continuing to receive nusinersen or risdiplam
Investigate Efficacy and Safety of Carisbamate as Adjunctive Treatment for Seizures Associated With LGS in Children and Adults
NCT05219617
PI: Brenda Porter, MD
Research Coordinator: Jeilo Gauna childneuroresearch@stanford.edu
Status: Enrolling
Autism Spectrum Disorder (ASD) and Intellectual Disability (ID) Determinants in Tuberous Sclerosis Complex (TSC)
NCT02461459
PI: Brenda Porter, MD
Research Coordinator: Jeilo Gauna childneuroresearch@stanford.edu
Status: Active not recruiting
Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study (TSC-STEPS)
NCT05104983
PI: Brenda Porter, MD
Research Coordinator: Rayann Solidum childneuroresearch@stanford.edu
Status: Active not recruiting
Open-Label Continuation Treatment Study with Oral MC-1 for the Treatment of Patients with PNPO Deficiency
NCT04706013
PI: Ann Hyslop Segeren, MD
Research Coordinator: Rayann Solidum childneuroresearch@stanford.edu
Status: Active not recruiting
A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathies (DEE)
NCT06719141
PI: Ann Hyslop Segeren, MD
Research Coordinator: Jeilo Gauna childneuroresearch@stanford.edu
Status: Active and Recruiting
A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)
NCT06660394
PI: Ann Hyslop Segeren, MD
Research Coordinator: Jeilo Gauna childneuroresearch@stanford.edu
Status: Active and Recruiting
A Phase 3 Open-Label Study to Investigate LP352 in Children and Adults with Developmental and Epileptic Encephalopathy
PI: Ann Hyslop Segeren, MD
Research Coordinator: Jeilo Gauna childneuroresearch@stanford.edu
Status: Active and Recruiting
Multinational, Multicenter Study With an Open-Label Phase 1b and a Randomized, Double-Blind, Placebo-Controlled Phase 3 Followed by an Open-Label Extension to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Radiprodil in Participants With GRIN-Related Neurodevelopmental Disorder
NCT07224581
PI: Emily Spelbrink, MD
Research Coordinator: Rayann Solidum childneuroresearch@stanford.edu
Status: Active and Recruiting
STXBP1 and SYNGAP1 Related Disorders Natural History Study
NCT06555965
PI: Juliet Knowles, MD, PhD
Research Coordinator: Rayann Solidum childneuroresearch@stanford.edu
Status: Active and Recruiting
The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network
NCT03047369
PI: Keith Van Haren, MD
Research Coordinator: Jeilo Gauna childneuroresearch@stanford.edu
Status: Active
Safety and Efficacy Study of Cenobamate in Pediatric Subjects 2-17 Years of Age with Partial-onset (focal) Seizures
NCT05067634
PI: William Brian Gallentine, MD
Research Coordinator: Rayann Solidum childneuroresearch@stanford.edu
Status: Active not recruiting
Focal Cerebral Arteriopathy Steroid Trial (FOCAS)
NCT06040255
PI: Sarah Lee
Research Coordinator: Jeilo Gauna childneuroresearch@stanford.edu
Status: Active
Adult Clinical Trials
For questions about clinical trials please contact our research team at neurotrials@stanford.edu.
Pediatric Clinical Trials
To find out the current status of any pediatric study please contact us at: arushi.gehani@stanford.edu
For pediatric neuro-oncology studies, please contact: Neuroonc@stanfordchildrens.org