Natural History Study of Synucleinopathies
PI: Mitchell Miglis, MD
Study Status: Open to enrollment
Research Coordinator: Ruba Shaik or Jordan Seliger
Contact: rubas@stanford.edu or jseliger@stanford.edu

A Phase 3, 4-Week, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of TD-9855 in Treating Symptomatic Neurogenic Orthostatic Hypotension in Subjects With Primary Autonomic Failure
PI: Dong-In Sinn, MD
NCT03750552
Study Status: Open to enrollment
Research Coordinator: Ruba Shaik or Jordan Seliger
Contact: rubas@stanford.edu or jseliger@stanford.edu

A Phase 3, 22-week, Multi-center, Randomized Withdrawal Study of TD˗9855 in Treating Symptomatic Neurogenic Orthostatic Hypotension in Subjects with Primary Autonomic Failure
PI: Dong-In Sinn, MD
NCT03829657
Study Status: Open to enrollment
Research Coordinator: Ruba Shaik or Jordan Seliger
Contact: rubas@stanford.edu or  jseliger@stanford.edu

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of BHV-3241 in Subjects With Multiple System Atrophy
PI: Mitchell Miglis, MD
NCT03952806
Study Status: Closed to enrollment
Research Coordinator: Ruba Shaik or Jordan Seliger
Contact: rubas@stanford.edu or  jseliger@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: Jordan Seliger
Contact: jseliger@stanford.edu

RNS System Responsive Thalamic Stimulation for Primary Generalized Seizures Study (NAUTILUS Study)
NCT05147571
Study Status: Enrolling Soon
Research Coordinator: Jordan Seliger
Contact: jseliger@stanford.edu

Medtronic Deep Brain Stimulation (DBS) Therapy for Epilepsy Post-Approval Study (EPAS)
PI: Kevin Graber, MD
NCT03900468
Study Status: Enrolling
Research Coordinator: Jordan Seliger
Contact: jseliger@stanford.edu

Maternal Outcomes and Neurodevelopmental Effects of Antiepileptic Drugs (MONEAD)
PI: Kimford Meador, MD
NCT01730170
Study Status: Closed to Enrollment
Research Coordinator: Jordan Seliger
Contact: jseliger@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: Jordan Seliger
Contact: jseliger@stanford.edu

Electroencephalographic Biomarkers of Neuro-Cognitive Function
PI: Kimford Meador, MD
Study Status: Enrolling
Research Coordinator: Jordan Seliger
Contact: jseliger@stanford.edu

Using Artificial Intelligence to Choose the Optimal Antiepileptic Drug
Protocol ID : 46059
PI: Robert S. Fisher, MD, PhD
Status: Enrolling subjects

Transcranial Direct Current Stimulation to Treat Epilepsy
Protocol ID : 47711
PI: Robert S. Fisher, MD, PhD
Status: Enrolling subjects             

Focused ultrasound to treat epilepsy (EP001)
Protocol ID : 55981
PI: Robert S. Fisher, MD, PhD
Status: Enrolling subjects

AI-Guided 3D Video Analysis for Seizure Detection
Protocol ID : 53035
PI: Robert S. Fisher, MD, PhD
Status: Pending loosening of COVID travel requirements

Cognitive Effects of Vinpocetine in Patients with Epilepsy
PI: Kimford Meador, MD
NCT02011971
Study Status: Closed to Enrollment
Research Coordinator: Jordan Seliger
Contact: jseliger@stanford.edu

Effects of Titration Rate on Cognitive and Behavioral Side Effects of Perampanel
PI: Kimford Meador, MD
NCT# Pending
Study Status: Enrolling
Research Coordinator: Jordan Seliger
Contact: jseliger@stanford.edu

RNS® System Post-Approval Study in Epilepsy Clinical Investigational Plan (CIP)
PI: Babak Razavi
NCT02403843
Study Status: Closed to enrollment
Research Coordinator: Jordan Seliger, Ruba Shaik
Contact: jseliger@stanford.edu or rubas@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

Interventional, randomized, double-blind, parallel-group, placebo-controlled delayed-start study to evaluate the efficacy and safety of eptinezumab in patients with episodic Cluster Headache (Alleviate)
PI: Addie Peretz, MD
NCT04688775
Study Status: Enrolling
Contact: neurotrials@stanford.edu

Sunstar: The Stanford Neuroscience Headache Biorepository
PI: Robert Cowan, MD, FAAN
NCT03231241
Study Status: Enrolling
Contact: neurotrials@stanford.edu

Efficacy of ‘Green Glasses’ to Reduce Headache Symptoms in Individuals with Migraine Disorders
PI: Robert Cowan, MD, FAAN
Study Status: Not Yet Enrolling

A prospective, multi-centre, randomized, double-blind, sham-controlled, parallel-group, group-sequential study to investigate safety and effectiveness of the Rehaler partial rebreathing device, in adults suffering from migraine with aura: PAREMA1
PI: Robert Cowan, MD, FAAN
Study Status: Not Yet Enrolling

For patient’s referral please reach out to:
Kaila Sevilla @ kailas44@stanfod.edu
Pragya Tripathi @pragyat@stanford.edu

Active, Open for Enrollment

CELIA Trial
Sponsor: Biogen MA, Inc.
Intervention: BIIB080
Indication:MCI and Dementia
Age: 50-80 Years old
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: NCT05399888
PI: Irina Skylar-Scott, MD
Contact: Olivia Lu; Olivialu@stanford.edu;  650-374-9286

Who can participate:

• Ability of the participant and/or his/her legally authorized representative (e.g., parent, spouse, or legal guardian), as appropriate and applicable, to understand the purpose and risks of the study, to provide informed consent, and to authorize the use of confidential health information in accordance with national and local privacy regulations.
• Male and females aged 50 to 80 years, inclusive, at the time of informed consent.
• All women of childbearing potential and all men must practice highly effective contraception during the study and for 52 weeks after their last dose of study treatment. In addition, participants should not donate sperm or eggs during the study and for at least 52 weeks after their last dose of study treatment.
• Must meet all of the clinical criteria for MCI due to AD or mild AD dementia and must have the following at Screening Visit 1:
• RBANS Delayed Memory Index score of ≤ 85, indicative of objective evidence of memory impairment.
• CDR global score of 0.5 for MCI due to AD or 0.5 or 1 for mild AD dementia.
• MMSE score of 22 to 30 (inclusive).
• CDR Memory Box score of ≥ 0.5.
• Evidence of amyloid pathology as measured by PET or CSF sampling.
•  Confirmation of evidence of cerebral Aβ accumulation, as determined by a centrally read amyloid PET scan of the brain or by CSF testing. Any of the following amyloid PET tracers may be used to confirm cerebral Aβ pathology on PET: 18Fflorbetapir, 18Fflutemetamol, or 18Fflorbetaben. A previously obtained amyloid PET scan within approximately 24 months of Screening Visit 1 is permissible for participants and must be submitted to the central imaging vendor to confirm that study inclusion criteria are met. A previously obtained CSF confirmation of amyloid positivity is not sufficient to meet this criterion.

Note: If a participant enters the study based on a cerebral Aβ positivity as determined by a previous amyloid PET scan, the PET result takes precedence for purposes of determining eligibility.

• Must consent to APOE genotyping. The participant’s APOE status may be disclosed to him/her at the Investigator’s discretion.
• Must have 1 care partner who, in the Investigator’s judgment, has frequent and sufficient contact with the participant (at least 10 hours/week) as to be able to provide accurate information about the participant’s cognitive and functional abilities. The care partner must agree to accompany the participant to clinic visits and/or be available by telephone at designated times to provide information to the Investigator and study staff about the participant (and to attend in-person clinic visits that require care partner input for scale completion) and must agree to monitor the participant’s administration of any prescribed medications. A care partner should be available for the duration of the study, and the participation of the same care partner for the duration of the study is encouraged. The care partner must be literate and provide informed consent.
• Apart from a clinical diagnosis of MCI due to AD or mild AD dementia, the participant must be in good health as determined by the Investigator, based on medical history and screening assessments.
   

AHEAD 3-45 Trial
Sponsor: Eisai and NIH (AHEAD 3-45 Study)
Intervention: BAN2401 (monoclonal antibody binding to amyloid)
Indication: Pre-clinical AD
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: NCT04468659
PI: Sharon Sha, MD
Contact: Jade Perry; jadep@stanford.edu; 650-521-7287
Study Link : https://studypages.com/s/the-ahead-study-440395/

Who can participate

1. Male or female, age 55 to 80 years inclusive at the time of informed consent.

a. Those 55 to 64 must have 1 of the following additional risk factors, given the relatively low rates of amyloid positivity <65 years:

i. First degree relative diagnosed with dementia onset before age 80, or

ii. Known to possess at least 1 apolipoprotein є4 variant (APOE4) allele, or

iii. Known before Screening to have elevated brain amyloid according to previous PET or CSF testing. Individuals with historical amyloid PET scans with Aβi (eg, from preclinical AD studies such as A4 or EARLY) are eligible to be screened, provided the subject did not participate in any clinical studies involving anti-amyloid therapies subsequent to the PET assessment.

2. Global CDR score of 0 at Screening

3. Mini Mental State Examination (MMSE) score ≥27 (with educational adjustments) at Screening

4. Wechsler Memory Scale-Revised Logical Memory subscale II (WMS-R LM II) score at Screening of ≥6

5. A45 Trial: Elevated brain amyloid pathology by amyloid PET: defined as approximately >40 centiloids on Screening scan.

A3 Trial: Intermediate levels of brain amyloid pathology by amyloid PET: defined as approximately 20 to 40 centiloids on screening scan.

6. Has a study partner that is willing to participate as a source of information and has approximately weekly contact with the participant (contact can be in-person, via telephone or electronic communication). The study partner must have sufficient contact such that the investigator feels the study partner can provide meaningful information about the subject’s daily function.

7. Provide written informed consent

8. Willing and able to comply with all aspects of the protocol

New IDEAS Trial
Sponsor: American College of Radiology(New IDEAS)
Intervention: N/A - Observational
Indication: MCI and dementia
Brief Summary: New IDEAS: Imaging Dementia—Evidence for Amyloid Scanning Study
A Study to Improve Precision in Amyloid PET Coverage and Patient Care
Clinicaltrials.gov identifier: NCT02420756
PI: Sharon Sha, MD
Contact:Warsameh Bulhan ;whbulhan@stanford.edu;650-460-4151

Who can participate:

• Medicare beneficiary with Medicare as primary insurance.
• Meets clinical criteria for Mild Cognitive Impairment (MCI) or Dementia as defined by the 2018 National Institute on Aging – Alzheimer’s Association Research Framework
• Brain MRI and/or CT within 24 months prior to enrollment.
• Clinical laboratory assessment (complete blood count [CBC], comprehensive metabolic panel [CMP], thyroid stimulating hormone [TSH], vitamin B12) within the 12 months prior to enrollment.
• Able to tolerate amyloid PET required by protocol, to be performed at a participating PET facility.
• English or Spanish speaking (for the purposes of informed consent);
• Willing and able to provide consent. Consent may be by proxy.
• Neuropsychiatric syndrome can be classified into “clinically typical” or “clinically atypical” categories.

Cognition (Shimmer) Trial
Sponsor: Cognition Therapeutics Inc.
Intervention: CT1812
Indication: Mild or moderate Dementia with Lewy Bodies
Age: 50-85 years old
Brief Summary: A Randomized, Double-blind, Placebo-controlled, Phase 2, 6-month Study to Evaluate the Safety, Tolerability and Exploratory Efficacy of CT1812 in Subjects with Mild to Moderate Dementia with Lewy Bodies
Clinicaltrials.gov identifier: NCT05225415
PI: Sharon Sha, MD
Contact: Stephanie Tran; trans@stanford.edu; 650-521-7287

Who can participate

1) Subjects or their Legally Authorized Representative (LAR) (or designee as applicable by local law) must provide written informed consent to the study procedures prior to any study procedures.

2) Subjects must have a caregiver/ study partner who in the opinion of the site principal investigator, has contact with the study subject for a sufficient number of hours per week to provide informative responses on the protocol assessments, oversee the administration of study drug, and is willing and able to participate in all clinic visits and some study assessments. The caregiver/ study partner must provide written informed consent to participate in the study.

3) Men or women 50-85 years of age (inclusive), meeting criteria for probable DLB .

4) Men willing to comply with acceptable form of contraception or women of non-childbearing potential as defined below:

 i) Non-childbearing potential for women is defined as postmenopausal (last natural menses greater than 24 months) or undergone a documented bilateral tubal ligation or hysterectomy. If last natural menses less than 24 months, a serum FSH value confirming post-menopausal status can be employed.

 ii) Male subjects who are sexually active with a woman of childbearing potential must agree to use condoms during the trial and for 3 months after last dose unless the woman is using an acceptable means of birth control. Acceptable forms of birth control include abstinence, birth control pills, or any double combination of: intrauterine device (IUD), male or female condom, diaphragm, sponge, and cervical cap.

5) MRI (or CT scan due to contraindication of MRI, if approved by medical monitor) obtained during screening consistent with the clinical diagnosis of DLB and without findings of significant exclusionary abnormalities. An historical MRI (or CT scan), up to 1 year prior to screening, may be used as long as there is no history of intervening neurologic disease or clinical events (such as a stroke, head trauma etc.) and the subject is without clinical symptoms or signs suggestive of such intervening events.

 6) MMSE 18-27 inclusive.

7) If receiving acetylcholinesterase inhibitors (AChEI), memantine or a combination of the two, must have been on a stable dose for at least 12 weeks before the screening visit, with no plans for dose adjustment during the study. Treatment-naive subjects can be entered into the study, but there should be no plans to initiate treatment with AChEIs or memantine from Screening to the end of the study.

8) Stable pharmacological treatment of any other chronic conditions for at least 30 days prior to screening.

9) Formal education of eight or more years.

10) Subjects living at home or in an assisted living facility.

11) Ability to swallow capsules.

12) Must consent to apolipoprotein E (ApoE) genotyping.

13) Subjects shall be generally healthy with mobility (ambulatory or ambulatory-aided, i.e., walker or cane), vision and hearing (hearing aid permissible) sufficient for compliance with testing procedures.

14) Must be able to complete all screening evaluations.

LEADS Trial
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)
Age: 40-64 years old
Brief Summary: A natural history, non-treatment study designed to look at disease progression in individuals with early onset cognitive impairment (less than 65 year old).
Clinicaltrials.gov identifier: NCT03507257
PI: Sharon Sha, MD
Contact: Stephanie Tran; trans@stanford.edu; 650-521-7287

Who can participate

Cognitively impaired (EOAD and EOnonAD) Cohorts Only:

• Meets NIA-AA criteria for MCI due to AD or probable AD dementia
• Have a global CDR score ≤ _1.0
• Have capacity to provide informed consent (IC) or has a legal authorized representative or guardian who provides IC
• Age between 40-64 years (inclusive) at the time of consent
• Must have a study partner (informant) who spends a minimum average of 10 hours per week with the participant (e.g., family member, significant other, friend, caregiver) who is generally aware of the participants’ daily activities and can provide information about the participant’s cognitive and functional performance. If the participant does not have a study partner who spends at least 10 face-to-face hours per week, other arrangements for identifying a viable study partner will be granted on a case-by-case basis by the Site PI
• Willing and able to complete longitudinal study procedures aside from LP which is an optional procedure
• Not pregnant or lactating. Women must be two years post-menopausal, be surgically sterile, or have a negative pregnancy test prior to each PET scan
• Fluent in English or Spanish

Cognitively Normal (CN) Cohort Only:

• Meets criteria for cognitively normal, based on an absence of significant impairment in cognitive functions or activities of daily living
• Have a global CDR score = 0
• Have capacity to provide informed consent
• Have a Mini-Mental State Exam score between 26-30 (inclusive). Exceptions may be made for participant with less than 8 years of education at the discretion of the Site PI
• Age between 40-64 years (inclusive) at the time of consent
• Must have a study partner (informant) who spends a minimum average of 10 hours per week with the participant (e.g., family member, significant other, friend, caregiver) who is generally aware of the participants’ daily activities and can provide information about the participant’s cognitive and functional performance. If the participant does not have a study partner who spends 10 face-to-face hours per week, other arrangements for identifying a viable study partner will be granted on a case-by-case basis by the Site PI
• Willing and able to complete longitudinal study procedures aside from LP which is an optional procedure
• Not pregnant or lactating. Women must be two years post-menopausal, be surgically sterile, or have a negative pregnancy test prior to each PET scan
• Fluent in English or Spanish

Coming soon in December 2023

EIP Pharma DLB Phase 2bTrial:
Sponsor: EIP Pharma, Inc.
Intervention: Neflamapimod
Indication: Dementia with Lewy Bodies
Age: ≥55 years
Brief Summary:  A Phase 2b Clinical Study of the P38 Alpha Kinase Inhibitor Neflamapimod in Patients with Dementia with Lewy Bodies (DLB)
Clinicaltrials.gov identifier: NCT04001517
PI: Sharon Sha, MD
Contact: Kaila Sevilla; kailas44@stanford.edu; 650- 454-5458

Who can Participate:

1. Men and women aged ≥55 years.

2. Subject or subject’s legally authorized representative is willing and able to provide written informed consent.

3. Probable DLB by consensus criteria (McKeith et al, 2017), including a positive DaTscan™. If the DaTscan is negative, but the subject has historical polysomnography (PSG)-verified REM sleep behavioral disorder (RBD), this will also qualify as probable DLB.

4. CDR Global Score 0.5 or 1.0 during Screening.

5. If the patient is currently receiving cholinesterase inhibitor therapy, the patient must have received such therapy for greater than 3 months and on a stable dose for at least 6 weeks at the time of randomization. Except for reducing the dose for tolerability reasons, the dose of cholinesterase inhibitor may not be modified during the study. If the patient is not currently receiving cholinesterase inhibitor therapy, but received such therapy previously, that therapy must have been discontinued at least 3 months prior to randomization. Memantine therapy is allowed, if it had been started at least 3 months prior to randomization and the patients is also receiving cholinesterase inhibitor therapy (memantine monotherapy, i.e., without concomitant cholinesterase inhibitor therapy, is excluded).

6. Normal or corrected eyesight and auditory abilities, sufficient to perform all aspects of the cognitive and functional assessments.

7. No history of learning difficulties that may interfere with their ability to complete the cognitive tests.

8. Received vaccination for SARS-CoV-19, unless medical contraindications prevent being vaccinated.

9. Must have reliable informant or caregiver.

ENVISION Trial
Sponsor: Biogen MA. Inc.
Intervention: Aducanumab(221AD305)
Indication: MCI due to Alzheimer’s disease or mild Alzheimer’s disease dementia.
Age: 60-85 years old
Brief Summary: A Phase 3b/4 Randomized, Double-Blind, Placebo-Controlled, Parallel- Group Study to Verify the Clinical Benefit of Aducanumab (BIIB037) in Participants with Alzheimer's Disease
Clinicaltrials.gov identifier: NCT05310071
PI: Sharon Sha, MD
Contact: Olivia Lu; Olivialu@stanford.edu; 650-374-9286
Jade Perry; jadep@stanford.edu; 650-521-7287

Who can participate:

1. The participant and/or his/her legally authorized representative (e.g., parent, spouse, or legal guardian, where local regulations and institutional practices permit) must be able, as appropriate and applicable, to understand the purpose and risks of the study, to provide signed and dated informed consent, and to authorize the use of confidential health information in accordance with national and local privacy and ethics regulations.
2. The participant must be 60 to 85 years old, inclusive, at the time of informed consent.
3. All women of childbearing potential must practice effective contraception during the study and for 5 times the half-life or 24 weeks (whichever is longer) after their last dose of study treatment.
4. The participant must have a minimum of 9 years of education or vocational training or the equivalent education/vocational training until the age of 15 or, per Investigator judgment, work experience that indicates a lack of mental deficits other than early-stage dementia.
5. The participant must have confirmed amyloid beta pathology by CSF (historical CSF test results not allowed) or amyloid PET. If providing only a screening amyloid PET scan for amyloid positivity, a historical, amyloid PET scan obtained within 18 months of Screening Visit 2 is permissible. Sponsor-approved tracers must be used and scans must be submitted to the central imaging vendor to confirm that study inclusion criteria are met. In the case of discordant results between CSF and PET, PET results will be used to assess eligibility.
6. The participant must have a history of subjective memory decline with gradual onset and slow progression over the last 6 months before Screening, confirmed by study partner.
7. The participant must meet all of the following clinical criteria for MCI due to Alzheimer’s disease or mild Alzheimer’s disease:
   1. Have an MMSE score between 22 and 30 inclusive
   2. Have a CDR memory score ≥ 0.5
   3. Have a CDR-GS of 0.5 or 1.0
   4. Have an RBANS score of 85 or lower indicative of objective cognitive impairment (based upon the DMI score)
8. The participant must be in good health, apart from a clinical diagnosis of early Alzheimer’s disease, as determined by the Investigator based on medical history and screening assessments.
9. The participant must consent to ApoE genotyping.
10. The participant must have 1 informant/care partner who, in the Investigator’s opinion, has frequent and sufficient contact with the participant (at least 10 hours/week in person or by phone) as to be able to provide accurate information about the participant’s cognitive and functional abilities over time. The informant/care partner ideally has known the participant prior to their cognitive decline to have a reference point for change across time. The informant/care partner must be available by phone to provide information to the Investigator and study staff about the participant as well as agree to attend in-person clinic visits that require partner input for scale completion. The informant/care partner must be literate and provide informed consent and should be available for the duration of the study. The same informant/care partner is required to be consistent across all study visits except under rare, unavoidable circumstances (e.g., unexpected informant health crisis) that are approved by the Investigator and Sponsor.

ALZ-NET Trial
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
Age: ≥18 years
Brief Summary: Patient registry for Alzheimer's Network for Treatment and Diagnostics (ALZ-NET)
Clinicaltrials.gov identifier: N/A
PI: Kyan Younes, MD
Contact: Kaila Sevilla; kailas44@stanford.edu; 650- 454-5458

Who can participate:

1. 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 to use protected health information (PHI) in accordance with national and local patient privacy regulations.

2. Patient is at least 18 years of age at the time of informed consent.

3. Patient has memory concerns and/or may have diagnosis of Alzheimer’s disease (AD) and has been identified by an approved site investigator (as defined by protocol) to be appropriate for treatment with novel FDA-approved AD therapies in real world clinical practice.

4. Patient meets appropriate label designations according to appropriate use recommendations for novel FDA-approved AD therapy/therapies.

5. 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.

The Role of the Gut Microbiome in Parkinson’s Disease
Studies show that the gut microbiome in Parkinson’s patients is different from the gut microbiome in healthy individuals. What is not clear, however, is how these differences impact one’s risk for developing Parkinson’s Disease or how the different microbiome in Parkinson’s patients impacts disease course. Additionally, in Parkinson’s patients there is chronic systemic inflammation due to excessive immune activity, and the gut microbiome is known to impact immune functioning. Therefore, in this study we examine whether the differences in the microbiome of Parkinson’s patients are associated with increased inflammatory markers in the blood, and whether the differences in microbiome impact immune cell activation. Through this study, we hope to better understand the mechanism by which the microbiome in Parkinson’s patients may be contributing to the disease. In the future, these findings could be used to develop disease treatments that target the microbiome. We are recruiting individuals who have been diagnosed by a neurologist with Parkinson’s Disease and healthy individuals from the same house. Participation entails answering an online questionnaire and giving small amounts of blood and stool. Each participant will receive a gift card for their involvement.
PI: Bianca Palushaj, MD
Co-PI: Ami S. Bhatt, MD, PhD
Study status: Open, enrollment ongoing
Research Coordinators: Meena Chakraborty, Erin Brooks, Gabriella Green
Contact: mchakra@stanford.edu, efbrooks@stanford.edu, gzmg1@stanford.edu

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

Pacific Udall Center
Sponsor: NIH/NINDS Morris K. Udall Center of Excellence for Parkinson's Disease Research
PI: Tom Montine, MD, PhD
Study status: Open, Enrollment ongoing
Research coordinator: Hannah Schmitz
hschmitz@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

M-STAR
Sponsor: Biohaven
Site PI: Mitchell Miglis, MD
Study status: Closed for enrollment
Research coordinator:  Ruba Shaik
rubas@stanford.edu 650-546-1436

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

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 Coordinator: Julia Sumera, jsumera@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
NCT04047628
PI: Jeffrey Dunn, MD
Research Coordinator: Crystal Ton-Nu, ctonnu@stanford.edu
Status: Enrolling

A Multiple-center, Non-Randomized, Open-Label, Adaptive, Single Ascending Dose Phase I Study to Investigate the Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of RO7121932 Following Intravenous Administration in Patients with Multiple Sclerosis
NCT05704361
PI: Christopher Lock, MD
Research Coordinator: Safiyyah Bachar, sbachar@stanford.edu
Status: Enrolling

High Dimensional Analysis of Immune Subsets with Mass Cytometry and Multiparameter Flow Cytometry in Patients with Secondary Progressive Multiple Sclerosis Treated with Siponimod
PI: Lawrence Steinman MD
Research Coordinator: Julia Sumera, jsumera@stanford.edu
Status: Enrolling

Ozanimod (Zeposia) in Multiple Sclerosis and Ulcerative Colitis: High Dimensional Immunometabolomic Analysis of Immune Cell Subsets with CyToF, CITE-seq, and Multiparameter Flow Cytometry In Treated Individuals Compared with Baseline.
PI: Lawrence Steinman MD
Research Coordinator: Julia Sumera, jsumera@stanford.edu
Status: Enrolling

A Phase III Multicenter, Randomized, Double-Blind, Double-Dummy, Parallel-Group Study to Evaluate the Efficacy and Safety of Fenebrutinib Compared with Teriflunomide in Adult Patients with Relapsing Multiple Sclerosis (GN42272)
NCT04586023
PI: Lucas Kipp MD
Research Coordinator: Julia Sumera, jsumera@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: Tara Sarkar, tarats@stanford.edu
Status: Enrolling

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)
NCT05063162
PI: May Han, MD
Research Coordinator: Tara Sarkar, tarats@stanford.edu
Status: Enrolling

Retinal Biomarkers of Inflammation and Degeneration in Multiple Sclerosis
PI: Heather Moss, MD
Research Coordinator: Tara Sarkar, tarats@stanford.edu
Status: Enrolling

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: Paxton Ataide, 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. This also includes a Biobank
Status: Recruiting

Tissue Banking of Blood, Spinal Fluid or Skin Biopsy for the Research of Neurological Diseases
Protocol ID: 16472
NCT: N/A
PI: Dr. Yuen So
Study coordinator: Shirley Paulose, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: To collect blood, spinal fluid, or skin biopsy specimens to create a tissue bank for current or future neuroscience research, which would help to learn more about various neurological conditions
Status: Recruiting

MOVR Registry
Protocol ID: 52620
NCT: N/A
PI: Dr. Jacinda Sampson
Study coordinator: Whitney Tang, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Muscular Dystrophy Association
Purpose: Observational study of neuromuscular conditions including ALS, BMD/DMD, FSHD, LGMD, Pompe and SMA
Status: Recruiting soon in clinic

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: Paxton Ataide, 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

Amyotrophic Lateral Sclerosis (ALS)

Clinical Procedures to Support Research in ALS (CReATe CAPTURE-ALS)
Protocol ID: 54467
NCT03489278
PI: Dr. Yuen So
Study coordinator: Rabia Farooquee, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Natural History Study of ALS and PLS
Status: Recruiting in clinic

A Study to Evaluate the Efficacy and Safety of Reldesemtiv in Patients With Amyotrophic Lateral Sclerosis (ALS) (COURAGE-ALS)
Protocol ID: 60403
NCT04944784
PI: Dr. Yuen So
Study coordinator: Tia Lum, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Cytokinetics
Purpose: Assessing the safety and efficacy of Reldesemtiv in adults with ALS
Status: Recruiting
Recruitment Notes: ALS symptom onset must be less than 2 years ago

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: Veronica Stevens, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Ionis Pharmaceuticals, Inc
Purpose: Assessing the safety and efficacy of ION363 in adults with FUS ALS mutation
Status: Recruiting

A Phase 1 Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB105 Administered Intrathecally to Adults with Amyotrophic Lateral Sclerosis with or without Poly-CAG Expansion in the Ataxin-2 Gene (ALSpire)
Protocol ID: 56187
NCT04494256
PI: Dr. John W. Day
Study coordinator: Habib Mofakham Fini, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen Pharmaceuticals
Purpose: Assess the safety and tolerability of BIIB105 in adults with ALS
Status: Recruiting

Becker and Duchenne Muscular Dystrophy (DMD/BMD)

Wearable Technology to Assess Gait Function in SMA and DMD
Protocol ID: 55106
NCT: N/A
PI: Dr. John W. Day
Study coordinator: Sally Dunaway Young, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Assessing Wearable Technology to Assess Gait Function in SMA and DMD
Status: Recruiting

A Gene Delivery Study to Evaluate the Safety of and Expression from SRP-9001 in Duchenne Muscular Dystrophy (DMD) (ENDEAVOR)
Protocol ID: 59527
NCT04626674
PI: Dr. John W. Day
Study coordinator: Lesly Welsh, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Sarepta Therapeutics, Inc
Purpose: Gene therapy trial testing the safety and efficacy of SRP-9001 in DMD
Status: Active, not recruiting

A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)
PI: Dr. Carolina Tesi-Rocha
Study coordinator: Lesly Welsh, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Sarepta Therapeutics, Inc.
Purpose: Assessing the safety and efficacy of gene transfer therapy with SRP-9001 in older participants with DMD who are ambulatory or non-ambulatory
Status: Recruiting soon

Charcot-Marie Tooth Disease (CMT)

Inherited Neuropathies Consortium
Protocol ID: 23094
NCT01193088
PI: Dr. John W. Day
Study coordinator: Whitney Tang, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: NIH and Muscular Dystrophy Association
Purpose: Examining the natural history of CMT, identifying new mutations, and determining if there are genetic modifiers altering the course of the condition
Status: Recruiting in clinic

Facioscapulohumeral Muscular Dystrophy (FSHD)

Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)
Protocol ID: 57072
NCT04635891
PI: Dr. John W. Day
Study coordinator: Veronica Stevens, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Natural history study of FSHD to validate motor outcomes
Status: Recruiting paused

Phase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD) (FORTITUDE)
Protocol ID: 67690
NCT05747924
PI: Dr. John W. Day
Study coordinator: Ayesha Zaina, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avidity Biosciences
Purpose: Assess the safety and efficacy of AOC 1020 in adults with FSHD
Status: Recruiting soon

GNE Myopathy

Observational study of adults with GNE myopathy
Protocol ID: 23888
NCT: N/A
PI: Dr. John W. Day
Study coordinator: Nicole Corso, 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

Limb Girdle Muscular Dystrophy (LGMD)

An International Clinical Outcome Study of Dysferlinopathy
Protocol ID: 51930
NCT: N/A
PI: Dr. John W. Day
Study coordinator: Habib Mofakham Fini, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Jain Foundation
Purpose: Validation of clinical outcome measures in participants with dysferlinopathy
Status: Active, not recruiting

Myasthenia Gravis (MG)

Study of ALXN2050 in Adult Participants With Generalized Myasthenia Gravis
Protocol ID: 65495
NCT05218096
PI: Dr. Srikanth Muppidi
Study coordinator: Emily Lien, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Alexion
Purpose: Assess the safety and efficacy of Alexion 2050 oral medication in adults with generalized MG
Status: Recruiting

MOM-M281-011/Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Nipocalimab Administered to Adults with Generalized Myasthenia Gravis
Protocol ID: 61457
NCT04951622
PI: Dr. Yuen So
Study coordinator: Yan Yang, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Momenta
Purpose: Assess the safety and efficacy of Nipocalimab in adults with generalized MG
Status: Recruiting
Recruiting notes: Participants with inadequately controlled but stably maintained MG, off IVIG

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
PI: Dr. Carolina Tesi-Rocha
Study coordinator: Yan Yang, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Janssen
Purpose: Assess the safety and efficacy of Nipocalimab in adults with generalized MG
Status: Recruiting
Recruiting notes: Children ages 6-18 with inadequately controlled but stably maintained MG, off IVIG

A Long-Term, Single-Arm, Open-label, Multicenter Phase 3 Study to Evaluate the Safety and Tolerability of Multiple Subcutaneous Injections of Efgartigimod PH20 SC in Patients With Generalized Myasthenia Gravis
Protocol ID: 61629
NCT04818671
PI: Dr. Srikanth Muppidi
Study coordinator: Rabia Farooquee, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: ArgenX
Purpose: Assessing the long term effects of subcutaneous treatment with Efgartigimod PH20 SC in adults with generalized MG
Status: Active, not recruiting

Myotonic Dystrophy

END-DM1 Natural history study of myotonic dystrophy
Protocol ID: 43760
NCT03981575
PI: Dr. John W. Day
Study coordinator: Lesly Welsh, NeuromuscularResearch@stanford.edu, 650-725-4341
Purpose: Characterize baseline status and disease progression over two years in 500 DM1 patients with myotonic dystrophy type 1 (DM1)
Status: Recruiting paused
Study Criteria Notes: Adults with DM1 aged 18-70

Clinical and Genetic Characterization of Myotonic Dystrophy
Protocol ID: 22947
NCT: N/A
PI: Dr. John W. Day
Study coordinator: Whitney Tang,NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: NIH/Investigator Initiated
Purpose: This is an umbrella protocol for Myotonic Dystrophy to study various aspects of the disease
Status: Recruiting

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
Protocol ID: 42616
NCT03692312
PI: Dr. John W. Day
Study coordinator: Rabia Farooquee, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: AMO Pharmaceuticals
Purpose: Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
Status: Active, not recruiting

Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients (MARINA)
Protocol ID: 61989
NCT05027269
PI: Dr. John W. Day
Study coordinator: Tia Lum, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avidity Biosciences, Inc.
Purpose: Assess the safety of AOC 1001 in adults with DM1
Status: Active, not recruiting

Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients (MARINA-OLE)
Protocol ID: 65924
NCT05479981
PI: Dr. John W. Day
Study coordinator: Tia Lum, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avidity Biosciences, Inc.
Purpose: A phase 2 extension of the MARINA study assessing the long term effects of AOC 1001 in adults with DM1
Status: Recruiting by invitation only

Digital wearables study to assess walking and hand myotonia (MYOCAP)
Protocol ID: 62522
NCT: N/A
PI: Dr. John W. Day
Study coordinator: Christina Frater, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Develop and validate new methods of measuring handgrip myotonia and gait in pediatric and adult participants with DM to prepare for new clinical trials
Status: Recruiting

Pompe Disease

The Rare Disease Registry Program
Protocol ID: 12372
Sponsor: Genzyme Corporation/Sanofi
PI: Dr. Gregory Enns, Co-I: Dr. John W. Day
Study coordinator: Lesly Welsh, NeuromuscularResearch@stanford.edu, 650-725-4341
Purpose: To collect information on the subjects with rare diseases like Pompe Disease and other lysosomal storage disorders longitudinally
Status: Recruiting in clinic

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: Veronica Stevens, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Audentes
Purpose: Evaluate the safety and efficacy of AT845 in adults with late onset Pompe disease
Status: Recruiting

Spinal and Bulbar Muscular Atrophy (SBMA)

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients
Protocol ID: 67773
NCT05517603
PI: Dr. John W. Day
Study coordinator: Emily Lien, NeuromuscularResearch@stanford.edu , 650-725-4341
Sponsor: AnnJi Pharmaceutical Co., Ltd.
Purpose: Assessing the safety and efficacy of AJ201 in adults with SBMA
Status: Recruiting

Spinal Muscular Atrophy (SMA)

Clinical Study of Spinal Muscular Atrophy (PNCR/iSMAC study)
Protocol ID: 31140
NCT00443066
PI: Dr. John W. Day
Study coordinator: Whitney Tang, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsors: Spinal Muscular Atrophy Foundation, CureSMA
Purpose: To study the natural history and treatment effects in SMA
Status: Recruiting in clinic

SRK-015-002: Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy (TOPAZ)
Protocol ID: 49533
NCT 03921528
Sponsor: Scholar Rock
PI: Dr. Carolina Tesi Rocha
Coordinator: Tia Lum, NeuromuscularResearch@stanford.edu, 650-725-4341
Purpose: Study the safety and efficacy of the study medication in patients with late onset Spinal Muscular Atrophy
Status: Active, not recruiting

Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam (SAPPHIRE)
Protocol ID: 64058
NCT05156320
PI: Dr. Carolina Tesi-Rocha
Study coordinator: Ayesha Zaina, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Scholar Rock
Purpose: Assess the safety and efficacy of apitegromab in children and adults with later-onset SMA currently receiving nusinersen or risdiplam treatment
Status: Recruiting

Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab-ONYX (ONYX)
Protocol ID: 68228
NCT05626855
PI: Dr. Carolina Tesi-Rocha
Study coordinator: Ayesha Zaina, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Scholar Rock
Purpose: Assess the long term effects of apitegromab in children and adults with late-onset SMA
Status: Recruiting soon by invitation only

Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy (DEVOTE)
Protocol ID: 53421
NCT04089566
PI: Dr. John W. Day
Study coordinator: Lesly Welsh, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen Pharmaceuticals
Purpose: Assessing the efficacy of higher doses of nusinersen in SMA
Status: Recruiting cohort B (<7 months old)

A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec (RESPOND)
Protocol ID: 58724
NCT04488133
PI: Dr. John W. Day
Study coordinator: Susan Thomas, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen Pharmaceuticals
Purpose: Assess the safety and efficacy of Spinraza treatment after Zolgensma treatment
Status: Recruiting

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: Veronica Stevens, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen Pharmaceuticals
Purpose: Test the safety and efficacy of higher doses of nusinersen
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: Emily Lien, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Biogen Pharmaceuticals
Purpose: Assessing the safety and efficacy of a higher dose of nusinersen (Spinraza) in people with SMA who have previously taken risdiplam (Evrysdi)
Status: Recruiting

Nusinersen in Adults with Spinal Muscular Atrophy: An Open Label Study to Assess Efficacy and Exploratory Endpoints (ASE study)
Protocol ID: 55518
NCT: N/A
PI: Dr. John W. Day
Study coordinator: Christina Frater, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Assess the neuromuscular effects of SMA
Status: Recruiting in clinic

CureSMA registry
Protocol ID: 49895
NCT: N/A
PI: Dr. Jacinda Sampson
Study coordinator: Whitney Tang, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: CureSMA
Purpose: Clinical data registry with CureSMA
Status: Recruiting in clinic

Wearable Technology to Assess Gait Function in SMA and DMD
Protocol ID: 55106
NCT: N/A
PI: Dr. John W. Day
Study coordinator: Sally Dunaway Young, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Assessing Wearable Technology to Assess Gait Function in SMA and DMD
Status: Recruiting

Development and Validation for the Adult Test of Neuromuscular Disorders (ATEND), a Functional Motor Outcome Measure
Protocol ID: 31140
NCT: N/A
PI: Dr. John W. Day
Study coordinator: Dr. Tina Duong
Sponsor: Investigator Initiated
Purpose: Development and evaluation of psychometric properties of the ATEND for weaker individuals with neuromuscular disorders
Status: Recruiting in clinic

A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
Protocol ID: 52085
NCT04042025
PI: Dr. John W. Day
Study coordinator: Ayesha Zaina, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Novartis
Purpose: Assessing the long term effects of AVXS-101
Status: Active, not recruiting

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

ARCADIA - AtRial Cardiopathy and Antithrombotic Drugs Intervention After cryptogenic stroke
The purpose of this research study is to compare the effects (good and bad) of apixaban with the effects (good and bad) of aspirin in patients with unexplained strokes and atrial cardiopathy to see which is better at prevention of future strokes. 
NCT03192215
Protocol ID: 45088
PI: Nirali Vora, MD
STATUS: RECRUITING

Crest 2 - Carotid Revascularization And Medical Management For Asymptomatic Carotid Stenosis Trial
The main purpose of the study is to find out if the incidence of stroke or death is different or the same between subjects that receive medical management alone compared to subjects that receive medical management in combination with carotid endarterectomy (CEA) or carotid artery stenting (CAS).  The occurrence of stroke and death may be higher, lower, or the same between groups.
NCT: 02089217
Protocol ID: 41678
PI: Gary Steinberg, MD
STATUS: RECRUITING

TIMELESS: Tenecteplase in Stroke Patients Between 4.5 and 24 Hours
This study is testing a drug called tenecteplase. The purpose of this study is to compare the effects, good or bad, of tenecteplase versus placebo on patients with stroke symptoms who present within 4.5 to 24 hours after the onset of the stroke symptoms.
NCT: NCT03785678
IRB: 49467
PI: Neil Schwartz, MD, PhD
Status: RECRUITING

MASTERS 2 - MultiStem Administration for Stroke Treatment and Enhanced Recovery Study  (Phase III,  blinded, placebo controlled)
The purpose of this study is to compare the effects (good and bad) of an investigational human stem cell (the body’s master cells) product with placebo (no active ingredients) in patients who suffered a recent ischemic stroke.
NCT: NCT03545607
Protocol ID: 50889
PI:  Neil Schwartz , MD, PhD
Study coordinator: Irina Eyngorn
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: RECRUITING

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: Leonel Lugo
Status: RECRUITING

ARCADIA CSI  - AtRial Cardiopathy and Antithrombotic Drugs Intervention After cryptogenic stroke (Cognition and Silent Infarcts)
This is a research study looking to compare the effects (good and bad) of two drugs -apixaban and aspirin- on memory, thinking, reasoning and understanding after stroke.
Protocol ID: 51535
PI: Nirali Vora, MD
Study coordinator: Madelleine Garcia
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: Madelleine Garcia
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: Madelleine Garcia
Status: RECRUITING

AirX BP Control Study Virtual BP Monitoring Pilot Study
The purpose of this study is to learn if individuals who had a stroke can use a home blood pressure monitoring system and a remote medical team to help get their blood pressure under control and reduce their risk of future strokes.
Protocol ID: 61821
PI: Lironn Kraler, MD
Study Coordiantor:  Sarah Drobny
Status: RECRUITING

RECOVERY STUDIES

vREHAB - Virtual Reality Glove for Hand and Arm Rehabilitation After Stroke
The purpose of the study is to demonstrate (a) the feasibility of increasing the dose of rehabilitation in acute stroke patients with a “Smart Glove”, (b) the effect of the “Smart Glove” use on functional recovery, and (c) the effect of the “Smart Glove” use on quality of life.
Protocol ID: 46423
PI: Maarten Lansberg, MD, PhD
STATUS: RECRUITING

StrokeCog
The purpose of this research 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: Maarten Lansberg, MD, PhD
STATUS: RECRUITING

Newly Diagnosed Glioma

A Phase I Study of Tumor Treating Fields with 5-Day Hypofractionated Stereotactic Radiosurgery and Concurrent and Maintenance Temozolomide in Newly Diagnosed Glioblastoma
PI: Scott Soltys, MD
ClinicalTrials.gov#: NCT04474353

A Phase II Study of BPM31510 with Vitamin K1 in Subjects with Newly Diagnosed Glioblastoma (GB)
PI: Seema Nagpal, MD
ClinicalTrials.gov#: NCT04752813

Pivotal, Randomized, Open-label Study of Optune (Tumor Treating Fields) Concomitant with RT & TMZ for the Treatment of Newly Diagnosed GBM (EF-32)
PI: Seema Nagpal, MD
ClinicalTrials.gov#: NCT04471844

ONC201 in H3 K27M-mutant Diffuse Glioma Following Radiotherapy (the ACTION Study)
PI: Kate Therkelsen, MD
ClinicalTrials.gov#: NCT05580562

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

Study to Evaluate Safety, Tolerability, and Optimal Dose of Candidate GBM Vaccine VBI-1901 in Recurrent GBM Subjects
PI: Seema Nagpal, MD
ClinicalTrials.gov#: NCT03382977

A Phase Ib Clinical Trial to Evaluate Early Immunologic Pharmacodynamic Parameters Following Neoadjuvant Anti-PD-1 (Nivolumab), or the Combination of Anti-PD-1 Plus Anti-CTLA-4 (Nivolumab Plus Ipilimumab) in Patients With Surgically Accessible Glioblastoma
PI: Michael Lim, MD
ClinicalTrials.gov#: NCT04606316

Brain Metastases

A Phase IIa Study Assessing QBS72S For Treating Brain Metastases of Breast Cancer
PI: Melanie Hayden Gephart, MD
ClinicalTrials.gov#: NCT05305365

BRAF V600 Mutations

A Phase I, First-In-Human, Multicenter, Open-Label Study of ABM-1310, Administered Orally in Adult Patients with Advanced Solid Tumors
PI: Seema Nagpal, MD
ClinicalTrials.gov#: NCT04190628

Primary CNS Lymphoma

An Open-Label Phase II Study to Investigate the Efficacy, Safety, and Pharmacokinetics of Tirabrutinib in Patients with Primary Central Nervous System Lymphoma (PCNSL)
PI: Neel Gupta, MD
ClinicalTrials.gov#: NCT04947319

LGG

Newly Diagnosed

ACNS1831: A Phase 3 Randomized Study of Selumetinib (IND #77782) Versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)
Diagnosis: Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)

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

PBTC-055: Phase I/II Trial of Dabrafenib, Trametinib, and Hydroxychloroquine (HCQ) for BRAF V600E-Mutant or Trametinib and HCQ for BRAF Fusion/Duplication Positive or NF1-Associated Recurrent or Progressive Gliomas in Children and Young Adults
Diagnosis: LGG/HGG & LGG with NF1

ACNS1931: A Phase 3 Study of Selumetinib (NSC# 748727) or Selumetinib in Combination With Vinblastine for Non-NF1, Non-TSC Patients With Recurrent or Progressive Low-Grade Gliomas (LGGs) Lacking BRAFV600E or IDH1 Mutations
Diagnosis: LGG lacking BRAFV600E or IDH1 Mutations

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

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: Diffuse Intrinsic Pontine Glioma (DIPG) and High-Grade Glioma (HGG)

Recurrent/Refractory/Progressive

PBTC-049: A Phase I Study of Savolitinib in Recurrent, Progressive or Refractory Primary CNS Tumors
Diagnosis: Recurrent, progressive, or refractory primary CNS tumor with evidence of genetic activation of the MET pathway

HGG

Newly Diagnosed

ACNS1723: A Phase 2 Study of Dabrafenib (NSC# 763760) With Trametinib (NSC# 763093) After Local Irradiation in Newly-Diagnosed BRAF V600-Mutant High-Grade Glioma (HGG)

Diagnosis: BRAF V600-Mutant HGG

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: Diffuse Intrinsic Pontine Glioma (DIPG) and High-Grade Glioma (HGG)

Recurrent/Refractory/Progressive

PBTC048: A Feasibility Trial of Optune for Children with Recurrent or Progressive Supratentorial High-Grade Glioma and Ependymoma
Diagnosis: Supratentorial HGG

PBTC-045: A Safety and Preliminary Efficacy trial of MK-3475 (pembrolizumab; anti-PD-1) in children with recurrent, progressive or refractory high-grade gliomas (HGG), DIPGs and hypermutated brain tumors
Diagnosis: Stratum B- histologically confirmed diagnosis of a non-brainstem high-grade glioma (NB-HGG) that is recurrent, progressive or refractory following therapy which included radiotherapy.  Spinal primary disease is eligible

PBTC-049: A Phase I Study of Savolitinib in Recurrent, Progressive or Refractory Primary CNS Tumors
Diagnosis: Recurrent, progressive, or refractory primary CNS tumor with evidence of genetic activation of the MET pathway

PBTC-055: Phase I/II Trial of Dabrafenib, Trametinib, and Hydroxychloroquine (HCQ) for BRAF V600E-Mutant or Trametinib and HCQ for BRAF Fusion/Duplication Positive or NF1-Associated Recurrent or Progressive Gliomas in Children and Young Adults
Diagnosis: Phase 2 stratum 4: HGG with BRAF V600E/D/K mutation

Ependymoma

Newly Diagnosed

There are currently no open trials

Recurrent/Refractory/Progressive

PBTC048: A Feasibility Trial of Optune for Children with Recurrent or Progressive Supratentorial High-Grade Glioma and Ependymoma
Diagnosis: Supratentorial ependymoma

MEDULLOBLASTOMA

Newly Diagnosed

ACNS1422: A Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-Risk WNT-Driven Medulloblastoma Patients
Diagnosis: Medulloblastoma

Recurrent/Refractory/Progressive

PBTC-049: A Phase I Study of Savolitinib in Recurrent, Progressive or Refractory Primary CNS Tumors
Diagnosis: Recurrent, progressive, or refractory primary CNS tumor with evidence of genetic activation of the MET pathway

PBTC-053: A Pediatric Brain Tumor Consortium Phase I/ II and Surgical Study of CX-4945 in Patients with Recurrent SHH Medulloblastoma
Diagnosis: Medulloblastoma

ATRT

Newly Diagnosed

No current Atypical Teratoid Rhabdoid Tumor trials

Recurrent/Refractory/Progressive

PEPN2121: A Phase 1/2 Study of Tiragolumab (NSC# 827799, IND# 161266) and Atezolizumab (NSC# 783608, IND# 161266) in Patients with Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors
Diagnosis: Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors

Neurofibromatosis-1 (NF1)

Newly Diagnosed

ACNS1831- A Phase 3 Randomized Study of Selumetinib (IND # 77782) versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)
Diagnosis: NF1 associated LGG

Recurrent/Refractory/Progressive

PBTC-055: Phase I/II Trial of Dabrafenib, Trametinib, and Hydroxychloroquine (HCQ) for BRAF V600E-Mutant or Trametinib and HCQ for BRAF Fusion/Duplication Positive or NF1-Associated Recurrent or Progressive Gliomas in Children and Young Adults
Diagnosis: NF1-associated recurrent or progressive gliomas

Other- nontreatment

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: Optic pathway glioma

OTHER STUDIES & MOLECULAR BASED THERAPIES

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

LOXO RET: A Phase 1/2 Study of the Oral RET Inhibitor LOXO 292 in Pediatric Patients With Advanced RET-Altered Solid or Primary Central Nervous System Tumors
Diagnosis: Advanced or metastatic solid or primary CNS tumor which has failed standard of care therapies

POE16-01: A Phase I/II Study of Neratinib in Pediatric Patients with Relapsed/ Refractory Solid Tumors or Hematologic Malignancies
Diagnosis: Solid & Central Nervous System (CNS) Tumor, Lymphoma, or Leukemia-

ALTE07C1: Neuropsychological, Social, Emotional and Behavioral Outcomes in Children with Cancer
Diagnosis: Non-disease specific, nontreatment

LOXO-ON-TRK: Prospective Non-interventional study in patients with locally advanced or metastatic TRK fusion cancer treated with larotrectinib
Diagnosis: Non-treatment. locally advanced or metastatic solid tumor harboring an NTRK gene fusion

APEC14B1: The Project: Everychild Protocol: A Registry, Eligibility, Screening, Biology, and Outcome Study
Diagnosis: Registry or eligibility arm

APEC1621: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)
Diagnosis: CNS diagnosis with specific mutation

APEC1621A: Phase 2 study of LOXO-101 (Larotrectinib) in patients with tumors harboring NTRK fusions

APEC1621D: Phase 2 study of LY3023414 in patients with solid tumors- temp closed

APEC1621K: Phase 2 Subprotocol of AG-120 (Ivosidenib) in Patients with Tumors Harboring IDH1 Mutations

APEC1621M: Phase 2 subprotocol of Tipifarnib in patients with tumors harboring HRAS genomic alterations

APEC1621N: Phase 2 Subprotocol of LOXO-292 in Patients with Tumors Harboring RET Gene Alterations

FURTHER INFORMATION AND SUMMARIES FOR HEALTH CARE PROFESSIONALS

Pediatric Brain Tumor Consortium

The Children's Oncology Group

LGG

Newly Diagnosed

ACNS1831: A Phase 3 Randomized Study of Selumetinib (IND #77782) Versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)
Diagnosis: Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)

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

PBTC-055: Phase I/II Trial of Dabrafenib, Trametinib, and Hydroxychloroquine (HCQ) for BRAF V600E-Mutant or Trametinib and HCQ for BRAF Fusion/Duplication Positive or NF1-Associated Recurrent or Progressive Gliomas in Children and Young Adults
Diagnosis: LGG/HGG & LGG with NF1

ACNS1931: A Phase 3 Study of Selumetinib (NSC# 748727) or Selumetinib in Combination With Vinblastine for Non-NF1, Non-TSC Patients With Recurrent or Progressive Low-Grade Gliomas (LGGs) Lacking BRAFV600E or IDH1 Mutations
Diagnosis: LGG lacking BRAFV600E or IDH1 Mutations

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

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: Diffuse Intrinsic Pontine Glioma (DIPG) and High-Grade Glioma (HGG)

Recurrent/Refractory/Progressive

PBTC-049: A Phase I Study of Savolitinib in Recurrent, Progressive or Refractory Primary CNS Tumors
Diagnosis: Recurrent, progressive, or refractory primary CNS tumor with evidence of genetic activation of the MET pathway

HGG

Newly Diagnosed

ACNS1723: A Phase 2 Study of Dabrafenib (NSC# 763760) With Trametinib (NSC# 763093) After Local Irradiation in Newly-Diagnosed BRAF V600-Mutant High-Grade Glioma (HGG)

Diagnosis: BRAF V600-Mutant HGG

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: Diffuse Intrinsic Pontine Glioma (DIPG) and High-Grade Glioma (HGG)

Recurrent/Refractory/Progressive

PBTC048: A Feasibility Trial of Optune for Children with Recurrent or Progressive Supratentorial High-Grade Glioma and Ependymoma
Diagnosis: Supratentorial HGG

PBTC-045: A Safety and Preliminary Efficacy trial of MK-3475 (pembrolizumab; anti-PD-1) in children with recurrent, progressive or refractory high-grade gliomas (HGG), DIPGs and hypermutated brain tumors
Diagnosis: Stratum B- histologically confirmed diagnosis of a non-brainstem high-grade glioma (NB-HGG) that is recurrent, progressive or refractory following therapy which included radiotherapy.  Spinal primary disease is eligible

PBTC-049: A Phase I Study of Savolitinib in Recurrent, Progressive or Refractory Primary CNS Tumors
Diagnosis: Recurrent, progressive, or refractory primary CNS tumor with evidence of genetic activation of the MET pathway

PBTC-055: Phase I/II Trial of Dabrafenib, Trametinib, and Hydroxychloroquine (HCQ) for BRAF V600E-Mutant or Trametinib and HCQ for BRAF Fusion/Duplication Positive or NF1-Associated Recurrent or Progressive Gliomas in Children and Young Adults
Diagnosis: Phase 2 stratum 4: HGG with BRAF V600E/D/K mutation

Ependymoma

Newly Diagnosed

There are currently no open trials

Recurrent/Refractory/Progressive

PBTC048: A Feasibility Trial of Optune for Children with Recurrent or Progressive Supratentorial High-Grade Glioma and Ependymoma
Diagnosis: Supratentorial ependymoma

MEDULLOBLASTOMA

Newly Diagnosed

ACNS1422: A Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-Risk WNT-Driven Medulloblastoma Patients
Diagnosis: Medulloblastoma

Recurrent/Refractory/Progressive

PBTC-049: A Phase I Study of Savolitinib in Recurrent, Progressive or Refractory Primary CNS Tumors
Diagnosis: Recurrent, progressive, or refractory primary CNS tumor with evidence of genetic activation of the MET pathway

PBTC-053: A Pediatric Brain Tumor Consortium Phase I/ II and Surgical Study of CX-4945 in Patients with Recurrent SHH Medulloblastoma
Diagnosis: Medulloblastoma

ATRT

Newly Diagnosed

No current Atypical Teratoid Rhabdoid Tumor trials

Recurrent/Refractory/Progressive

PEPN2121: A Phase 1/2 Study of Tiragolumab (NSC# 827799, IND# 161266) and Atezolizumab (NSC# 783608, IND# 161266) in Patients with Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors
Diagnosis: Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors

Neurofibromatosis-1 (NF1)

Newly Diagnosed

ACNS1831- A Phase 3 Randomized Study of Selumetinib (IND # 77782) versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)
Diagnosis: NF1 associated LGG

Recurrent/Refractory/Progressive

PBTC-055: Phase I/II Trial of Dabrafenib, Trametinib, and Hydroxychloroquine (HCQ) for BRAF V600E-Mutant or Trametinib and HCQ for BRAF Fusion/Duplication Positive or NF1-Associated Recurrent or Progressive Gliomas in Children and Young Adults
Diagnosis: NF1-associated recurrent or progressive gliomas

Other- nontreatment

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: Optic pathway glioma

OTHER STUDIES & MOLECULAR BASED THERAPIES

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

LOXO RET: A Phase 1/2 Study of the Oral RET Inhibitor LOXO 292 in Pediatric Patients With Advanced RET-Altered Solid or Primary Central Nervous System Tumors
Diagnosis: Advanced or metastatic solid or primary CNS tumor which has failed standard of care therapies

POE16-01: A Phase I/II Study of Neratinib in Pediatric Patients with Relapsed/ Refractory Solid Tumors or Hematologic Malignancies
Diagnosis: Solid & Central Nervous System (CNS) Tumor, Lymphoma, or Leukemia-

ALTE07C1: Neuropsychological, Social, Emotional and Behavioral Outcomes in Children with Cancer
Diagnosis: Non-disease specific, nontreatment

LOXO-ON-TRK: Prospective Non-interventional study in patients with locally advanced or metastatic TRK fusion cancer treated with larotrectinib
Diagnosis: Non-treatment. locally advanced or metastatic solid tumor harboring an NTRK gene fusion

APEC14B1: The Project: Everychild Protocol: A Registry, Eligibility, Screening, Biology, and Outcome Study
Diagnosis: Registry or eligibility arm

APEC1621: NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice)
Diagnosis: CNS diagnosis with specific mutation

APEC1621A: Phase 2 study of LOXO-101 (Larotrectinib) in patients with tumors harboring NTRK fusions

APEC1621D: Phase 2 study of LY3023414 in patients with solid tumors- temp closed

APEC1621K: Phase 2 Subprotocol of AG-120 (Ivosidenib) in Patients with Tumors Harboring IDH1 Mutations

APEC1621M: Phase 2 subprotocol of Tipifarnib in patients with tumors harboring HRAS genomic alterations

APEC1621N: Phase 2 Subprotocol of LOXO-292 in Patients with Tumors Harboring RET Gene Alterations

FURTHER INFORMATION AND SUMMARIES FOR HEALTH CARE PROFESSIONALS

Pediatric Brain Tumor Consortium

The Children's Oncology Group

Inherited Neuropathies Consortium
PI: John W. Day, MD, PhD

AMO-02-MD-2-003: 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
PI: John W. Day, MD, PhD

An open-label study to evaluate the Long-term Safety and Efficacy of Tideglusib for the Treatment of Congenital DM1 or Childhood Onset (REACH CDM X)
PI: John W. Day, MD, PhD

PTC124-GD-016-DMD An Open-Label, Safety Study for Ataluren (PTC124) Patients with Nonsense Mutation Dystrophinopathy
PI: John W. Day, MD, PhD

An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects With Duchenne Muscular Dystrophy (ENDEAVOR) - (Gene Transfer)
PI: John W. Day, MD, PhD

Wearable Technology to Assess Gait Function in SMA and DMD
PI: John W. Day, MD, PhD

Motor Outcomes to Validate Evaluations in FSHD (MOVE/+FSHD)
PI: John W. Day, MD, PhD

Determination of standards for Maximal and Submaximal Exercise testing for Individuals with Neuromuscular Disease
PI: John W. Day, MD, PhD

A Two Part Seamless, Open-Label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of R07034067 in Infants With Type 1 Spinal Muscular Atrophy
PI: John W. Day, MD, PhD

Jewelfish: An Open-Label Study To Investigate The Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Risdiplam (RO7034067) in Adult and Pediatric Patients with Spinal Muscular Atrophy
PI: John W. Day, MD, PhD

A Two Part Seamless Multi-Center Randomized Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinets, Pharmacodynamics, and Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients
PI: John W. Day, MD, PhD

AVXS-101-LT-002, A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101 (Gene Transfer)
PI: John W. Day, MD, PhD

A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
PI: John W. Day, MD, PhD

Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy
PI: John W. Day, MD, PhD

ASCEND; A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients with Spinal Muscular Atrophy Previously Treated with Risdiplam
PI: John W. Day, MD, PhD

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)
PI: John W. Day, MD, PhD

A Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients with Alexander Disease (Gene Transfer)
PI: Jacinda Sampson, MD, PhD

SRP-9001-301 A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK)(Gene Transfer)
PI: Ana Tesi-Rocha, MD

SRP-9001-303 (ENVISION) - A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (Gene Transfer)
PI: Ana Tesi-Rocha, MD

80202135MYG2001 / 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
PI: Ana Tesi-Rocha, MD

SRK-015-002: Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy (TOPAZ)
PI: Ana Tesi-Rocha, MD

Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy
PI: Ana Tesi-Rocha, MD

Compassionate Distribution Treatment Protocol: Treatment of Lambert-Eaton Syndrome and Congenital Myasthenic Syndromes with 3,4-Diaminopyridine 
PI: Ana Tesi-Rocha, MD

Phenotypes of Swallowing Physiology & Function Among Patients with Spinal Muscular Atrophy Type 1 
PI: Ana Tesi-Rocha, MD

SRK-015-004:An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegromab in Patients with Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab (ONYX)
PI: Ana Tesi-Rocha, MD

Sunflower Syndrome - Self-Induced Photosensitive Epilepsy
PI: Fiona Baumer, MD    

Epileptic encephalopathy with continuous spike-and-wave during sleep (CSWS): a review of current treatment practices
PI: Fiona Baumer, MD

Cortical Excitability, Synaptic Plasticity and Learning in Benign Epilepsy with Centrotemporal Spikes (BECTS)
PI: Fiona Baumer, MD

Consortium for Standardizing TMS (Transcranial Mangetic Stimulation) Language Mapping Protocols and Establishing Best Practice Guidelines for Using TMS in Presurgical Evaluation of Language in Children
PI: Fiona Baumer, MD

Consequences of Prolonged Febrile Seizures in Childhood
PI: William Gallentine, DO

Novel Early Imaging Markers Following Febrile Status Epilepticus
PI: William Gallentine, DO

Open-Label Safety and Efficacy Study of Cenobamate (YKP3089) in Pediatric Subjects with Partial-onset (Focal) Seizures
PI: William Gallentine, DO

Vascular Effects of Infection in Pediatric Stroke (VIPS II) Study
PI: Sarah Lee, MD

Stanford Children's Health Pediatric Stroke Database
PI: Sarah Lee, MD

International Pediatric Stroke Registry: Risk Factors and Outcome Observational Study
PI: Sarah Lee, MD

Seizures and Children’'s Outcomes after Stroke (SCOUTS)
PI: Sarah Lee, MD

Leducq Trans-Atlantic Network of Excellence On Circadian Effects in Stroke (CIRCA-Stroke)
PI: Sarah Lee, MD

acutE Perfusion Imaging in Childhood LVO (EPIC-LVO): A Prospective Neuroimaging Pilot Study
PI: Sarah Lee, MD

A Natural History Study of Rare Neurogenetic Disorders
PI: Rebecca Levy, MD, PhD

A Retrospective Case Series of Timothy Syndrome
PI: Rebecca Levy, MD, PhD

Pediatric Epilepsy Surgery: Decision-Making Survey in Patients with Tuberous Sclerosis
PI: Brenda Porter, MD, PhD

Preventing Epilepsy Using Vigabatrin in Infants with Tuberous Sclerosis Complex (PREVeNT Trial)
PI: Brenda Porter, MD, PhD

SLC13A5 Deficiency:A Prospective Natural History Study
PI: Brenda Porter, MD, PhD

Stopping Tuberous Sclerosis Complex Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study (TSC-STEPS)
PI: Brenda Porter, MD, PhD

Autism Spectrum Disorder (ASD) and Intellectual Disability (ID) Determinants in Tuberous Sclerosis Complex (TSC)
PI: Brenda Porter, MD, PhD

A Randomized, Double-Blind, Placebo-Controlled Study To Investigate The Efficacy And Safety Of Carisbamate (YKP509) As Adjunctive Treatment For Seizures Associated With Lennox-Gastaut Syndrome In Children And Adults, With Optional Open-Label Extension (SK-LGS)
PI: Brenda Porter, MD, PhD

A Multimodal Biomarker-based Predictive Model for Infantile Spasms
PI: Ann Hyslop, MD

A Multi-Center Examination of Pediatric Epilepsy Surgery Evaluation in the US
PI: Ann Hyslop, MD

A phase 1-3, Double Bind, Randomized Placebo- Controlled Study to Evaluate the Efficacy, Safety, PharmacoKinetics and Pharmacodynamics Intrathecally Administered ION373 in Patients with Alexander Disease (Gene Transfer)
PI: Keith Van Haren, MD

Project BIG: The Stanford Brain Immune Gut Research Initiative
PI: Keith Van Haren, MD

A phase 3 Study of Lenti-D Drug Product After Myeloblative Conditioning Using Busulfan and Fludarabine in Subjetcs 17 Years of Age and Under With Cerebral Adrenoleukodystrophy (CALD) (BMT/GeneTransfer)
PI: Keith Van Haren, MD

The Effect of Vitamin D3 on Markers of Oxidative Stress in Boys with X-Linked Adrenoleukodystrophy (ALD)
PI: Keith Van Haren, MD

Biobank for Neuroinflammatory Disorders
PI: Keith Van Haren, MD

New Diagnostic Approaches in Leukodystrophy - The Myelin Disorders Biorepository and Natural History Project
PI: Keith Van Haren, MD

Neonatal Seizure Registry
PI: Courtney Wusthoff, MD

High Frequency Oscillations in Neonates
PI: Courtney Wusthoff, MD

Temporal-spatial quantification of neonatal seizure burden caused by arterial ischemic stroke
PI: Courtney Wusthoff, MD

Practices in the Evaluation of Pediatric Brain Death
PI: Courtney Wusthoff, MD

Critical Care EEG Monitoring Research Consortium
PI: Courtney Wusthoff, MD

Neonatal Seizure Registry- Developmental functional Evaluation (NSR-DEV)
PI: Courtney Wusthoff, MD

Creation and Development of Pediatric Neurocritical Care Database
PI: Courtney Wusthoff, MD

Continued Anticonvulsants After Resolution of Neonatal Seizures: A Patient-centered Comparative Effectiveness Study
PI: Courtney Wusthoff, MD

Study of Oral MC-1 for the Treatment of Patients with PNPO Deficiency
PI: Courtney Wusthoff, MD

MRI and EEG findings in preterm neonates with HIE
PI: Courtney Wusthoff, MD

Children with Lennox-Gastaut Syndrome (LGS) treated with Deep Brain Stimulation (DBS)
PI: Juliet Knowles, MD, PhD

MRI Microstructure Mapping to Improve Detection of Focal Cortical Dysplasia
PI: Juliet Knowles, MD, PhD

Outcomes Following Discharge in the Pediatric Neurocritical Care Population; Understanding the need
PI: Lindsey Rasmussen, MD

AAP Resident Research Grant: Near-infrared spectroscopy (NIRS) and blood pressure monitoring in pediatric acute ischemic stroke: an observational, prospective pilot study
PI: Lindsey Rasmussen, MD

Pediatric Influence of Cooling duration on Efficacy in Cardiac Arrest Patients (P-ICECAP)
PI: Lindsey Rasmussen, MD

Optic Disc Drusen in children
PI: Shannon Beres, MD

Continuation of the analysis of Cytokine Modulation with Tecfidera in Human Immune Cell Subsets using High Dimensional Flow Cytometry and CYTOF Mass Spectrometry
PI: Lawrence Steinman, MD

Single Cell RNA-seq and CITE-seq on B cells in the peripheral blood and CSF of MS patients prior and during therapy with ATA-188
PI: Lawrence Steinman, MD

MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
PI: Cynthia Campen, MD

A Phase 2b Trial of MEK ½ Inhibitor (MEKi) PD-0325901 in Adult and Pediatric Patients with Neurofibromatosis Type 1 (NF1)-Associated Inoperable Plexiform Neurofibromas (PNs) that are Causing Significant Morbidity
PI: Cynthia Campen, MD              

Developing Evidence-Based Criteria for Initiating Treatment for NF1-Associated Optic Pathway Glioma:  A Natural History Observational Study
PI: Cynthia Campen, MD

Neuroimaging Correlates of Neurocognitive Deficits in Children with NF1
PI: Cynthia Campen, MD