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: Nada Hindiyeh, MD
NCT04688775
Study Status: Enrolling
Research Coordinator: Stephanie Tran
Contact: trans@stanford.edu

Sunstar: The Stanford Neuroscience Headache Biorepository
PI: Robert Cowan, MD, FAAN
NCT03231241
Study Status: Enrolling
Research Coordinator: Bharati Sanjanwala
Contact: bharatis@stanford.edu

Sphenopalatine Ganglion Stimulation for the Treatment of Chronic Cluster Headache
PI: Meredith Barad, MD
NCT02168764
Study Status: Closed to Enrollment
Research Coordinator: Stephanie Tran
Contact: trans@stanford.edu

Diagnostic comparison of an online questionnaire versus  a semi-structured interview for the diagnosis of primary headache disorders, using ICHD 3 as the Gold Standard
PI: Robert Cowan, MD, FAAN
NCT03304886
Study Status: Closed to Enrollment
Research Coordinator: Bharati Sanjanwala
Contact: bharatis@stanford.edu

A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel- Group Study to Evaluate the Efficacy, Safety and Tolerability of Oral Atogepant for the Prevention of Migraine in Participants with Episodic Migraine (ADVANCE)
PI: Nada Hindiyeh, MD
NCT03777059
Study Status: Closed to Enrollment
Research Coordinator: Stephanie Tran
Contact: trans@stanford.edu

A Multicenter, Randomized, Double-Blind, Double-Dummy, Placebo-Controlled, Parallel-Group Study Comparing the Efficacy and Safety of 2 Dose Regimens (Intravenous/Subcutaneous and Subcutaneous) of TEV-48125 Versus Placebo for the Prevention of Epidosic Cluster Headache
PI: Nada Hindiyeh, MD
NCT02945046
Study Status: Closed to Enrollment
Research Coordinator: Stephanie Tran
Contact: trans@stanford.edu

A Multicenter, Randomized, Double-Blind, Double-Dummy, Placebo-Controlled, Parallel-Group Study Comparing the Efficacy and Safety of 2 Dose Regimens (Intravenous/Subcutaneous and Subcutaneous) of TEV-48125 Versus Placebo for the Prevention of Chronic Cluster Headache
PI: Nada Hindiyeh, MD
NCT02964338
Study Status: Closed to Enrollment
Research Coordinator: Stephanie Tran
Contact: trans@stanford.edu

A Multicenter, Double-Blind, Double-Dummy Study to Explore the Long-Term Safety of TEV-48125 for the Prevention of Cluster Headache
PI: Nada Hindiyeh, MD
NCT03107052
Study Status: Closed to Enrollment
Research Coordinator: Stephanie Tran
Contact: trans@stanford.edu

A Randomized, Multicenter, Double-Blind, Parallel, Sham-Controlled Study of Non-Invasive Vagus Nerve Stimulation (NVNS) for the Prevention of Migraines. (PREMIUM II)
PI: Nada Hindiyeh, MD
NCT03716505
Study Status: Closed to enrollment
Research Coordinator: Stephanie Tran
Contact: trans@stanford.edu

A Phase 3b Multicenter, SIngle-arm, Open-label Safety Study of Ly2951742 (Galcanezumab) in Patients with Episodic or Chronic Cluster Headache
PI: Nada Hindiyeh, MD
NCT02797951
Study Status: Closed to Enrollment
Research Coordinator: Stephanie Tran
Contact: trans@stanford.edu

A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of LY2951742 in Patients with Episodic Cluster Headache
PI: Nada Hindiyeh, MD
NCT02397473
Study Status: Closed to Enrollment
Research Coordinator: Stephanie Tran
Contact: trans@stanford.edu

A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of LY2951742 with a Long-Term Open-Label Extension in Patients with Chronic Cluster Headache
PI: Nada Hindiyeh, MD
NCT02438826
Study Status: Closed to Enrollment
Research Coordinator: Stephanie Tran
Contact: trans@stanford.edu

A 12-Month Prospective, Randomized, Interventional, Global, Multi-Center, Active-Controlled Study Comparing Sustained Benefit of Two Treatment Paradigms (Erenumab GM vs. Oral Prophylactics) in Adult Episodic Migraine Patients (CAMG334A2401)
PI: Nada Hindiyeh, MD
NCT03927144
Study Status: Closed to enrollment
Research Coordinator: Stephanie Tran
Contact: trans@stanford.edu

A Randomized, Double-Blind, Parallel Group, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy, Safety, and Tolerability of Single Doses of STS101 (Dihydroergotamine Nasal Powder) in the Acute Treatment of Migraine
PI: Nada Hindiyeh, MD
NCT03901482
Study Status: Closed to enrollment
Research Coordinator: Stephanie Tran
Contact: trans@stanford.edu

A Parallel Group Double-Blind Randomized PlaceboControlled Study to Evaluate the Efficacy and Safety of Eptinezumab Administered Intravenously in Subjects Experiencing an Acute Attack of Migraine
PI: Nada Hindiyeh, MD
NCT04152083
Study Status: Closed to Enrollment
Research Coordinator: Stephanie Tran
Contact: trans@stanford.edu

Randomized, Double-Blind, Multi-Center, Parallel-Group Comparison of the Efficacy and Safety of the C213 (Zolmitriptan Microneedle System) to Placebo for the Acute Treatment of Cluster Headaches
PI: Nada Hindiyeh, MD
NCT04066023
Study Status: Closed to enrollment
Research Coordinator: Stephanie Tran
Contact: trans@stanford.edu

A PHASE 3, MULTICENTER, OPEN-LABEL 40-WEEK EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY AND TOLERABILITY OF ORAL ATOGEPANT FOR THE PREVENTION OF MIGRAINE IN PARTICIPANTS WITH EPISODIC MIGRAINE
PI: Nada Hindiyeh, MD
NCT03777059
Study Status: Closed to Enrollment
Research Coordinator: Stephanie Tran
Contact: trans@stanford.edu

A Long-Term, Open-Label Safety Study to Evaluate the Safety of M207 (Intracutaneous Microneedle System) in the Acute Treatment of Migraine
PI: Nada Hindiyeh, MD
NCT03282227
Study Status: Closed to Enrollment
Research Coordinator: Stephanie Tran
Contact: trans@stanford.edu

Active, recruiting:

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)
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

Sponsor: Janssen Research & Development (Autonomy Study)
Intervention: JNJ-63733657 (monoclonal anti-tau antibody)
Indication: mild cognitive impairment and mild AD
Age: 55-80 years old
Brief Summary: A Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicenter Study to Assess the Efficacy and Safety of JNJ-63733657, an Anti-tau Monoclonal Antibody, in Participants with Early Alzheimer’s Disease
Clinicaltrials.gov identifier: NCT04619420
PI: Sharon Sha, MD
Contact: Santi Decunto; decunto@stanford.edu; 650- 421-1284

Who can participate

• Adults and older adults between the ages of 55 to 80.
• Early AD: Gradual and progressive subjective decline in the participant’s cognition over at least the past 6 months, as reported by the participant and informant (study partner) and CDR-GS of 0.5 and memory box score ≥0.5 at screening.
• Evidence of pathologically elevated tau (T+) as defined first in plasma as directed in a separate Laboratory Charter/Manual. Only plasma T+ participants will undergo a tau PET scan at screening to confirm T+ status.
• Evidence of pathologic tau on a screening tau PET scan reviewed centrally by a qualified reader, as prespecified in a separate Imaging Charter.
• Able to read and write and with a minimum 5 years of formal education as reported by participant and study partner at screening.
• Willing to participate in this study (signed written informed consent) and to comply with the study protocol.
• Have a designated study partner who has adequate literacy to participate and be judged to have high likelihood of completing the study with the participant.

Sponsor: Eisai and NIH (AHEAD 3-45 Study)
Intervention: BAN2401 (monoclonal antibody binding to amyloid)
Indication: Pre-clinical AD 
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: https://studypages.com/s/the-ahead-study-440395/

Upcoming Study:

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:Jade Perry; jadep@stanford.edu; 650-521-7287

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.

Active, not recruiting:

Sponsor: Eli Lilly (Trailblazer Study)
Intervention: Donanemab (monoclonal antibody binding to amyloid)
Protocol NCT#: NCT04437511
PI: Sharon Sha, MD

Sponsor: Eisai (Clarity AD)
Intervention: BAN2401 (monoclonal antibody binding to amyloid)
Indication: MCI/Mild AD
Clinicaltrials.gov identifier:  NCT03887455
PI: Sharon Sha, MD

Sponsor: Biogen (Embark Study)
Intervention: Aducanumab (Monoclonal antibody binding to amyloid)
Indication: MCI/Mild AD (Recruiting by invitation only: participants who were previously enrolled in aducanumab trials) 
Clinicaltrials.gov identifier: NCT04241068
PI: Sharon Sha, MD

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

Vibrotactile Coordinated Reset: A Non-invasive Treatment for Parkinson's Disease
Purpose: The purpose of our study is to evaluate Vibrotactile Coordinated Reset stimulation (vCR) and its effects on motor ability within Parkinson's patients. vCR will be administered with a device called the VT Brain Glove. VCR is expected to provide patients with a non-invasive alternative to the most widely used treatments such as levodopa and or deep brain stimulation. This study will include a dedicated sham that will aid in understanding true treatment effects from vCR.
Sponsor: Stanford University, Synergic Medical Technologies, Inc.
PI: Peter Tass, MD, PhD
Co-PI: Leila Montaser Kouhsari, MD, PhD
Study status: Open, enrollment ongoing
Research Coordinator: Jessica Kalinova Yankulova
Contact: parkinsonsvcr@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: Maria-Lucia Campos
udallcenter@stanford.edu  (650) 721-5351

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
Status: Active, Enrolling
PI: Jeffrey Dunn, MD
Research Coordinator: Anna Tomczak
Contact: atomc96@stanford.edu

A Multicenter Randomized Controlled Trial of Best Available Therapy versus Autologous Hematopoietic Stem Cell Transplant for Treatment-Resistant Relapsing Multiple Sclerosis (BEAT-MS-ITN077AI)
PI: Jeffrey Dunn, MD
NCT04047628
Status: Active, Enrolling
Research Coordinator: Sujatha Kalle
Contact: skalle@stanford.edu

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
PI: May Han, MD
Study Status: Active, Enrolling
Research Coordinator: Sujatha Kalle
Contact: skalle@stanford.edu

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)
PI: Lucas Kipp, MD
NCT04548999
Study Status: Active, Enrolling
Research Coordinator: Julia Sumera
Contact: jsumera@stanford.edu

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)
PI: Lucas Kipp, MD
NCT04586023
Study Status: Active, Enrolling
Research Coordinator: Julia Sumera
Contact: jsumera@stanford.edu

North American Registry for Care and Research in Multiple Sclerosis (NARCRMS) Study
PI: Jeffrey Dunn, MD
Study Status: Active, Enrolling
Research Coordinator: Anna Tomczak
Contact: atomc96@stanford.edu

CorEvitas SPHERES (Synergy of Prospective Health & Experimental Research for Emerging Solutions) Registry for Neuromyelitis Optica Spectrum Disorder (NMOSD)
PI: May Han, MD
Study Status: Active, Enrolling
Research Coordinator: Anna Tomczak
Contact: atomc96@stanford.edu

A randomized, double-blind, placebo-controlled, multicenter, Phase 3, pivotal study with an open-label extension period to evaluate the efficacy and safety of rozanolixizumab in adult participants with myelin oligodendrocyte glycoprotein (MOG) antibody-associated disease (MOG-AD)
PI: May Han, MD
NCT05063162
Study Status: Active, Enrolling
Research Coordinator: Tara Tripathi Sarkar
Contact: tarats@stanford.edu

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
Study Status: Active, Enrolling
Research Coordinator: Anna Tomczak
Contact: atomc96@stanford.edu

A Phase 1, Double-blind, Placebo-controlled Dose-expansion Study with an Open-label Extension to Evaluate the Safety and Efficacy of ATA188 in Subjects with Progressive Multiple Sclerosis (ATA188)
PI: Lawrence Steinman, MD
Study Status: Closed to Enrollment
Research Coordinator: Anna Tomczak
Contact: atomc96@stanford.edu

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 Relapsing Multiple Sclerosis (BN42082)
PI: Lucas Kipp, MD
NCT04544436
Study Status: Closed to Enrollment
Research Coordinator: Julia Sumera
Contact: jsumera@stanford.edu

A Randomized, Double-blind, Double-dummy, Parallel-group Study to Evaluate the Efficacy and Safety of Ocrelizumab in Comparison to Interferon Beta-1a (Rebif ®) in Patients with Relapsing Multiple Sclerosis
PI:  Jeffrey Dunn, MD
NCT01412333
Study Status: Closed to Enrollment
Research Coordinator: Yamuna Joseph
yamuna@stanford.edu

An open-label, multicenter, biomarker study to explore the mechanism of action of ocrelizumab and B-cell biology in patients with relapsing multiple sclerosis
PI: Christopher Lock, MD
NCT02688985
Study Status: Closed to Enrollment
Research Coordinator: Yamuna Joseph
yamuna@stanford.edu

Development and Validation for the Adult Test of Neuromuscular Disorders (ATEND), a Functional Motor Outcome Measure

Protocol ID: 58208
NCT: NA
PI: Dr. John W. Day
Study coordinator: Whitney Tang, whitneyt@stanford.edu, 650-475-6580
Purpose: This is a multi-center, prospective, observational study aimed to develop and validate a wheelchair based functional motor outcome measure for chronic, weak, non-ambulatory individuals diagnosed with a neuromuscular disease.
Status: Active

An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443

Protocol ID: 36172
NCT02594124
Sponsor: Ionis Pharmaceuticals, Inc.
PI: Dr. John W. Day
Study coordinator: Shirley Paulose, spaulose@stanford.edu, 650-725-4341
Purpose: To evaluate the long-term safety and tolerability, and to examine the cerebrospinal fluid pharmacokinetics of ISIS 396443 administered intrathecally to patients with SMA who previously participated in investigational studies of ISIS 396443.
Status: Active, but closed to enrollment.

Defining and Managing the Neuropsychological Abnormalities of Myotonic Dystrophy (CHRI protocol on DM)

Protocol ID: 28486
NCT02269865
PI: Dr. John W. Day
Study coordinator: Mitchell Reddan, mreddan@stanford.edu, 650-725-4341
Sponsor: Childrens’ Health Research Institute/investigator initiated
Purpose: To clarify mechanisms that underlie cognitive and behavioral changes in DM, some of which may also be involved in more common childhood behavioral abnormalities
Status: Recruitment paused

Clinical and Genetic Characterization of Myotonic Dystrophy

Protocol ID: 22947
NCT: NA
PI: Dr. John W. Day
Study coordinator: Mitchell Reddan, mreddan@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 including sleep abnormalities.
Status: Recruiting

Inherited Neuropathies Consortium

Protocol ID: 23094
NCT01193088
PI: Dr. John W. Day
Study coordinator: Shirley Paulose, spaulose@stanford.edu, , 650-725-4341
Sponsor: NIH and Muscular Dystrophy Association
Purpose: to learn more information about the way that Charcot Marie Tooth disease (CMT) progresses over time, and also looking for new genetic types of CMT and looking  to see if there are things in the DNA that may change the presentation of the condition by making it better or worse
Status: Recruiting

Subject Database and Specimen Repository for Neuromuscular and Neurodegenerative Disorders

Protocol ID: 23888
NCT: NA
PI: Dr. John W. Day
Study coordinator:  Mitchell Reddan, mreddan@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 subject 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

Compassionate Distribution Treatment Protocol: Treatment of Lambert-Eaton Syndrome with 3,4-Diaminopyridine

Protocol ID: 33296
NCT: NA
PI: Dr. John W. Day
Study coordinator: Shirley Paulose, spaulose@stanford.edu, 650-725-4341
Sponsor: Jacobus Pharmaceuticals
Purpose: To provide access to 3,4-diaminopyridine (3,4-DAP), a drug which has been demonstrated to be effective in treating the weakness associated with Lambert-Eaton Myasthenic Syndrome (LEMS) but is currently not approved by the FDA for use in the United States. This study is for one patient only and to provide the medication on a compassionate use basis.
Status: Active, NOT Recruiting

An Open-Label, Expanded Access Protocol for Firdapse® (Amifampridine Phosphate; 3,4-Diaminopyridine Phosphate) Treatment in Patients with Lambert-Eaton Myasthenic Syndrome (LEMS), Congenital Myasthenic Syndromes (CMS) and Downbeat Nystagmus

Protocol ID: 36811
NCT02189720
PI: Dr. Yuen So
Sponsor: Catalyst Pharmaceuticals
Study coordinator: Lesly Welsh, lwelsh@stanford.edu, 650-725-4341
Purpose:To provide patients with LEMS/CMS/downbeat nystagmus access to amifampridine phosphate therapy until the product becomes commercially available.
Status: Active, enrollment open by invitation only

Tissue Banking of Blood, Spinal Fluid or Skin Biopsy for the Research of Neurological Diseases

Protocol ID: 16472
NCT: NA
PI: Dr. Yuen So
Study coordinator: Shirley Paulose, spaulose@stanford.edu, 650-725-4341
Sponsor: investigator initiated
Purpose: To collect blood, spinal fluid, or skin biopsy specimen to create a tissue bank for current or future neuroscience research, which would help to learn more about various neurological conditions.
Status: Recruiting

Clinical Study of Spinal Muscular Atrophy (PNCR/iSMAC SMA study)

Protocol ID: 31140
NCT00443066
PI: Dr. John W. Day
Study coordinators: Monica Sangco, msangco@stanford.edu and Katharine Hagerman, khagerma@stanford.edu, 650-725-4341
Sponsor: Columbia University, Collaborator: Spinal Muscular Atrophy Foundation
Purpose: To study the natural history of Spinal Muscular Atrophy to help with clinical trials in future.
Status: Recruiting in clinic

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, lwelsh@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

WN40226 A Multi-Site, Randomized, Placebo-Controlled, Double-Blind, Multiple Ascending Subcutaneous Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of RO07239361 (BMS-986089) in Ambulatory Boys with Duchenne Muscular Dystrophy.

Protocol ID: 35232
NCT02515669
Sponsor: Bristol-Myers Squibb
PI: Dr. John W. Day
Study coordinator:  Tia Nguyen, tinguyen@stanford.edu, 650-725-4341
Purpose: To study the safety, tolerability and pharmacokinetics of the study medication, BMS-986089, in Duchenne muscular dystrophy
Status: Active, Closed to enrollment.

Collection of Confirmed DMD Positive and Presumptive Negative Newborn Screening DBS Specimens

Protocol ID: 36312
NCT: NA
Sponsor: Parent Project Muscular Dystrophy
PI: Dr. John W. Day
Study coordinated by Senior Genetic Counselor, Carly Siskind, csiskind@stanfordhealthcare.org, 650-725-4341
Purpose: The purpose of this study is to help develop and validate a kit for future newborn screening for Duchenne muscular dystrophy.
Status: Recruiting

CReATe/ Phenotype, Genotype and Biomarkers in Amyotrophic Lateral Sclerosis and Related Disorders

Protocol: 40338
NCT02327845
Sponsor: NINDS/NIH (sub award through University of Miami)
PI: Dr. Yuen So
Coordinator: Mitchell Reddan, mreddan@stanford.edu, 650-725-4341
Purpose: to learn more about Amyotrophic Lateral Sclerosis and some related disorders
Status: Recruiting

A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 n SRP-4053 in Patients With Duchenne Muscular Dystrophy (ESSENCE)

Protocol: 38263
NCT02500381
Sponsor: Sarepta Therapeutics, Inc.
PI: Dr. John W. Day
Coordinator: Monica Sangco, msangco@stanford.edu 650-725-4341
Purpose: To evaluate the efficacy of SRP-4045 and SRP-4053 compared to placebo in Duchenne muscular dystrophy (DMD) patients with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53 respectively.
Status: Active, Closed to recruitment

Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting but Non-ambulatory Patients with Spinal Muscular Atrophy

Protocol: 38893
NCT03381729
Sponsor: Avexis Pharmaceuticals, Inc.
PI: Dr. John W. Day
Coordinator: Carolyn McLaughlin Savage, cmcsavage@stanford.edu  650-725-4341
Purpose: It is a gene therapy study to compare doses of the investigational product for a group of subjects with Spinal Muscular Atrophy
Status: Open for recruitment.

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 (Firefish)

Protocol: 40452
NCT02913482
Sponsor: Roche Pharmaceuticals, Inc.
PI: Dr. John W. Day
Coordinator: Lesly Welsh, lwelsh@stanford.edu, 650-725-4341
Purpose: it is to assess an investigational product for spinal muscular atrophy Type 1
Status: Active, but closed to enrollment

AVXS-101-CL-303: Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN 2 CopiesPhase I, Open-Label, Dose Comparison Study of AVXS-101 by Intravenous Infusion (STRIVE) GENE TRANSFER.

Protocol: 40902
NCT03306277
Sponsor: Avexis Pharmaceuticals, Inc.
PI: Dr. John W. Day
Coordinator: Carolyn McLaughlin Savage, cmcsavage@stanford.edu  650-725-4341
Purpose: It is a gene therapy study in subjects with Spinal Muscular Atrophy Type 1.
Status: Will be open for recruitment soon.

WN40227 A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 (BMS-986089) in Ambulatory Boys with Duchenne Muscular Dystrophy

Protocol: 40357
NCT03039686
Sponsor: Roche Pharmaceuticals
PI: Dr. John W. Day
Coordinator: Tia Nguyen, tinguyen@stanford.edu, 650-725-4341
Purpose: An anti-myostatin compound is tried in subjects with any type of Duchenne Muscular Dystrophy.
Status: Will be recruiting soon.

CY 5022/ a Phase 2, Multi-Center, Double-Blind, Randomized, Dose-Ranging, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of CK-2127107 in Patients with Amyotrophic Lateral Sclerosis

Protocol: 41454
NCT03160898
Sponsor: Cytokinetics, Inc.
PI: Dr. Yuen So
Coordinator: Tia Nguyen, tnguyen@stanford.edu, 650-725-4341
Purpose: This study is mainly to assess the improvement in respiratory function in patients with Amyotrophic Lateral Sclerosis
Status: Will be open soon for recruitment

Compassionate Distribution Treatment Protocol: Treatment of Lambert-Eaton Syndrome and Congenital Myasthenic Syndromes with 3,4 Diaminopyridine

Protocol: 39538
NCT: NA
Sponsor: Jacobus Pharmaceuticals, Inc.
PI: Dr. Carolina Tesi Rocha
Coordinator: Lesly Welsh, 650-725-4341, lwelsh@stanford.edu
Purpose: This protocol is specifically for a child with congenital myasthenia syndrome  to treat the muscle weakness.
Status: Active, but not recruiting.

A Phase 3 Randomized, Multicenter, Multinational, Double-Blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of NeoGAA (GZ402666) and Alglucosidase Alfa in Treatment-Naïve Patients with Late Onset Pompe Disease

Protocol ID: 38164
NCT02782741
Sponsor: Sanofi Genzyme
PI: Dr. John W. Day
Coordinator: Monica Sangco, msangco@stanford.edu, 650-725-4341
Purpose: To study the possible risks, efficacy, and pharmacokinetics of the study drug when compared to Lumizyme which is available now commercially to treat Pompe Disease.
Status: Open to recruitment.

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension

Protocol ID: 42313
NCT03179631
Sponsor: PTC Therapeutics
PI: Dr. John W. Day
Coordinator: Monica Sangco, msangco@stanford.edu, 650-725-4341
Purpose: to characterize the long-term effects of ataluren-mediated dystrophin restoration on disease progression.
Status: Open for recruitment.

Collection of Confirmed SMA, XLA, and SCID Positive Newborn Screening Dried Blood Spot Specimens

Protocol: 45467
NCT:NA
Sponsor: Perkin Elmer
PI: Dr. John W. Day
Coordinator: Managed in clinic by senior genetic counsellor Carly Siskind, csiskind@stanfordhealthcare.org, 650-725-4341
Purpose: to develop a newborn SMA screening kit.
Status: Open by invitation only

Individual Patient Expanded Access Protocol for the Treatment of a Single Patient with Late-Onset Tay Sachs.

Protocol: 46346
NCT: NA
PI: Dr. Yuen So
Coordinator: Tia Nguyen, tinguyen@stanford.edu, 650-725-4341
Purpose: To evaluate the safety, tolerability, and feasibility of administration of RT001 in a single subject with Late Onset Tay Sachs.
Status: Closed for enrollment, but active.

A Two Part Seamless Multi-center Randomized Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients (SUNFISH Trial)

Protocol: 44102
NCT02908685
PI: Dr. John W. Day
Coordinator: Lesly Welsh, lwelsh@stanford.edu, 650-725-4341
Purpose: to assess the safety and tolerability, efficacy, pharmacokinetics and pharmacodynamics of RO7034067 in pediatric and adults patients with SMA Type 2 and 3.
Status: Active, closed to recruitment

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

CHARM - Study to Evaluate the Efficacy and Safety of Intravenous BIIB093 for Severe Cerebral Edema following Large Hemispheric Infarction.
The purpose of this study is to evaluate the safety and effectiveness of the study drug, Intravenous BIIB093 (Glibenclamide), in improving functional outcome in subjects with large strokes. NCT02864953
Protocol ID: 46054
PI: Chitra Venkatasubramanian, MD
Status: RECRUITING

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 Follow-Up Study to Add Whole Brain Radiotherapy (WBRT) to Standard Temozolomide Chemo-Radiotherapy in Newly Diagnosed Glioblastoma (GBM) Treated with 4 Weeks of Continuous Infusion Plerixafor
PI: Lawrence Recht, MD
ClinicalTrials.gov#: NCT03746080

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

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

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

NTRK-Fusion Positive

A Phase I/II Study of PBI-200 in Subjects with NTRK-Fusion-Positive Advanced or Metastatic Solid Tumors
PI: Seema Nagpal, MD
ClinicalTrials.gov#: NCT04901806

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

CNS Diagnostic Imaging

A Phase I Study of [18F]DASA-23 as a PET Tracer for Evaluating Pyruvate Kinase M2 (PKM2) Expression in Healthy Volunteers and in Patients with Intracranial Tumors
PI: Guido Davidzon, MD SM
ClinicalTrials.gov#: NCT03539731

Brain Metastases

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: 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)

Relapse/Refractory

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

PBTC-051: Phase I Study to Evaluate the Safety and Tolerability of the CD40 Agonistic Monoclonal Antibody APX005M in Pediatric Subjects with Recurrent/Refractory Brain Tumors and Newly Diagnosed Brain Stem Glioma
Diagnosis: Stratum 2 newly diagnosed Diffuse Intrinsic Pontine Glioma (DIPG)

Relapse/Refractory

PBTC-049: A Phase I Study of Savolitinib in Recurrent, Progressive or Refractory Primary CNS Tumors
Diagnosis: Medulloblastoma, DIPG, and HGG

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

Relapse/Refractory

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: Medulloblastoma, DIPG, and HGG

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

PBTC056: A Phase I Study of the Adam-10 Inhibitor, INCB7839 in Children With Recurrent/Progressive High-Grade Gliomas to Target Microenvironmental Neuroligin-3
Diagnosis: HGG

Ependymoma

Newly Diagnosed

There are currently no open trials

Relapse/Refractory

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 D-Patients must have a histologically confirmed diagnosis of ependymoma that is recurrent, progressive or refractory following therapy which included radiotherapy.

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

Relapse/ Refractory

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 E- Patients must have a histologically confirmed diagnosis of medulloblastoma that is recurrent, progressive or refractory following therapy which included radiotherapy.

PBTC-049: A Phase I Study of Savolitinib in Recurrent, Progressive or Refractory Primary CNS Tumors
Diagnosis: Medulloblastoma, DIPG, and HGG

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

SJATRT: Phase 2 Study of Alisertib as a single agent in Recurrent or Progressive Central Nervous System (CNS) Atypical Teratoid Rhabdoid Tumors (ATRT) and Extra-CNS Malignant Rhabdoid Tumors (MRT) and in Combination Therapy in Newly Diagnoses ATRT
Diagnosis: Atypical Teratoid Rhabdoid Tumors (ATRT)

Relapse/Refractory

PBTC-051: Phase I Study to Evaluate the Safety and Tolerability of the CD40 Agonistic Monoclonal Antibody APX005M in Pediatric Subjects with Recurrent/Refractory Brain Tumors and Newly Diagnosed Brain Stem Glioma
Diagnosis: Stratum I Recurrent or Refractory Primary Malignant CNS tumor patients

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

Relapse/Refractory

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

Other

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 & 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

Cellectar: An Open-Label, Dose Escalation, Efficacy, and Safety Study of CLR 131 in Children, Adolescents, and Young Adults With Select Solid Tumors, Lymphoma, and Malignant Brain Tumors
Diagnosis: Previously confirmed (histologically or cytologically) pediatric solid tumor (e.g., neuroblastoma, sarcoma), lymphoma (including Hodgkin's lymphoma), or malignant brain tumors that are clinically or radiographically suspected to be relapsed, refractory, or recurrent for which there are no standard treatment options with curative potential

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: 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)

Relapse/Refractory

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

PBTC-051: Phase I Study to Evaluate the Safety and Tolerability of the CD40 Agonistic Monoclonal Antibody APX005M in Pediatric Subjects with Recurrent/Refractory Brain Tumors and Newly Diagnosed Brain Stem Glioma
Diagnosis: Stratum 2 newly diagnosed Diffuse Intrinsic Pontine Glioma (DIPG)

Relapse/Refractory

PBTC-049: A Phase I Study of Savolitinib in Recurrent, Progressive or Refractory Primary CNS Tumors
Diagnosis: Medulloblastoma, DIPG, and HGG

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

Relapse/Refractory

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: Medulloblastoma, DIPG, and HGG

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

PBTC056: A Phase I Study of the Adam-10 Inhibitor, INCB7839 in Children With Recurrent/Progressive High-Grade Gliomas to Target Microenvironmental Neuroligin-3
Diagnosis: HGG

Ependymoma

Newly Diagnosed

There are currently no open trials

Relapse/Refractory

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 D-Patients must have a histologically confirmed diagnosis of ependymoma that is recurrent, progressive or refractory following therapy which included radiotherapy.

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

Relapse/ Refractory

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 E- Patients must have a histologically confirmed diagnosis of medulloblastoma that is recurrent, progressive or refractory following therapy which included radiotherapy.

PBTC-049: A Phase I Study of Savolitinib in Recurrent, Progressive or Refractory Primary CNS Tumors
Diagnosis: Medulloblastoma, DIPG, and HGG

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

SJATRT: Phase 2 Study of Alisertib as a single agent in Recurrent or Progressive Central Nervous System (CNS) Atypical Teratoid Rhabdoid Tumors (ATRT) and Extra-CNS Malignant Rhabdoid Tumors (MRT) and in Combination Therapy in Newly Diagnoses ATRT
Diagnosis: Atypical Teratoid Rhabdoid Tumors (ATRT)

Relapse/Refractory

PBTC-051: Phase I Study to Evaluate the Safety and Tolerability of the CD40 Agonistic Monoclonal Antibody APX005M in Pediatric Subjects with Recurrent/Refractory Brain Tumors and Newly Diagnosed Brain Stem Glioma
Diagnosis: Stratum I Recurrent or Refractory Primary Malignant CNS tumor patients

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

Relapse/Refractory

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

Other

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 & 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

Cellectar: An Open-Label, Dose Escalation, Efficacy, and Safety Study of CLR 131 in Children, Adolescents, and Young Adults With Select Solid Tumors, Lymphoma, and Malignant Brain Tumors
Diagnosis: Previously confirmed (histologically or cytologically) pediatric solid tumor (e.g., neuroblastoma, sarcoma), lymphoma (including Hodgkin's lymphoma), or malignant brain tumors that are clinically or radiographically suspected to be relapsed, refractory, or recurrent for which there are no standard treatment options with curative potential

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