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ClinicalTrials.gov public records Last synced Oct 20, 2026, 3:57 PM EDT

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Showing 625–648 of 1,147 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Completed Phase 1 Interventional

A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of SAGE-718 Oral Solution in Patients With Huntington's Disease - Part B

NCT03787758
View directory record Official record
Conditions
Huntington Disease
Interventions
SAGE-718
Drug
Lead sponsor
Supernus Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019
U.S. locations
2
States / cities
Long Beach, California • Marlton, New Jersey
Source: ClinicalTrials.gov public record
Updated Sep 17, 2025 · Synced Oct 20, 2026, 3:57 PM EDT
Completed No phase listed Observational

Making Sense of a Positive Genetic Test Result for Huntington Disease

NCT00539747
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
4
States / cities
Baltimore, Maryland • Bethesda, Maryland • Pittsburgh, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 20, 2026, 3:57 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease

NCT06609889
View directory record Official record
Conditions
Lafora Disease
Interventions
ION283
Genetic
Lead sponsor
Berge Minassian
Other
Eligibility
10 Years to 18 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Dec 18, 2025 · Synced Oct 20, 2026, 3:57 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Characterization of the Cardiac Phenotype of Friedreich's Ataxia (FRDA)

NCT02316314
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Cardiac magnetic resonance imaging (CMR), Exercise-stress test, Echocardiogram (ECHO), Cardiac-related blood studies
Procedure
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
12 Years to 50 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Oct 20, 2026, 3:57 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry

NCT00082108
View directory record Official record
Conditions
Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophy, Muscular Dystrophy, Myotonic Dystrophy Type 1, Myotonic Dystrophy Type 2, Congenital Myotonic Dystrophy, PROMM (Proximal Myotonic Myopathy), Steinert's Disease, Myotonic Muscular Dystrophy
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
Not listed
Enrollment
3,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2028
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Oct 15, 2025 · Synced Oct 20, 2026, 3:57 PM EDT
Completed Phase 2 Interventional Results available

A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia

NCT02255435
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg, Placebo, Omaveloxolone Capsules, 20 mg, Omaveloxolone Capsules, 40 mg, Omaveloxolone Capsules, 80 mg, Omaveloxolone Capsules, 160 mg, Omaveloxolone Capsules, 300 mg, Omaveloxolone Capsules, 150 mg
Drug
Lead sponsor
Biogen
Industry
Eligibility
16 Years to 40 Years
Enrollment
172 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2025
U.S. locations
7
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 20, 2026, 3:57 PM EDT
Completed Phase 1 Interventional Results available

Safety and Efficacy of Pembrolizumab (MK-3475) Plus Binimetinib Alone or Pembrolizumab Plus Chemotherapy With or Without Binimetinib in Metastatic Colorectal Cancer (mCRC) Participants (MK-3475-651/KEYNOTE-651)

NCT03374254
View directory record Official record
Conditions
Metastatic Colorectal Cancer
Interventions
Pembrolizumab, Binimetinib, Oxaliplatin, Leucovorin, 5-Fluorouracil [5-FU], Irinotecan
Biological · Drug
Lead sponsor
Merck Sharp & Dohme LLC
Industry
Eligibility
18 Years and older
Enrollment
116 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
10
States / cities
Duarte, California • Aurora, Colorado • New Haven, Connecticut + 7 more
Source: ClinicalTrials.gov public record
Updated Nov 21, 2024 · Synced Oct 20, 2026, 3:57 PM EDT
Not yet recruiting Phase 2 Interventional

Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade Glioma

NCT07521657
View directory record Official record
Conditions
Neurofibromatosis 1 (NF1)
Interventions
mirdametinib with radiation, mirdametinib
Combination Product · Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
12 Years to 89 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2027 – 2032
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 20, 2026, 3:57 PM EDT
Completed Phase 2 Interventional Results available

Ecopipam Tablets to Study Tourette Syndrome in Children and Adolescents - Open Label Extension

NCT04114539
View directory record Official record
Conditions
Tourette Syndrome in Children, Tourette Syndrome in Adolescence
Interventions
Ecopipam
Drug
Lead sponsor
Emalex Biosciences Inc.
Industry
Eligibility
6 Years to 18 Years
Enrollment
124 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
53
States / cities
Dothan, Alabama • Phoenix, Arizona • Anaheim, California + 45 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2024 · Synced Oct 20, 2026, 3:57 PM EDT
Terminated No phase listed Observational

Natural History of Autosomal Dominant Optic Atrophy (ADOA), Caused by OPA1 Mutation

NCT06140329
View directory record Official record
Conditions
Autosomal Dominant Optic Atrophy, Optic Atrophy, Autosomal Dominant, Optic Atrophies, Hereditary, Kjer Optic Atrophy
Interventions
Not listed
Lead sponsor
PYC Therapeutics
Industry
Eligibility
8 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
2
States / cities
Miami, Florida • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Mar 14, 2025 · Synced Oct 20, 2026, 3:57 PM EDT
Active, not recruiting Phase 3 Interventional

Open Pilot Trial of BHV-4157

NCT03408080
View directory record Official record
Conditions
Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia 3, Spinocerebellar Ataxia Type 6, MSA-C
Interventions
BHV-4157
Drug
Lead sponsor
University of California, Los Angeles
Other
Eligibility
18 Years to 75 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated May 9, 2024 · Synced Oct 20, 2026, 3:57 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Advancing Understanding of Transportation Options

NCT04141891
View directory record Official record
Conditions
Diabetic Retinopathy, Macular Degeneration, Glaucoma, Retinitis Pigmentosa, Vision Disorders, Acute Coronary Syndrome, Implantable Defibrillator User, Congestive Heart Failure, Hypertrophic Obstructive Cardiomyopathy, Orthostatic Hypotension, Syncope, Presyncope, Narcolepsy, Dementia, Multiple Sclerosis, Parkinson Disease, Brain Injuries, Spinal Cord Injuries, Stroke, Vertigo, Dizziness, Seizures, Substance Use, Insulin Dependent Diabetes Mellitus, Arthritis, Foot--Abnormalities, Chronic Obstructive Pulmonary Disease, Obstructive Sleep Apnea, End Stage Renal Disease, Sleep Apnea, Insomnia, Restless Legs Syndrome
Interventions
Driving Decision Aid, Older Drivers Website
Behavioral
Lead sponsor
University of Colorado, Denver
Other
Eligibility
18 Years and older
Enrollment
529 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2023
U.S. locations
3
States / cities
La Jolla, California • Aurora, Colorado • Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Feb 21, 2025 · Synced Oct 20, 2026, 3:57 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Nicotine Treatment of Cognitive Decline in Down Syndrome

NCT01778946
View directory record Official record
Conditions
Down Syndrome, Mild Cognitive Impairment
Interventions
Low Dose Nicotine (7mg), Moderate Dose Nicotine (14mg)
Drug
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
25 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2021
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated May 10, 2022 · Synced Oct 20, 2026, 3:57 PM EDT
Not yet recruiting Not applicable Interventional

iBCI Optimization for Veterans With Paralysis

NCT05470478
View directory record Official record
Conditions
Spinal Cord Injury, Amyotrophic Lateral Sclerosis, Brain Stem Infarctions, Locked-in Syndrome, Muscular Dystrophy
Interventions
Mobile neural decoding platform (mobile iBCI)
Device
Lead sponsor
VA Office of Research and Development
Federal
Eligibility
18 Years to 80 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Providence, Rhode Island
Source: ClinicalTrials.gov public record
Updated Jul 27, 2026 · Synced Oct 20, 2026, 3:57 PM EDT
Completed No phase listed Observational

Apathy in Tourette Syndrome and Changes in Quality of Life

NCT02765178
View directory record Official record
Conditions
Tourette Syndrome, Apathy
Interventions
Children's Motivation Scale, Yale Global Tic Severity Scale (YGTSS), Studies Depression Scale for Children (CES-DC), Gilles de la Tourette Syndrome Quality Of Life scale, Demographic Data
Other
Lead sponsor
University of Florida
Other
Eligibility
12 Years to 16 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jul 6, 2017 · Synced Oct 20, 2026, 3:57 PM EDT
Recruiting Not applicable Interventional

Investigating Changes in Premonitory Urges During Habit Reversal Training for Tics

NCT06825520
View directory record Official record
Conditions
Tics, Tourette Disorder, Tourette Syndrome in Children, Tourette Syndrome in Adolescence
Interventions
Habit Reversal Training
Behavioral
Lead sponsor
University of Utah
Other
Eligibility
8 Years to 17 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 28, 2025 · Synced Oct 20, 2026, 3:57 PM EDT
Terminated Phase 2 Interventional Results available

Study of Sutent®/Sunitinib (SU11248) in Subjects With NF-1 Plexiform Neurofibromas

NCT01402817
View directory record Official record
Conditions
Neurofibromatosis, NF1, Plexiform Neurofibromas
Interventions
Sutent®/Sunitinib
Drug
Lead sponsor
Indiana University
Other
Eligibility
3 Years to 65 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Mar 14, 2018 · Synced Oct 20, 2026, 3:57 PM EDT
Completed Phase 3 Interventional Results available

An Open-label Extension Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)

NCT02544750
View directory record Official record
Conditions
Tuberous Sclerosis Complex, Seizures
Interventions
GWP42003-P
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
1 Year to 65 Years
Enrollment
199 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 14, 2022 · Synced Oct 20, 2026, 3:57 PM EDT
Active, not recruiting Phase 3 Interventional

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

NCT04042025
View directory record Official record
Conditions
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA
Interventions
Onasemnogene Abeparvovec-xioi
Biological
Lead sponsor
Novartis Gene Therapies
Industry
Eligibility
Not listed
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2035
U.S. locations
18
States / cities
Palo Alto, California • Aurora, Colorado • Chicago, Illinois + 14 more
Source: ClinicalTrials.gov public record
Updated Sep 24, 2026 · Synced Oct 20, 2026, 3:57 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Dominantly Inherited Alzheimer Network (DIAN)

NCT00869817
View directory record Official record
Conditions
Alzheimer's Disease
Interventions
Not listed
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years and older
Enrollment
700 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2031
U.S. locations
8
States / cities
Jacksonville, Florida • Indianapolis, Indiana • Boston, Massachusetts + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 20, 2026, 3:57 PM EDT
Completed No phase listed Observational

A Pre-Cellular Therapy Observational Study in Early Huntington's Disease

NCT01937923
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
University of California, Davis
Other
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Nov 1, 2016 · Synced Oct 20, 2026, 3:57 PM EDT
Completed Phase 2 Interventional

The Nutritional Supplement Phosphatidylserine in Patients With Familial Dysautonomia

NCT02276716
View directory record Official record
Conditions
Familial Dysautonomia
Interventions
Phosphatidylserine
Dietary Supplement
Lead sponsor
NYU Langone Health
Other
Eligibility
12 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 21, 2021 · Synced Oct 20, 2026, 3:57 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT

NCT05739643
View directory record Official record
Conditions
Krabbe Disease
Interventions
FBX-101
Biological
Lead sponsor
Forge Biologics, Inc
Industry
Eligibility
Up to 18 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
3
States / cities
Orange, California • Ann Arbor, Michigan • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Nov 20, 2025 · Synced Oct 20, 2026, 3:57 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Wearable Technology to Assess Gait Function in SMA and DMD

NCT04193085
View directory record Official record
Conditions
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy
Interventions
Observational
Other
Lead sponsor
Columbia University
Other
Eligibility
5 Years and older
Enrollment
39 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2023
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 23, 2024 · Synced Oct 20, 2026, 3:57 PM EDT