Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Oct 14, 2026, 3:50 PM EDT

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Showing 121–144 of 1,146 matching trials from the live ClinicalTrials.gov search.
Conditions
Alzheimer's Disease, Huntington's Disease
Interventions
Dimebon
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 55 Years
Enrollment
45 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009
U.S. locations
1
States / cities
Glendale, California
Conditions
Alzheimer's Disease, Huntington's Disease
Interventions
Dimebon
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 75 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009
U.S. locations
2
States / cities
Miami, Florida • Saint Paul, Minnesota
Conditions
Tourette Syndrome
Interventions
PF-04457845, Placebo
Drug
Lead sponsor
Yale University
Other
Eligibility
18 Years to 60 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
New Haven, Connecticut
Conditions
Parkinson Disease, Huntington Disease
Interventions
[123I]MNI-420
Drug
Lead sponsor
Institute for Neurodegenerative Disorders
Other
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2015
U.S. locations
1
States / cities
New Haven, Connecticut
Conditions
Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth, Charcot-Marie-Tooth Disease, Type IA, Charcot-Marie-Tooth Disease Type 2A, Charcot-Marie-Tooth Disease Type 2, Charcot-Marie-Tooth Disease, Type 2C, Charcot-Marie-Tooth Disease Type 2A2B, Charcot-Marie-Tooth Disease Type 2B2, Charcot-Marie-Tooth Disease Type 2A1, Charcot-Marie-Tooth Disease Type 4B1, Charcot-Marie-Tooth Disease, Type IB, Charcot-Marie-Tooth Disease Type 2B1, Charcot-Marie-Tooth Disease Type 2U (Diagnosis), Charcot-Marie-Tooth Disease Type 4A, Charcot-Marie-Tooth Disease, Type 4A, Axonal Form, Charcot-Marie-Tooth Disease Type 2A2A, Charcot-Marie-Tooth Disease Type 2S (Disorder), Charcot-Marie-Tooth Disease and Deafness, Charcot-Marie-Tooth Disease Type 4B2, Charcot-Marie-Tooth Disease Type 4H, Charcot-Marie-Tooth Disease Type 1F, Charcot-Marie-Tooth Disease Type 4C, Charcot-Marie-Tooth Disease Type 4E, Charcot-Marie-Tooth Disease Type 1D, Charcot-Marie-Tooth Disease Type 2Q (Diagnosis), Charcot-Marie-Tooth Disease Type 2A2, Charcot-Marie-Tooth Disease Type 2N (Diagnosis), Charcot-Marie-Tooth Disease Type 2B5, Charcot-Marie-Tooth Disease Type 2D, Charcot-Marie-Tooth Disease Type 4D, Charcot-Marie-Tooth Disease Type 2K, Charcot-Marie-Tooth Disease Type 2L (Diagnosis), Charcot-Marie-Tooth Disease Type 2T, Charcot-Marie-Tooth Disease Type 2I, Charcot-Marie-Tooth Disease Type 2J, Charcot-Marie-Tooth Disease Type 2E, Charcot-Marie-Tooth Disease Type 2G, Charcot-Marie-Tooth Disease Type 1C, Charcot-Marie-Tooth Disease Type 2R, Charcot-Marie-Tooth Disease Type 2O (Diagnosis), Charcot-Marie-Tooth Disease Type 2M, Charcot-Marie-Tooth Disease Type 2P, Charcot-Marie-Tooth Disease Type 2Y, Charcot-Marie-Tooth Disease Type 4F (Diagnosis), Charcot-Marie-Tooth Disease Type 4B3, Charcot-Marie-Tooth Disease Type 2H, HNPP, X-Linked Charcot-Marie-Tooth Disease, Charcot Marie Tooth Disease (CMT)
Interventions
Not listed
Lead sponsor
Hereditary Neuropathy Foundation
Government
Eligibility
Not listed
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2029
U.S. locations
1
States / cities
New York, New York
Recruiting No phase listed Observational Accepts healthy volunteers

Extracellular RNA Biomarkers of Myotonic Dystrophy

NCT05020002
Conditions
Myotonic Dystrophy
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
5 Years and older
Enrollment
215 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2027
U.S. locations
3
States / cities
Boston, Massachusetts • Dallas, Texas
Recruiting No phase listed Observational Accepts healthy volunteers

Muscle Health Measurements Using Electrical Impedance Myography

NCT07502989
Conditions
Myopathy, Muscular Dystrophies, Myositis, Myofibrillar Myopathy, Congenital Myopathy, Distal Myopathy, Myopathies
Interventions
Electrical Impedance Myography
Device
Lead sponsor
Beth Israel Deaconess Medical Center
Other
Eligibility
18 Years to 89 Years
Enrollment
150 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Giant Axonal Neuropathy (GAN)
Interventions
scAAV9/JeT-GAN
Genetic
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
10 Years to 25 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
Dallas, Texas
Active, not recruiting Phase 1Phase 2 Interventional Results available

A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease

NCT04537377
Conditions
Wilson's Disease
Interventions
VTX-801
Genetic
Lead sponsor
Vivet Therapeutics SAS
Industry
Eligibility
18 Years to 65 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
6
States / cities
Sacramento, California • New Haven, Connecticut • Orlando, Florida + 3 more
Conditions
Tourette's Disorder
Interventions
Aripiprazole, Placebo
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
7 Years to 17 Years
Enrollment
135 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
20
States / cities
Little Rock, Arkansas • Sacramento, California • Santa Ana, California + 16 more
Conditions
Huntington's Disease
Interventions
Not listed
Lead sponsor
University College, London
Other
Eligibility
11 Years to 75 Years
Enrollment
2,500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2017
U.S. locations
6
States / cities
Englewood, Colorado • Washington D.C., District of Columbia • Baltimore, Maryland + 3 more
Conditions
Wilson Disease
Interventions
NCC-Sp Assay
Diagnostic Test
Lead sponsor
Orphalan
Industry
Eligibility
18 Years and older
Enrollment
50 participants
Timeline
2023 – 2025
U.S. locations
9
States / cities
Sacramento, California • New Haven, Connecticut • Miami, Florida + 6 more
Not listed No phase listed Observational Accepts healthy volunteers

Glucose Metabolic, Amyloid, and Tau Brain Imaging in Down Syndrome and Dementia

NCT00966017
Conditions
Down Syndrome, Alzheimer's Disease
Interventions
Not listed
Lead sponsor
National Institute on Aging (NIA)
NIH
Eligibility
45 Years and older
Enrollment
108 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2014
U.S. locations
1
States / cities
Los Angeles, California
Completed Phase 2 Interventional Results available

Creatine Safety & Tolerability in Huntington's Disease

NCT01412151
Conditions
Huntington's Disease (HD)
Interventions
Creatine monohydrate
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2011
U.S. locations
1
States / cities
Charlestown, Massachusetts
Conditions
Leiomyomatosis
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Alzheimer's Disease, Dementia, Alzheimer's Disease, Familial
Interventions
lecanemab
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
4
States / cities
Birmingham, Alabama • Indianapolis, Indiana • St Louis, Missouri + 1 more
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers

Palliative Care Needs of Children With Rare Diseases and Their Families

NCT06938542
Conditions
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome, Early Infantile Epileptic Encephalopathy, FOXG1 Syndrome, KBG Syndrome, Noonan Syndrome, Severe Hemophilia A, Short Bowel Syndrome, Beta-Propeller Protein-Associated Neurodegeneration, Brain Injury of Prematurity With Periventricular Leukomalacia, Chromosome 17p13.3 Microdeletion Syndrome, Chromosome 1q43-1q44 Deletion, Cockayne Syndrome, Congenital Diaphragmatic Hernia, End-Stage Renal Disease With Cloacal Anomaly, Mitochondrial Depletion Disorder, Severe Factor VII Deficiency
Interventions
Family Centered pediatric palliative care for family caregivers of children with rare diseases.
Behavioral
Lead sponsor
Children's National Research Institute
Other
Eligibility
12 Months to 99 Years
Enrollment
480 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2029
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Conditions
Tuberous Sclerosis Complex, Epilepsy
Interventions
TAVT-18 (sirolimus)
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
1 Day to 6 Months
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Cincinnati, Ohio
Conditions
Muscular Dystrophies, Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Limb Girdle Muscular Dystrophy, FSHD, Cerebral Palsy, Becker Muscular Dystrophy
Interventions
Abilitech Assist
Device
Lead sponsor
AbiliTech Medical Inc.
Industry
Eligibility
10 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Saint Paul, Minnesota
Conditions
NF1 Mutation, Neurofibroma Plexiform, Neurofibroma, Plexiform, Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PNs), Neurofibromatosis Type 1 (NF1)
Interventions
PAS-004 Tablets
Drug
Lead sponsor
Pasithea Therapeutics Corp.
Industry
Eligibility
18 Years and older
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Birmingham, Alabama
Conditions
Friedreich Ataxia, Friedreich's Ataxia
Interventions
Nomlabofusp, Placebo
Drug
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
12 Years to 40 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
4
States / cities
Tampa, Florida • Chicago, Illinois • Eatontown, New Jersey + 1 more
Conditions
Neurofibromatosis
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
8 Years to 75 Years
Enrollment
269 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy Study for Children With CLN5 Batten Disease

NCT05228145
Conditions
Neuronal Ceroid Lipofuscinosis CLN5
Interventions
NGN-101
Genetic
Lead sponsor
Neurogene Inc.
Industry
Eligibility
3 Years to 9 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
1
States / cities
Rochester, New York
Conditions
Myotonic Dystrophy 1, DM1
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
18 Years to 70 Years
Enrollment
700 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
10
States / cities
La Jolla, California • Los Angeles, California • Denver, Colorado + 7 more