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ClinicalTrials.gov public records Last synced Oct 23, 2026, 7:54 AM EDT

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Showing 961–984 of 1,148 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Completed No phase listed Observational Accepts healthy volunteers

Genetic Risk: Whether, When, and How to Tell Adolescents

NCT03421327
View directory record Official record
Conditions
Huntington Disease, Hereditary Breast and Ovarian Cancer, Hereditary Cancer, Hereditary Non-polyposis Colon Cancer, Hereditary Non-Polyposis Colorectal Cancer Syndrome
Interventions
Not listed
Lead sponsor
Johns Hopkins University
Other
Eligibility
15 Years and older
Enrollment
85 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 7, 2019 · Synced Oct 23, 2026, 7:54 AM EDT
Completed Not applicable Interventional

NCGENES: North Carolina Clinical Genomic Evaluation by NextGen Exome Sequencing

NCT01969370
View directory record Official record
Conditions
Cancer, Cardiovascular Disease, Neurologic Dysfunction, Congenital Abnormalities, Hearing Loss
Interventions
Experimental
Behavioral
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
Not listed
Enrollment
645 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated May 3, 2017 · Synced Oct 23, 2026, 7:54 AM EDT
Terminated Phase 4 Interventional

Safety Study of Galantamine in Tic Disorders

NCT00226824
View directory record Official record
Conditions
Tourette's Syndrome, Motor Tic Disorder, Vocal Tic Disorder
Interventions
galantamine
Drug
Lead sponsor
Parkinson's Disease and Movement Disorders Center
Other
Eligibility
18 Years to 50 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
1
States / cities
Albany, New York
Source: ClinicalTrials.gov public record
Updated Aug 25, 2009 · Synced Oct 23, 2026, 7:54 AM EDT
Active, not recruiting Phase 3 Interventional

A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia

NCT06953583
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Omaveloxolone, Placebo
Drug
Lead sponsor
Biogen
Industry
Eligibility
2 Years to 15 Years
Enrollment
255 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
7
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 23, 2026, 7:54 AM EDT
Completed No phase listed Observational

Project AWARE 2.0 to Improve Awareness, Willingness and Ability for Research and Enrollment in Clinical Research

NCT06343220
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
Huntington Study Group
Network
Eligibility
18 Years and older
Enrollment
131 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Dec 23, 2025 · Synced Oct 23, 2026, 7:54 AM EDT
Recruiting No phase listed Observational

Myotonic Dystrophy Family Registry

NCT02398786
View directory record Official record
Conditions
Myotonic Dystrophy, Congenital Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Dystrophy 2, Dystrophia Myotonica, Dystrophia Myotonica 1, Dystrophia Myotonica 2, Myotonia Dystrophica, Myotonic Dystrophy, Congenital, Myotonic Myopathy, Proximal, PROMM (Proximal Myotonic Myopathy), Proximal Myotonic Myopathy, Steinert Disease, Steinert Myotonic Dystrophy, Steinert's Disease, Myotonia Atrophica
Interventions
Patient-entered data
Other
Lead sponsor
Myotonic Dystrophy Foundation
Other
Eligibility
Not listed
Enrollment
3,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2030
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Nov 21, 2024 · Synced Oct 23, 2026, 7:54 AM EDT
Completed Phase 1 Interventional Results available

Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1

NCT03959189
View directory record Official record
Conditions
Myotonic Dystrophy, Type 1 (DM1), Myotonic Dystrophy
Interventions
ERX-963, Placebo
Drug
Lead sponsor
Expansion Therapeutics, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
4
States / cities
Palo Alto, California • Miami, Florida • Iowa City, Iowa + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2021 · Synced Oct 23, 2026, 7:54 AM 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 23, 2026, 7:54 AM EDT
Completed Phase 3 Interventional Results available

Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

NCT05386680
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
OAV101
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 17 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
3
States / cities
Boston, Massachusetts • Norfolk, Virginia • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 23, 2026, 7:54 AM EDT
Completed Phase 1 Interventional Results available

Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy

NCT02161380
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy
Interventions
injection of scAAV2-P1ND4v2 1.18x10e9 vg (Low),, injection of scAAV2-P1ND4v2 5.81 X10e9 vg (Med), injection of scAAV2-P1ND4v2 2.4 X10e10vg (High), injection of scAAV2-P1ND4v2 1.0 X10e11vg (Higher)
Drug
Lead sponsor
Byron Lam
Other
Eligibility
15 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2025
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Jun 5, 2025 · Synced Oct 23, 2026, 7:54 AM EDT
Recruiting No phase listed Observational

Clinical Course Of Disease In Participants With FA-CM

NCT06865482
View directory record Official record
Conditions
Friedreich Ataxia, Cardiomyopathy
Interventions
Not listed
Lead sponsor
Lexeo Therapeutics
Industry
Eligibility
6 Years and older
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
8
States / cities
La Jolla, California • Tampa, Florida • Indianapolis, Indiana + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 19, 2026 · Synced Oct 23, 2026, 7:54 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

Impact of Deutetrabenazine on Functional Speech and Gait Dynamics in Huntington Disease

NCT04713982
View directory record Official record
Conditions
Huntington Disease
Interventions
Deutetrabenazine
Drug
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Aug 5, 2026 · Synced Oct 23, 2026, 7:54 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Cognitive Assessment Battery (CAB) Beta Study

NCT01290861
View directory record Official record
Conditions
Huntington's Disease
Interventions
Not listed
Lead sponsor
CHDI Foundation, Inc.
Other
Eligibility
25 Years to 55 Years
Enrollment
255 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2011
U.S. locations
12
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 9 more
Source: ClinicalTrials.gov public record
Updated Aug 3, 2012 · Synced Oct 23, 2026, 7:54 AM EDT
Completed Not applicable Interventional

Evaluation of an Online Comprehensive Behavioral Intervention for Tics (CBIT) Therapist Training Program

NCT05547854
View directory record Official record
Conditions
Tics, Tic Disorders, Tourette Syndrome
Interventions
Behavior Therapy Training Institute, CBIT-Trainer
Behavioral
Lead sponsor
University of Utah
Other
Eligibility
7 Years and older
Enrollment
284 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
2
States / cities
Salt Lake City, Utah • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 23, 2026, 7:54 AM EDT
Completed No phase listed Observational

Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis

NCT01035424
View directory record Official record
Conditions
Batten Disease, Late Infantile Neuronal Ceroid Lipofuscinosis
Interventions
Not listed
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
2 Years to 18 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 29, 2020 · Synced Oct 23, 2026, 7:54 AM EDT
Completed Phase 1Phase 2 Interventional

Creatine Therapy for Huntington's Disease

NCT00026988
View directory record Official record
Conditions
Huntington's Disease
Interventions
Creatine
Drug
Lead sponsor
National Center for Complementary and Integrative Health (NCCIH)
NIH
Eligibility
18 Years and older
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
4
States / cities
Boston, Massachusetts • Long Island City, New York • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 18, 2006 · Synced Oct 23, 2026, 7:54 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders

NCT06839469
View directory record Official record
Conditions
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy (DMD)
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
5 Years and older
Enrollment
106 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2028
U.S. locations
3
States / cities
Palo Alto, California • Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 9, 2026 · Synced Oct 23, 2026, 7:54 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives

NCT05635266
View directory record Official record
Conditions
Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease, Amyloidosis, Ankylosing Spondylitis, Arthritis, Alopecia Areata, Asthma, Atopic Dermatitis, Autism, Autoimmune Hepatitis, Behcet's Disease, Beta-Thalassemia, Cancer, Celiac Disease, Kidney Diseases, COPD, Crohn Disease, Cystic Fibrosis, Diabetes, Dravet Syndrome, DMD, Fibromyalgia, Graves Disease, Thyroid Diseases, Hepatitis, Hidradenitis Suppurativa, ITP, Leukemia, ALS, Lupus or SLE, Lymphoma, Multiple Sclerosis, Myasthenia Gravis, Heart Diseases, Parkinson Disease, Pemphigus Vulgaris, Cirrhosis, Psoriasis, Schizophrenia, Scleroderma, Sickle Cell Disease, Stroke, Ulcerative Colitis, Vasculitis, Vitiligo
Interventions
Specimen sample
Diagnostic Test
Lead sponsor
Sanguine Biosciences
Industry
Eligibility
18 Years to 85 Years
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2025
U.S. locations
1
States / cities
Waltham, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 7, 2024 · Synced Oct 23, 2026, 7:54 AM EDT
Completed No phase listed Observational

Development of the Virtual Unified Huntington's Disease Rating Scale

NCT05748288
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
Huntington Study Group
Network
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
17
States / cities
Birmingham, Alabama • Little Rock, Arkansas • La Jolla, California + 13 more
Source: ClinicalTrials.gov public record
Updated Sep 28, 2023 · Synced Oct 23, 2026, 7:54 AM EDT
Recruiting No phase listed Observational

Cardiovascular Multimodality Imaging Study

NCT06610019
View directory record Official record
Conditions
Non-ischemic Cardiomyopathy, Cardiomyopathies, Hypertrophic Cardiomyopathy, Right Ventricular Arrhythmogenic Cardiomyopathy, Cardiac Amyloidosis, Anderson Fabry Disease, Sarcoidosis, Cancer Therapy-related Cardiac Dysfunction, Ventricular Arrythmia, Heart Failure
Interventions
Not listed
Lead sponsor
Montefiore Medical Center
Other
Eligibility
18 Years and older
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
1
States / cities
The Bronx, New York
Source: ClinicalTrials.gov public record
Updated Aug 10, 2026 · Synced Oct 23, 2026, 7:54 AM EDT
Active, not recruiting No phase listed Observational

An Observational Study Using Artificial Intelligence (AI) Algorithms on Electrocardiography (ECG), Point-of-care Ultrasound (POCUS), and Transthoracic Echocardiophy (TTE) to Estimate the Under-diagnosis of Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Across a Diverse Range of US Health Systems.

NCT07062848
View directory record Official record
Conditions
Transthyretin (TTR) Amyloid Cardiomyopathy
Interventions
AI Toolkit for ATTR-CM Diagnosis
Diagnostic Test
Lead sponsor
Yale University
Other
Eligibility
50 Years to 95 Years
Enrollment
1,500,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
12
States / cities
San Francisco, California • New Haven, Connecticut • Chicago, Illinois + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Oct 23, 2026, 7:54 AM EDT
Recruiting Phase 2 Interventional

Multimodal Profiling of Response to Pediatric Comprehensive Behavioral Intervention for Tics

NCT06194305
View directory record Official record
Conditions
Tourette Syndrome, Persistent Tic Disorder
Interventions
CBIT
Other
Lead sponsor
University of Minnesota
Other
Eligibility
10 Years to 17 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
2
States / cities
San Diego, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Oct 23, 2026, 7:54 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Phenotype, Genotype and Biomarkers 2

NCT04875416
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Hereditary Spastic Paraplegia, Primary Lateral Sclerosis, Progressive Muscular Atrophy, Frontotemporal Dementia
Interventions
Not listed
Lead sponsor
University of Miami
Other
Eligibility
7 Years and older
Enrollment
217 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2031
U.S. locations
3
States / cities
Miami, Florida • Kansas City, Kansas • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 15, 2026 · Synced Oct 23, 2026, 7:54 AM EDT
Completed No phase listed Observational

Study of Disease Severity in Adults With Neurofibromatosis Type 1 (NF1)

NCT00111384
View directory record Official record
Conditions
Neurofibromatosis Type 1, Legius Syndrome
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
2 Years to 100 Years
Enrollment
313 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Oct 23, 2026, 7:54 AM EDT