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ClinicalTrials.gov public records Last synced Oct 23, 2026, 1:11 AM EDT

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Showing 889–912 of 1,148 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Recruiting Phase 2 Interventional

A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1

NCT06541847
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
HLX-1502
Drug
Lead sponsor
Healx Limited
Industry
Eligibility
12 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
14
States / cities
Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Oct 23, 2026, 1:11 AM EDT
Not listed No phase listed Observational

Exercise Effect on Transthyretin Stability

NCT04986826
View directory record Official record
Conditions
Amyloidosis Transthyretin, Athlete Heart
Interventions
Not listed
Lead sponsor
Oregon Health and Science University
Other
Eligibility
18 Years and older
Enrollment
24 participants
Timeline
2021
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Aug 3, 2021 · Synced Oct 23, 2026, 1:11 AM EDT
Recruiting Phase 2 Interventional

Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy

NCT07721025
View directory record Official record
Conditions
Friedreich Ataxia, Cardiomyopathy, Secondary
Interventions
LX2006, Usual Care
Genetic · Other
Lead sponsor
Lexeo Therapeutics
Industry
Eligibility
6 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Oct 23, 2026, 1:11 AM EDT
Terminated No phase listed Observational

Giant Axonal Neuropathy Natural History Study

NCT01503125
View directory record Official record
Conditions
Giant Axonal Neuropathy
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
Not listed
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 27, 2017 · Synced Oct 23, 2026, 1:11 AM EDT
Recruiting No phase listed Observational

Sensory Symptoms in Tourette Syndrome

NCT03851484
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Not listed
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years and older
Enrollment
214 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2028
U.S. locations
2
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 13, 2026 · Synced Oct 23, 2026, 1:11 AM EDT
Active, not recruiting Phase 2 Interventional

Efficacy and Safety of Gemlapodect (NOE-105) in Adults and Adolescents With Tourette Syndrome

NCT06315751
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Gemlapodect, Placebo
Drug
Lead sponsor
Noema Pharma AG
Industry
Eligibility
18 Years and older
Enrollment
164 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
31
States / cities
Sun City, Arizona • Bellflower, California • Glendale, California + 28 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2026 · Synced Oct 23, 2026, 1:11 AM EDT
Completed Phase 1 Interventional

Pomalidomide in Treating Younger Patients With Recurrent, Progressive, or Refractory Central Nervous System Tumors

NCT02415153
View directory record Official record
Conditions
Neurofibromatosis Type 1, Recurrent Childhood Brain Stem Glioma, Recurrent Childhood Visual Pathway Glioma, Recurrent Primary Central Nervous System Neoplasm, Refractory Primary Central Nervous System Neoplasm
Interventions
Laboratory Biomarker Analysis, Pharmacological Study, Pomalidomide
Other · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 20 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
12
States / cities
Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 9 more
Source: ClinicalTrials.gov public record
Updated Apr 25, 2022 · Synced Oct 23, 2026, 1:11 AM EDT
Completed Phase 1 Interventional

Study of Ranolazine in Myotonia Congenita, Paramyotonia Congenita and Myotonic Dystrophy Type 1

NCT02251457
View directory record Official record
Conditions
Myotonia Congenita, Paramyotonia Congenita, Myotonic Dystrophy 1
Interventions
Ranolazine
Drug
Lead sponsor
Ohio State University
Other
Eligibility
18 Years to 100 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 5, 2019 · Synced Oct 23, 2026, 1:11 AM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Brain Tissue Collection for Neuropathological Studies

NCT00001260
View directory record Official record
Conditions
Bipolar Disorder, Depression, Anxiety Disorders, Schizophrenia, Tourette's Syndrome, Brain Diseases
Interventions
Not listed
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
Not listed
Enrollment
2,161 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1990 – 2017
U.S. locations
3
States / cities
Washington D.C., District of Columbia • Bethesda, Maryland • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Dec 9, 2019 · Synced Oct 23, 2026, 1:11 AM EDT
Recruiting Phase 4 Interventional Accepts healthy volunteers

Ultrasound Therapy In Cardiac Amyloidosis

NCT04667494
View directory record Official record
Conditions
Amyloidosis Cardiac
Interventions
Sonotherapy
Combination Product
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years to 90 Years
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 21, 2026 · Synced Oct 23, 2026, 1:11 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma

NCT01089101
View directory record Official record
Conditions
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma, Recurrent Neurofibromatosis Type 1, Recurrent Visual Pathway Glioma, Refractory Neurofibromatosis Type 1, Refractory Visual Pathway Glioma
Interventions
Biospecimen Collection, Selumetinib
Procedure · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 21 Years
Enrollment
217 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2027
U.S. locations
17
States / cities
Los Angeles, California • Palo Alto, California • San Francisco, California + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Oct 23, 2026, 1:11 AM EDT
Completed Phase 4 Interventional Results available

Effects of Ondansetron in Obsessive-compulsive and Tic Disorders

NCT03239210
View directory record Official record
Conditions
Obsessive-Compulsive Disorder, Tic Disorders, Tourette Syndrome
Interventions
Ondansetron, Placebo
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years to 60 Years
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
2
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 3, 2024 · Synced Oct 23, 2026, 1:11 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Nuclear Magnetic Spectroscopy Imaging to Evaluate Primary Lateral Sclerosis, Hereditary Spastic Paraplegia and Amyotrophic Lateral Sclerosis

NCT00023075
View directory record Official record
Conditions
Primary Lateral Sclerosis, Hereditary Spastic Paraplegia, Amyotrophic Lateral Sclerosis
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
55 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2001 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 23, 2026, 1:11 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Phenotype, Genotype & Biomarkers in ALS and Related Disorders

NCT02327845
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, Primary Lateral Sclerosis, Hereditary Spastic Paraplegia, Progressive Muscular Atrophy, Multisystem Proteinopathy
Interventions
Not listed
Lead sponsor
University of Miami
Other
Eligibility
Not listed
Enrollment
708 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2031
U.S. locations
13
States / cities
Palo Alto, California • San Diego, California • San Francisco, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 15, 2026 · Synced Oct 23, 2026, 1:11 AM EDT
Completed Phase 1Phase 2 Interventional Results available

A First in Human Study of RT001 in Patients With Friedreich's Ataxia

NCT02445794
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Low dose cohort, High dose cohort
Drug
Lead sponsor
Biojiva LLC
Industry
Eligibility
18 Years to 50 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
2
States / cities
Long Beach, California • Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Nov 27, 2020 · Synced Oct 23, 2026, 1:11 AM EDT
Not listed Not applicable Interventional

PHysical Activity and Exercise Outcomes in Huntington's Disease

NCT03344601
View directory record Official record
Conditions
Huntington Disease
Interventions
physical activity
Behavioral
Lead sponsor
Cardiff University
Other
Eligibility
18 Years and older
Enrollment
116 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
2
States / cities
Los Angeles, California • New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 5, 2019 · Synced Oct 23, 2026, 1:11 AM EDT
Terminated Phase 3 Interventional Results available

Coenzyme Q10 in Huntington's Disease (HD)

NCT00608881
View directory record Official record
Conditions
Huntington's Disease
Interventions
coenzyme Q10, placebo
Drug · Other
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
16 Years and older
Enrollment
609 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
41
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Fayetteville, Arkansas + 37 more
Source: ClinicalTrials.gov public record
Updated Mar 30, 2016 · Synced Oct 23, 2026, 1:11 AM EDT
Completed No phase listed Observational

Ketogenic Diet in Lafora Disease

NCT00007124
View directory record Official record
Conditions
Lafora Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 23, 2026, 1:11 AM EDT
Completed Not applicable Interventional

Dissemination of Comprehensive Behavioral Intervention for Tics (CBIT) to Occupational Therapists: A Feasibility Study

NCT02114905
View directory record Official record
Conditions
Tourette Disorder, Chronic Tic Disorder
Interventions
OTs Trained and Deliver CBIT
Behavioral
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
7 Years to 17 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 20, 2019 · Synced Oct 23, 2026, 1:11 AM EDT
Recruiting Not applicable Interventional

CMT Gait, Mobility, Balance - AOFAS Grant

NCT07188415
View directory record Official record
Conditions
Charcot Marie Tooth Disease (CMT)
Interventions
Ankle-foot Orthoses
Device
Lead sponsor
Bopha Chrea
Other
Eligibility
25 Years to 55 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
North Liberty, Iowa
Source: ClinicalTrials.gov public record
Updated Sep 23, 2025 · Synced Oct 23, 2026, 1:11 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's Disease

NCT01427517
View directory record Official record
Conditions
Parkinson's Disease, Gaucher's Disease
Interventions
N-acetylcysteine
Drug
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2012
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Nov 1, 2019 · Synced Oct 23, 2026, 1:11 AM EDT
Completed Phase 1Phase 2 Interventional

Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies

NCT05364021
View directory record Official record
Conditions
Developmental and Epileptic Encephalopathy, Dravet Syndrome, Lennox Gastaut Syndrome
Interventions
LP352, Placebo
Drug
Lead sponsor
Longboard Pharmaceuticals
Industry
Eligibility
12 Years to 65 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
29
States / cities
Tucson, Arizona • Little Rock, Arkansas • Downey, California + 24 more
Source: ClinicalTrials.gov public record
Updated Nov 8, 2024 · Synced Oct 23, 2026, 1:11 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Prospective Huntington At Risk Observational Study

NCT00052143
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
Huntington Study Group
Network
Eligibility
26 Years to 55 Years
Enrollment
1,001 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1999 – 2009
U.S. locations
37
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 32 more
Source: ClinicalTrials.gov public record
Updated Sep 3, 2024 · Synced Oct 23, 2026, 1:11 AM 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 23, 2026, 1:11 AM EDT