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ClinicalTrials.gov public records Last synced Oct 22, 2026, 4:33 PM EDT

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Showing 841–864 of 1,148 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Completed Phase 2 Interventional

Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients

NCT05479981
View directory record Official record
Conditions
DM1, Muscular Dystrophies, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Disorders, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, Neurodegenerative Diseases
Interventions
Placebo, AOC 1001
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
18 Years to 66 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
8
States / cities
Los Angeles, California • Palo Alto, California • Denver, Colorado + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2026 · Synced Oct 22, 2026, 4:33 PM EDT
Terminated No phase listed Observational

Hearing and Balance Disorders in Peripheral Neuropathy

NCT05827419
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease
Interventions
Not listed
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
5 Years to 21 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Wilmington, Delaware
Source: ClinicalTrials.gov public record
Updated Apr 25, 2023 · Synced Oct 22, 2026, 4:33 PM EDT
Completed Phase 2Phase 3 Interventional Results available

The Effect of Diflunisal on Familial Amyloidosis

NCT00294671
View directory record Official record
Conditions
Familial Amyloid Polyneuropathy, Familial Amyloidosis
Interventions
diflunisal, placebo
Drug · Other
Lead sponsor
Boston University
Other
Eligibility
18 Years to 75 Years
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
3
States / cities
Boston, Massachusetts • Rochester, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 17, 2017 · Synced Oct 22, 2026, 4:33 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Brain Activation in Vocal and Motor Tics

NCT00026000
View directory record Official record
Conditions
Tourette's Syndrome, Chronic Motor Tic Disorder, Chronic Motor Vocal Disorder
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2001 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 22, 2026, 4:33 PM EDT
Completed Not applicable Interventional

Proprioception and Sensorimotor Control in Hereditary Sensory and Autonomic Neuropathy

NCT02876939
View directory record Official record
Conditions
Atunomic Neuropathy
Interventions
kinesiology tape around the hip, knee and ankle joints, No kinesiology tape around the hip, knee and ankle joints
Other
Lead sponsor
NYU Langone Health
Other
Eligibility
16 Years to 40 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 22, 2020 · Synced Oct 22, 2026, 4:33 PM EDT
Not listed Phase 3 Interventional

Vitamin E in Aging Persons With Down Syndrome

NCT00056329
View directory record Official record
Conditions
Down Syndrome, Alzheimer Disease
Interventions
Vitamin E, multivitamin, Placebo
Drug
Lead sponsor
New York State Institute for Basic Research
Government
Eligibility
50 Years and older
Enrollment
350 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2012
U.S. locations
14
States / cities
Farmington, Connecticut • Tampa, Florida • Atlanta, Georgia + 10 more
Source: ClinicalTrials.gov public record
Updated May 4, 2012 · Synced Oct 22, 2026, 4:33 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

RASopathy Biorepository

NCT04395495
View directory record Official record
Conditions
RAS Mutation, Neurofibromatosis 1, Noonan Syndrome, Noonan Syndrome With Multiple Lentigines, Noonan Neurofibromatosis Syndrome, Cardiofaciocutaneous Syndrome, Costello Syndrome, Legius Syndrome, Smith-Kingsmore Syndrome, MTOR Gene Mutation, GATOR-1 Gene Mutation, SYNGAP1-Related Intellectual Disability, DLG4, MAPK1 Gene Mutation
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2065
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 18, 2025 · Synced Oct 22, 2026, 4:33 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Child to Adult Neurodevelopment in Gene Expanded Huntington's Disease

NCT01860339
View directory record Official record
Conditions
Huntington's Disease
Interventions
Not listed
Lead sponsor
Peggy C Nopoulos
Other
Eligibility
6 Years to 30 Years
Enrollment
488 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2026
U.S. locations
6
States / cities
Sacramento, California • Iowa City, Iowa • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 30, 2026 · Synced Oct 22, 2026, 4:33 PM EDT
Active, not recruiting No phase listed Observational

Multi-parametric Biomarker Development to Predict Malignant Conversion in Patients With Neurofibromatosis Type 1

NCT05677594
View directory record Official record
Conditions
Neurofibromatosis 1, Malignant Peripheral Nerve Sheath Tumor (MPNST) of Soft Tissue (Diagnosis), Atypical Neurofibroma
Interventions
Whole Body Magnetic Resonance Imaging
Diagnostic Test
Lead sponsor
Johns Hopkins University
Other
Eligibility
6 Years and older
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced Oct 22, 2026, 4:33 PM EDT
Terminated Phase 2 Interventional Results available

SF1126 in Recurrent or Progressive SCCHN and Mutations in PIK3CA Gene and/or PI-3 Kinase Pathway Genes

NCT02644122
View directory record Official record
Conditions
Metastatic Squamous Neck Cancer With Occult Primary Squamous Cell Carcinoma
Interventions
SF1126
Drug
Lead sponsor
Ezra Cohen
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Jul 11, 2018 · Synced Oct 22, 2026, 4:33 PM EDT
Terminated Phase 3 Interventional Results available

Study of Safety and Efficacy of Brolucizumab 6 mg Dosed Every 4 Weeks Compared to Aflibercept 2 mg Dosed Every 4 Weeks in Patients With Retinal Fluid Despite Frequent Anti-VEGF Injections

NCT03710564
View directory record Official record
Conditions
Age-Related Macular Degeneration
Interventions
Brolucizumab, Aflibercept
Biological
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
50 Years and older
Enrollment
535 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
60
States / cities
Phoenix, Arizona • Beverly Hills, California • Huntington Beach, California + 51 more
Source: ClinicalTrials.gov public record
Updated Jan 30, 2023 · Synced Oct 22, 2026, 4:33 PM EDT
Terminated Phase 3 Interventional Results available

Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN Participants

NCT03041116
View directory record Official record
Conditions
Pantothenate Kinase-Associated Neurodegeneration
Interventions
Fosmetpantotenate, Placebo
Drug
Lead sponsor
Travere Therapeutics, Inc.
Industry
Eligibility
6 Years to 65 Years
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
9
States / cities
Irvine, California • Washington D.C., District of Columbia • Miami, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Jan 26, 2021 · Synced Oct 22, 2026, 4:33 PM EDT
Recruiting Not applicable Interventional

Cardiac Output and Fatigue in Friedreich's Ataxia

NCT07444333
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Aerobic Exercise
Behavioral
Lead sponsor
Scott Barbuto
Other
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 22, 2026, 4:33 PM EDT
Terminated Early Phase 1 Interventional Results available

Exploratory Evaluation of AR-42 Histone Deacetylase Inhibitor in the Treatment of Vestibular Schwannoma and Meningioma

NCT02282917
View directory record Official record
Conditions
Vestibular Schwannoma, Meningioma, Acoustic Neuroma, Neurofibromatosis Type 2
Interventions
AR-42
Drug
Lead sponsor
Massachusetts Eye and Ear Infirmary
Other
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
4
States / cities
Stanford, California • Baltimore, Maryland • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 5, 2025 · Synced Oct 22, 2026, 4:33 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Cortical Excitability in Obsessive-Compulsive and Related Disorders

NCT00029068
View directory record Official record
Conditions
Obsessive Compulsive Disorder, Healthy
Interventions
Not listed
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
Not listed
Enrollment
145 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2002 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 22, 2026, 4:33 PM EDT
Completed Not applicable Interventional

A Decentralized Clinical Trial to Promote Evidence-Based Care for Underserved Patients With Neurofibromatosis 1

NCT06262113
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
Letters about NF1 Care (Content Type 1), Letters about NF1 Care (Content Type 2)
Other
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
Not listed
Enrollment
294 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 21, 2026 · Synced Oct 22, 2026, 4:33 PM EDT
Completed Phase 3 Interventional Results available

Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy

NCT01994889
View directory record Official record
Conditions
Transthyretin (TTR) Amyloid Cardiomyopathy
Interventions
Tafamidis, Placebo
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 90 Years
Enrollment
441 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
50
States / cities
Birmingham, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 24 more
Source: ClinicalTrials.gov public record
Updated Apr 24, 2019 · Synced Oct 22, 2026, 4:33 PM EDT
Recruiting Not applicable Interventional

Neuromodulation to Enhance Motor Function in HSP

NCT07417943
View directory record Official record
Conditions
Hereditary Spastic Paraplegia
Interventions
transcutaneous spinal cord stimulation
Device
Lead sponsor
Rahul Sachdeva
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Lexington, Kentucky
Source: ClinicalTrials.gov public record
Updated Apr 22, 2026 · Synced Oct 22, 2026, 4:33 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Innovative Ultrasound Technology in Neuromuscular Disease

NCT02104921
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Muscular Dystrophy, Radiculopathy, Myopathy, Polyneuropathy and Mononeuropathies, Trauma Injury, Orthopedic Disorder
Interventions
Not listed
Lead sponsor
Beth Israel Deaconess Medical Center
Other
Eligibility
18 Years to 90 Years
Enrollment
223 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 11, 2022 · Synced Oct 22, 2026, 4:33 PM EDT
Recruiting Not applicable Interventional

Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)

NCT07521930
View directory record Official record
Conditions
Tetraplegia/Tetraparesis, Amyotrophic Lateral Sclerosis (ALS), Muscular Disorders, Atrophic, Brain Stem Stroke, Spinal Cord Injuries (SCI), Muscular Dystrophies, Muscular Atrophy, Progressive, Pontine Hemorrhage, Pontine Infarction
Interventions
INTENT Neural Interface System
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 80 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2027 – 2028
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 9, 2026 · Synced Oct 22, 2026, 4:33 PM EDT
Completed Phase 2Phase 3 Interventional

Cognitive Behavior Therapy and Habit Reversal Training for the Treatment of Chronic Tic Disorders in Children

NCT00218777
View directory record Official record
Conditions
Tourette Syndrome, Tic Disorders
Interventions
Comprehensive Behavioral Intervention for Tics
Behavioral
Lead sponsor
Tourette Association of America
Other
Eligibility
9 Years to 17 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2007
U.S. locations
3
States / cities
Los Angeles, California • Baltimore, Maryland • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Dec 14, 2015 · Synced Oct 22, 2026, 4:33 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy

NCT01737398
View directory record Official record
Conditions
FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis
Interventions
Inotersen, Placebo
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 82 Years
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
9
States / cities
Orange, California • Indianapolis, Indiana • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 17, 2019 · Synced Oct 22, 2026, 4:33 PM EDT
Completed Not applicable Interventional

TicHelper: A Computerized Comprehensive Behavioral Intervention for Tics (CBIT)

NCT02413216
View directory record Official record
Conditions
Tourette's Disorder, Persistent (Chronic) Motor or Vocal Tic Disorder
Interventions
TicHelper, Internet Based Resources
Behavioral · Other
Lead sponsor
University of Utah
Other
Eligibility
8 Years to 18 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
2
States / cities
Salt Lake City, Utah • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Dec 18, 2017 · Synced Oct 22, 2026, 4:33 PM EDT
Active, not recruiting No phase listed Observational

Spinraza in Adult Spinal Muscular Atrophy

NCT03709784
View directory record Official record
Conditions
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type 3
Interventions
Observational study to examine safety, tolerability, and effectiveness of SPINRAZA® prescribed as part of standard of care, One time survey
Drug · Other
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years to 70 Years
Enrollment
148 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
10
States / cities
Phoenix, Arizona • Washington D.C., District of Columbia • Baltimore, Maryland + 7 more
Source: ClinicalTrials.gov public record
Updated Nov 20, 2024 · Synced Oct 22, 2026, 4:33 PM EDT