Search | Clinical Trials Finder

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Nov 14, 2026, 9:23 PM EST

All trial information on this site comes from the official ClinicalTrials.gov public API (clinicaltrials.gov/api/v2), run by the U.S. National Library of Medicine. We only display that public information; we do not add opinions, endorsements, or any other intent. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 1,153 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Neurodegenerative Disease, Hereditary Clear all

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

Conditions: Juvenile Neuronal Ceroid Lipofuscinosis
Interventions: Mycophenolate mofetil, Liquid Placebo; Drug
Lead sponsor: University of Rochester; Other
Eligibility: 6 Years to 25 Years
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2015
U.S. locations: 1
States / cities: Rochester, New York

Conditions: Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6, Spinocerebellar Ataxia Type 7, Spinocerebellar Ataxia Type 8, Spinocerebellar Ataxia Type 10, RFC1 Gene Mutation, Spinocerebellar Ataxia Type 27b, Healthy Participants
Interventions: Genetic Testing, Blood Collection, Magnetic Resonance Imaging (MRI) Scan, Assessments and Questionnaires, Cerebrospinal Fluid Collection; Genetic · Other
Lead sponsor: Lauren Moore; Other
Eligibility: 6 Years and older
Enrollment: 1,400 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2010 – 2030
U.S. locations: 16
States / cities: Los Angeles, California • San Francisco, California • Gainesville, Florida + 12 more

Conditions: Neurodegenerative Diseases, Leukodystrophy, Ataxia, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, Cerebellar Ataxia, Adrenomyeloneuropathy
Interventions: Home exercise; Behavioral
Lead sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.; Other
Eligibility: 5 Years to 75 Years
Enrollment: 30 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2028
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Neurofibroma, Plexiform, Neurofibromatosis Type I
Interventions: tipifarnib, placebo; Drug · Other
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 3 Years to 25 Years
Enrollment: 62 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2009
U.S. locations: 11
States / cities: Birmingham, Alabama • Los Angeles, California • Chicago, Illinois + 8 more

Conditions: Cardiac Amyloidosis
Interventions: AI ECG Amyloid algorithm; Other
Lead sponsor: Mayo Clinic; Other
Eligibility: 18 Years and older
Enrollment: 20,143 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2025
U.S. locations: 4
States / cities: Scottsdale, Arizona • Jacksonville, Florida • Rochester, Minnesota + 1 more

Conditions: PIK3CA-Related Overgrowth Spectrum (PROS)
Interventions: alpelisib; Other
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 2 Years and older
Enrollment: 57 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2021
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Inherited Cardiac Arrythmias, Long QT Syndrome (LQTS), Brugada Syndrome (BrS), Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT), Early Repolarization Syndrome (ERS), Arrhythmogenic Cardiomyopathy (AC, ARVD/C), Hypertrophic Cardiomyopathy (HCM), Dilated Cardiomyopathy (DCM), Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Normal Control Subjects
Interventions: Not listed
Lead sponsor: Johns Hopkins University; Other
Eligibility: 18 Years to 85 Years
Enrollment: 100 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2013 – 2031
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Tourette Syndrome
Interventions: Ecopipam, Placebo; Drug
Lead sponsor: Emalex Biosciences Inc.; Industry
Eligibility: 6 Years to 17 Years
Enrollment: 153 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2021
U.S. locations: 53
States / cities: Dothan, Alabama • Phoenix, Arizona • Anaheim, California + 45 more

Conditions: Complex Partial Seizures
Interventions: vigabatrin; Drug
Lead sponsor: University of Pennsylvania; Other
Eligibility: 18 Years and older
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2013
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Tourette Syndrome in Children, Tourette Syndrome in Adolescence
Interventions: T92, Placebo; Dietary Supplement
Lead sponsor: Tasly Pharmaceuticals, Inc.; Industry
Eligibility: 6 Years to 17 Years
Enrollment: 150 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2024
U.S. locations: 1
States / cities: New Haven, Connecticut

Conditions: Advanced Solid Tumors
Interventions: PX-866; Drug
Lead sponsor: Cascadian Therapeutics Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 90 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2011
U.S. locations: 2
States / cities: Aurora, Colorado • Houston, Texas

Conditions: Tourette Syndrome, Tic Disorders
Interventions: Not listed
Lead sponsor: National Institute of Neurological Disorders and Stroke (NINDS); NIH
Eligibility: 21 Years to 65 Years
Enrollment: 54 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2003 – 2009
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Myotonic Dystrophy Type 2, Myotonic Dystrophy Type 1
Interventions: Non-interventional study; Other
Lead sponsor: Wake Forest University Health Sciences; Other
Eligibility: 30 Years to 65 Years
Enrollment: 100 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2027
U.S. locations: 1
States / cities: Winston-Salem, North Carolina

Conditions: Neurofibromatosis Type 1, Neurofibroma
Interventions: Arm 1; Other
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 5 Years to 120 Years
Enrollment: 110 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 1
States / cities: Bethesda, Maryland

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

Conditions: Tuberous Sclerosis, Neurofibromatoses, Angiofibroma, Neurofibroma
Interventions: Skincerity, Skincerity plus sirolimus/rapamycin, Skinercity plus sirolimus/rapamycin; Drug
Lead sponsor: The University of Texas Health Science Center, Houston; Other
Eligibility: 13 Years and older
Enrollment: 52 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2009 – 2011
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Recurrent Glioblastoma Multiforme, Primary Glioblastoma Multiforme, Brain Neoplasms, Malignant, Leptomeningeal Disease (LMD)
Interventions: Radiation Therapy, AZD1390; Radiation · Drug
Lead sponsor: AstraZeneca; Industry
Eligibility: 18 Years to 130 Years
Enrollment: 159 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2026
U.S. locations: 5
States / cities: Boston, Massachusetts • New York, New York • Pittsburgh, Pennsylvania + 1 more

Conditions: Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome, Duchenne Muscular Dystrophy, Phelan-McDermid Syndrome, Rett Syndrome, Smith Magenis Syndrome, Williams Syndrome, Turner Syndrome, Klinefelter Syndrome, Chromosome 22q11.2 Deletion Syndrome, Tuberous Sclerosis, Down Syndrome
Interventions: Parent-Infant Inter(X)action Intervention (PIXI); Behavioral
Lead sponsor: RTI International; Other
Eligibility: Up to 99 Years
Enrollment: 120 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2026
U.S. locations: 1
States / cities: Research Triangle Park, North Carolina

Conditions: Friedreich Ataxia
Interventions: Elamipretide; Drug
Lead sponsor: Children's Hospital of Philadelphia; Other
Eligibility: 16 Years and older
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2024
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Friedreich's Ataxia
Interventions: GV101, Placebo; Drug
Lead sponsor: Graviton Bioscience Corporation; Industry
Eligibility: 16 Years to 40 Years
Enrollment: 48 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2027
U.S. locations: 1
States / cities: Eatontown, New Jersey

Conditions: Wilson's Disease
Interventions: TETA 4HCl formulation, D-Penicillamine; Drug
Lead sponsor: Orphalan; Industry
Eligibility: 8 Years and older
Enrollment: 38 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 3
States / cities: Denver, Colorado • New Haven, Connecticut • Ann Arbor, Michigan

Conditions: Charcot-Marie-Tooth Disease Type 1A
Interventions: PXT3003 dose 1, PXT3003 dose 2, placebo; Drug
Lead sponsor: Pharnext S.C.A.; Other
Eligibility: 16 Years to 65 Years
Enrollment: 323 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 11
States / cities: Los Angeles, California • New Britain, Connecticut • Gainesville, Florida + 8 more

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

Search by objective public record fields.

Innovative Ultrasound Technology in Neuromuscular Disease

Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis

Natural History Study of and Genetic Modifiers in Spinocerebellar Ataxias

Home Exercise for Individuals With Neurodegenerative Disease

R115777 to Treat Children With Neurofibromatosis Type 1 and Progressive Plexiform Neurofibromas

Artificial Intelligence Enhanced ECG to Detect Cardiac Amyloidosis

Retrospective Chart Review Study of Patients With PIK3CA-Related Overgrowth Spectrum Who Have Received Alpelisib

Derivation of Human Induced Pluripotent Stem (iPS) Cells to Heritable Cardiac Arrhythmias

Ecopipam Tablets to Study Tourette's Syndrome in Children and Adolescents

Sabril for Complex Partial Seizures in Adult Tolerability Study (TS) Patients

A Clinical Study to Evaluate the Safety and Efficacy of T92 in Pediatric Patients With Tourette Syndrome

Phase I Trial of Oral PX-866

Brain Dynamics Involved in Generating Tics and Controlling Voluntary Movement

Brain Structure and Clinical Endpoints in Myotonic Dystrophy Type 2

Development of Patient-Reported Outcome Measures Assessing Tumor Visibility and Appearance Concerns in Neurofibromatosis Type 1: A Qualitative Study

Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry

Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)

A Study to Assess the Safety and Tolerability of AZD1390 Given With Radiation Therapy in Patients With Brain Cancer

Parent and Infant Inter(X)Action Intervention (PIXI)

FRDA Investigator Initiated Study (IIS) With Elamipretide

GV101 for Friedreich's Ataxia (FA) Treatment

Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.

Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT)

Investigating Changes in Premonitory Urges During Habit Reversal Training for Tics