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Showing 1–24 of 1,140 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Inherited Neurodegenerative Disorders Clear all
Completed Phase 2 Interventional Results available

A Study of Pridopidine (ACR16) for the Treatment of Participants With Huntington's Disease

NCT00724048
View directory record Official record
Conditions
Huntington Disease
Interventions
ACR16, Placebo
Drug · Other
Lead sponsor
Teva Branded Pharmaceutical Products R&D, Inc.
Industry
Eligibility
30 Years and older
Enrollment
227 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
22
States / cities
Birmingham, Alabama • San Diego, California • Littleton, Colorado + 18 more
Source: ClinicalTrials.gov public record
Updated Aug 30, 2023 · Synced Sep 12, 2026, 1:56 PM EDT
Completed No phase listed Observational Results available

Neurology Measures in FA Children

NCT03418740
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
2 Years to 18 Years
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
3
States / cities
Los Angeles, California • Gainesville, Florida • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 15, 2025 · Synced Sep 12, 2026, 1:56 PM EDT
Completed Phase 1 Interventional Results available

Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia

NCT01898884
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
VP 20629, Placebo
Drug
Lead sponsor
Shire
Industry
Eligibility
18 Years to 45 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
5
States / cities
Los Angeles, California • Tampa, Florida • Atlanta, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 1, 2021 · Synced Sep 12, 2026, 1:56 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Resiliency Training in Adolescents With NF1 and NF2

NCT03873610
View directory record Official record
Conditions
Neurofibromatoses
Interventions
Stress and Symptom Management Program 1, Stress and Symptom Management Program 2
Behavioral
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
12 Years to 17 Years
Enrollment
196 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 10, 2026 · Synced Sep 12, 2026, 1:56 PM EDT
Active, not recruiting No phase listed Observational

Natural History of Spinocerebellar Ataxia Type 7 (SCA7)

NCT02741440
View directory record Official record
Conditions
Spinocerebellar Ataxia
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
12 Years to 100 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2028
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 27, 2026 · Synced Sep 12, 2026, 1:56 PM EDT
Active, not recruiting Not applicable Interventional

Modification of Threat Interpretation Bias to Reduce Anxiety in Neurodegenerative Movement Disorders (Aim 3)

NCT07213648
View directory record Official record
Conditions
Huntington Disease, Parkinson Disease
Interventions
MindTrails-Movement, Waitlist Control
Behavioral · Other
Lead sponsor
University of Virginia
Other
Eligibility
21 Years and older
Enrollment
136 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Charlottesville, Virginia
Source: ClinicalTrials.gov public record
Updated Jul 19, 2026 · Synced Sep 12, 2026, 1:56 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Resiliency Training for Adolescent Neurofibromatosis Patients Via Videoconferencing With Skype

NCT02387177
View directory record Official record
Conditions
Neurofibromatoses
Interventions
Stress Management Group 1, Stress Management Group 2
Behavioral
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
12 Years to 17 Years
Enrollment
51 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2017
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 16, 2018 · Synced Sep 12, 2026, 1:56 PM EDT
Recruiting No phase listed Observational

Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)

NCT06222203
View directory record Official record
Conditions
Neurofibromatosis 1, Nerve Sheath Neoplasms
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 120 Years
Enrollment
225 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2035
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Sep 12, 2026, 1:56 PM EDT
Active, not recruiting No phase listed Observational

Cerliponase Alfa Observational Study in the US

NCT04476862
View directory record Official record
Conditions
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
Interventions
Cerliponase Alfa, Administration Kit
Drug · Device
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
16
States / cities
Little Rock, Arkansas • Orange, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Sep 12, 2026, 1:56 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Analysis of Lumbar Spine Stenosis Specimens for Identification of Transthyretin Cardiac Amyloidosis

NCT06034405
View directory record Official record
Conditions
Lumbar Spinal Stenosis, Cardiac Amyloidosis, ATTR Amyloidosis Wild Type, ATTR Gene Mutation, ATTRV122I Amyloidosis, Cardiomyopathy, Hypertrophic
Interventions
Tc99-PYP or Tc99-HDMP Scan
Diagnostic Test
Lead sponsor
Columbia University
Other
Eligibility
50 Years and older
Enrollment
1,663 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2028
U.S. locations
6
States / cities
Beverly Hills, California • Boston, Massachusetts • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Sep 12, 2026, 1:56 PM EDT
Completed Not applicable Interventional Results available

MN4000 for Treatment of CF and MND Patients in the Home Setting

NCT03797677
View directory record Official record
Conditions
Cystic Fibrosis, Motor Neuron Disease, Airway Clearance Impairment
Interventions
MN 4000
Device
Lead sponsor
Hill-Rom
Industry
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Aug 6, 2024 · Synced Sep 12, 2026, 1:56 PM EDT
Recruiting No phase listed Observational

Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

NCT03981575
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Sep 12, 2026, 1:56 PM EDT
Completed Phase 2 Interventional Results available

(+) Epicatechin to Treat Friedreich's Ataxia

NCT02660112
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
(+)-Epicatechin
Drug
Lead sponsor
Ralitza Gavrilova
Other
Eligibility
10 Years to 50 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 4, 2019 · Synced Sep 12, 2026, 1:56 PM EDT
Completed Phase 2 Interventional Results available

A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy

NCT02693119
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy
Interventions
elamipretide (MTP-131) 1% topical ophthalmic solution, Vehicle topical ophthalmic solution
Drug
Lead sponsor
Stealth BioTherapeutics Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Pasadena, California
Source: ClinicalTrials.gov public record
Updated Nov 29, 2021 · Synced Sep 12, 2026, 1:56 PM EDT
Completed Not applicable Interventional Results available

Open-label MNS for Tourette Syndrome

NCT05016765
View directory record Official record
Conditions
Tourette Syndrome, Tic Disorder, Chronic Motor or Vocal
Interventions
Active, self-directed electrical stimulation of the median nerve
Device
Lead sponsor
Washington University School of Medicine
Other
Eligibility
15 Years to 65 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 4, 2023 · Synced Sep 12, 2026, 1:56 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Children's Health Research Institute(CHRI), Stanford Lucile Packard Children Hospital (LPCH) Protocol on Myotonic Dystrophy

NCT02269865
View directory record Official record
Conditions
Myotonic Dystrophy Type 1
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
8 Years to 17 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2016
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Oct 20, 2014 · Synced Sep 12, 2026, 1:56 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

PatientSpot Formerly Known as ArthritisPower

NCT03840928
View directory record Official record
Conditions
Rheumatoid Arthritis, Ankylosing Spondylitis, Fibromyalgia, Gout, Crohn Disease, Juvenile Idiopathic Arthritis, Lupus Erythematosus, Myositis, Osteoarthritis, Osteoporosis, Psoriasis, Psoriatic Arthritis, Scleroderma, Dermatomyositis, Inflammatory Bowel Diseases, Polymyositis, Axial Spondyloarthritis, Diffuse Idiopathic Skeletal Hyperostosis, Polymyalgia Rheumatica, Giant Cell Arteritis, Temporal Arteritis, Wegener, Relapsing Polychondritis, Undifferentiated Connective Tissue Disease, Spinal Cord Injuries, Alzheimer Disease, Amyotrophic Lateral Sclerosis, Ataxia, Bell Palsy, Brain Tumor, Cerebral Aneurysm, Epilepsy, Guillain-Barre Syndrome, Headache, Head Injury, Hydrocephalus, Lumbar Disc Disease, Meningitis, Multiple Sclerosis, Muscular Dystrophy, Neurocutaneous Syndromes, Parkinson Disease, Stroke, Cluster Headache, Tension-Type Headache, Chronic Obstructive Pulmonary Disease, Asthma, Lung Cancer, Cystic Fibrosis, Sleep Apnea, Eczema, Alopecia, Chronic Inflammation, Unstable Angina, Heart Attack, Heart Failure, Arrythmia, Valve Heart Disease, High Blood Pressure, Congenital Heart Disease, Peripheral Arterial Disease, Diabetes, Chronic Liver Disease, Obesity
Interventions
Not listed
Lead sponsor
Global Healthy Living Foundation
Other
Eligibility
19 Years and older
Enrollment
40,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2025
U.S. locations
1
States / cities
Upper Nyack, New York
Source: ClinicalTrials.gov public record
Updated Dec 12, 2023 · Synced Sep 12, 2026, 1:56 PM EDT
Recruiting No phase listed Observational

Natural History Study of Patients with HPDL Mutations

NCT05848271
View directory record Official record
Conditions
Mitochondrial Encephalomyopathies, Hereditary Spastic Paraplegia, Spastic Paraplegia, White Matter Disease, Neonatal Encephalopathy, Mutation, Genetic Disease
Interventions
Patient Registry, Dry blood spots sampling
Other
Lead sponsor
University of California, San Diego
Other
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Mar 29, 2025 · Synced Sep 12, 2026, 1:56 PM EDT
Completed Phase 1 Interventional

Phase 1 Trial of Idebenone to Treat Patients With Friedreich's Ataxia

NCT00078481
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Idebenone
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 12, 2026, 1:56 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Biomarkers in Friedreich's Ataxia

NCT02497534
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Not listed
Lead sponsor
University of Florida
Other
Eligibility
6 Years to 70 Years
Enrollment
203 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2030
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Sep 22, 2025 · Synced Sep 12, 2026, 1:56 PM EDT
Withdrawn Not applicable Interventional

Use of a Tonometer to Identify Epileptogenic Lesions During Pediatric Epilepsy Surgery

NCT04344626
View directory record Official record
Conditions
Epilepsy, Focal Cortical Dysplasia, Tuberous Sclerosis, Hemimegalencephaly, Polymicrogyria, Rasmussen Encephalitis, Sturge-Weber Syndrome, Gliosis, Stroke, Tumor, Brain
Interventions
Intra-operative brain tonometry
Device
Lead sponsor
University of California, Los Angeles
Other
Eligibility
Not listed
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Oct 9, 2023 · Synced Sep 12, 2026, 1:56 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

A Phase Ib/II Study of BYL719 and Cetuximab in Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma

NCT01602315
View directory record Official record
Conditions
Recurrent Head and Neck Squamous Cell Carcinoma, Metastatic Head and Neck Squamous Cell Carcinoma
Interventions
BYL719 as film-coated (FC) whole tablets, BYL719 as dispersible tablets (DT), cetuximab, BYL719 drink suspension
Drug · Biological
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
179 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
14
States / cities
Sacramento, California • San Francisco, California • Aurora, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Dec 28, 2020 · Synced Sep 12, 2026, 1:56 PM EDT
Completed Phase 1Phase 2 Interventional Results available

FRDA Investigator Initiated Study (IIS) With Elamipretide

NCT05168774
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 11, 2025 · Synced Sep 12, 2026, 1:56 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Androgen Receptor, Implications for Health and Wellbeing: Natural History Study of Individuals With Androgen Insensitivity

NCT04708431
View directory record Official record
Conditions
Androgen Insensitivity Syndrome, Metabolic Parameters in AIS, CAIS, PAIS and MAIS, Tumor Formation in AIS, CAIS, PAIS and MAIS, Sexual Function AIS, CAIS, PAIS and MAIS
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
8 Years to 99 Years
Enrollment
650 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2040
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 12, 2026 · Synced Sep 12, 2026, 1:56 PM EDT