Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Sep 26, 2026, 4:16 PM EDT

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Showing 1–24 of 211 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Conditions
Rett Syndrome
Interventions
NNZ-2566, Placebo
Drug
Lead sponsor
Neuren Pharmaceuticals Limited
Industry
Eligibility
5 Years to 15 Years · Female only
Enrollment
82 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
12
States / cities
Birmingham, Alabama • Oakland, California • San Diego, California + 9 more
Conditions
Friedreich Ataxia
Interventions
Buccal Swabs and Blood Draws
Diagnostic Test
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
20 participants
Timeline
2020
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Friedreich Ataxia
Interventions
Respiratory Strength Training (RST)
Other
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 65 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Gainesville, Florida
Conditions
Metastatic Cancer
Interventions
Palliative radiotherapy
Radiation
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
7
States / cities
Basking Ridge, New Jersey • Middletown, New Jersey • Montvale, New Jersey + 4 more
Completed Not applicable Interventional Results available

EMG Training for Altering Activation Patterns After Stroke

NCT03619772
Conditions
Stroke, Weakness, Muscle, Coordination Lack
Interventions
Bilateral, Unilateral
Other
Lead sponsor
Shirley Ryan AbilityLab
Other
Eligibility
18 Years to 80 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Chicago, Illinois
Conditions
Spinocerebellar Ataxia Type 3
Interventions
BIIB132, BIIB132-Matching Placebo
Drug
Lead sponsor
Biogen
Industry
Eligibility
18 Years to 70 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
10
States / cities
Los Angeles, California • San Francisco, California • Gainesville, Florida + 7 more
Conditions
Friedreich Ataxia
Interventions
Vatiquinone
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Not listed
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
5
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 2 more
Conditions
Phosphomannomutase 2 Deficiency, PMM2-CDG
Interventions
GLM101 (Part A, Double-blind), Placebo (Part A, Double-blind), GLM101 (Part B, Open-label)
Drug
Lead sponsor
Glycomine, Inc.
Industry
Eligibility
4 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
4
States / cities
Minneapolis, Minnesota • New York, New York • Philadelphia, Pennsylvania + 1 more
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Investigating the Genetic and Phenotypic Presentation of Ataxia and Nucleotide Repeat Diseases

NCT04529252
Conditions
Spinocerebellar Ataxias, Cerebellar Ataxia, Nucleotide Repeat Disease
Interventions
Specimen collection
Other
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2026
U.S. locations
1
States / cities
Jacksonville, Florida
Conditions
Progressive Myoclonus Epilepsy Type 1, EPM1, CSTB-related Disease, Myoclonus Epilepsies, Progressive, Unverricht-Lundborg Disease, Progressive Epilepsy and/or Ataxia With Myoclonus as a Major Feature, PME, Progressive Myoclonus-Epilepsies
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Not listed
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Healthy
Interventions
Silenor 6 mg, zolpidem 10 mg, Placebo
Drug
Lead sponsor
Currax Pharmaceuticals
Industry
Eligibility
21 Years to 50 Years · Male only
Enrollment
52 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2016
U.S. locations
1
States / cities
Novi, Michigan
Conditions
Friedreich Ataxia
Interventions
plethysmography
Diagnostic Test
Lead sponsor
University of Pittsburgh
Other
Eligibility
18 Years to 79 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Conditions
Hereditary Ataxia
Interventions
Not listed
Lead sponsor
Office of Rare Diseases (ORD)
NIH
Eligibility
3 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2009
U.S. locations
1
States / cities
Galveston, Texas
Active, not recruiting Phase 3 Interventional

Open Pilot Trial of BHV-4157

NCT03408080
Conditions
Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia 3, Spinocerebellar Ataxia Type 6, MSA-C
Interventions
BHV-4157
Drug
Lead sponsor
University of California, Los Angeles
Other
Eligibility
18 Years to 75 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
1
States / cities
Los Angeles, California
Conditions
Ataxia
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 100 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2014
U.S. locations
1
States / cities
New York, New York
Conditions
Advanced Solid Tumors
Interventions
Elimusertib (BAY1895344), Pembrolizumab (Keytruda®)
Drug
Lead sponsor
Bayer
Industry
Eligibility
18 Years and older
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
8
States / cities
Palo Alto, California • New Haven, Connecticut • Baltimore, Maryland + 5 more
Conditions
Friedreich Ataxia
Interventions
Vatiquinone
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Up to 6 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Spinocerebellar Ataxia
Interventions
Not listed
Lead sponsor
University of Mississippi Medical Center
Other
Eligibility
18 Years to 80 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
2
States / cities
Jackson, Mississippi • Galveston, Texas
Conditions
Ataxia Telangiectasia
Interventions
Baclofen, Placebo
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
12 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
2
States / cities
Baltimore, Maryland
Conditions
Epilepsy in Children, Dyskinesias, Movement Disorders in Children, Neurologic Disorder, Chorea, Myoclonus, Ataxia, Epilepsy, Dystonia Disorder, Movement Disorders
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
0 Years to 18 Years
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
1
States / cities
Boston, Massachusetts
Completed No phase listed Observational Accepts healthy volunteers

Instrumented Data Exchange for Ataxia Study

NCT04268147
Conditions
Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6, Friedreich Ataxia
Interventions
Not listed
Lead sponsor
University of Chicago
Other
Eligibility
12 Years to 75 Years
Enrollment
128 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2024
U.S. locations
5
States / cities
Los Angeles, California • Chicago, Illinois • Baltimore, Maryland + 2 more
Conditions
Friedreich Ataxia, Cardiomyopathies, Cardiac Hypertrophy, Myocardial Fibrosis
Interventions
AAVrh.10hFXN, serotype rh.10 adeno-associated virus (AAV) gene transfer vector expressing the cDNA coding for human FXN, Prednisone
Biological · Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
12 Years to 50 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
1
States / cities
New York, New York
Conditions
Friedreich Ataxia
Interventions
Idebenone
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
9 Years to 17 Years
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
1
States / cities
Bethesda, Maryland
Active, not recruiting No phase listed Observational Accepts healthy volunteers

FA Clinical Outcome Measures

NCT03090789
Conditions
Friedreich Ataxia, Neuro-Degenerative Disease
Interventions
Not listed
Lead sponsor
Friedreich's Ataxia Research Alliance
Other
Eligibility
4 Years to 80 Years
Enrollment
2,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2001 – 2030
U.S. locations
9
States / cities
Los Angeles, California • Denver, Colorado • Gainesville, Florida + 6 more