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Showing 1–24 of 209 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Ataxia Clear all
Terminated Phase 2 Interventional Results available

N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)

NCT03759678
View directory record Official record
Conditions
Ataxia Telangiectasia
Interventions
IB1001
Drug
Lead sponsor
IntraBio Inc
Industry
Eligibility
6 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced Jul 21, 2026, 11:23 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Identification of Biomarkers in Spinocerebellar Ataxia 3

NCT03885167
View directory record Official record
Conditions
Spinocerebellar Ataxia Type 3
Interventions
Specimen Collection
Other
Lead sponsor
University of Michigan
Other
Eligibility
18 Years and older
Enrollment
22 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2023
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Nov 21, 2023 · Synced Jul 21, 2026, 11:23 AM EDT
Recruiting Phase 2 Interventional

An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia

NCT06447025
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
CTI-1601
Drug
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
2 Years to 60 Years
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
8
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced Jul 21, 2026, 11:23 AM EDT
Completed Phase 2 Interventional

Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia

NCT01728064
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Placebo, EPI-743 400 mg, EPI-743 200 mg
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
18 Years to 45 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
3
States / cities
Los Angeles, California • Tampa, Florida • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 30, 2020 · Synced Jul 21, 2026, 11:23 AM 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 Jul 21, 2026, 11:23 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Early and Longitudinal Assessment of Neurodegeneration in the Brain and Spinal Cord in Friedreich's Ataxia

NCT01776164
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
10 Years and older
Enrollment
85 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2022
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 8, 2023 · Synced Jul 21, 2026, 11:23 AM EDT
Completed Phase 2 Interventional

A Safety Study of NNZ-2566 in Patients With Rett Syndrome

NCT01703533
View directory record Official record
Conditions
Rett Syndrome
Interventions
NNZ-2566, Placebo
Drug
Lead sponsor
Neuren Pharmaceuticals Limited
Industry
Eligibility
16 Years to 45 Years · Female only
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
3
States / cities
Birmingham, Alabama • Saint Paul, Minnesota • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 4, 2018 · Synced Jul 21, 2026, 11:23 AM EDT
Completed Phase 3 Interventional Results available

Cyclophosphamide and Prednisone With or Without Immunoglobulin in Treating Abnormal Muscle Movement in Children With Neuroblastoma

NCT00033293
View directory record Official record
Conditions
Localized Resectable Neuroblastoma, Localized Unresectable Neuroblastoma, Regional Neuroblastoma, Stage 4 Neuroblastoma, Stage 4S Neuroblastoma
Interventions
Clinical Observation, Cyclophosphamide, Laboratory Biomarker Analysis, Magnetic Resonance Imaging, Prednisone, Therapeutic Immune Globulin
Other · Drug · Procedure + 1 more
Lead sponsor
Children's Oncology Group
Network
Eligibility
Up to 8 Years
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2022
U.S. locations
97
States / cities
Birmingham, Alabama • Tucson, Arizona • Little Rock, Arkansas + 78 more
Source: ClinicalTrials.gov public record
Updated Apr 17, 2023 · Synced Jul 21, 2026, 11:23 AM EDT
Recruiting Phase 1 Interventional

Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia

NCT05302271
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 30, 2026 · Synced Jul 21, 2026, 11:23 AM EDT
Completed Early Phase 1 Interventional

Rosuvastatin (Crestor) in Friedreich Ataxia

NCT02705547
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Rosuvastatin
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 24, 2021 · Synced Jul 21, 2026, 11:23 AM EDT
Terminated Phase 1 Interventional

A Pharmacokinetics and Safety Study of BIIB132 in Adults With Spinocerebellar Ataxia 3

NCT05160558
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 3, 2023 · Synced Jul 21, 2026, 11:23 AM EDT
Completed Phase 1 Interventional

Study to Test How Well Patients With Advanced Solid Tumors Respond to Treatment With the Elimusertib in Combination With Pembrolizumab, to Find the Optimal Dose for Patients, How the Drug is Tolerated and the Way the Body Absorbs, Distributes and Discharges the Drug

NCT04095273
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 1, 2024 · Synced Jul 21, 2026, 11:23 AM EDT
Recruiting No phase listed Observational

Clinical Course Of Disease In Participants With FA-CM

NCT06865482
View directory record Official record
Conditions
Friedreich Ataxia, Cardiomyopathy
Interventions
Not listed
Lead sponsor
Lexeo Therapeutics
Industry
Eligibility
6 Years and older
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
8
States / cities
La Jolla, California • Tampa, Florida • Indianapolis, Indiana + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 7, 2026 · Synced Jul 21, 2026, 11:23 AM EDT
Withdrawn Phase 1 Interventional

AMES Treatment of the Proximal Arm in Chronic Stroke

NCT01934439
View directory record Official record
Conditions
Stroke, Cerebrovascular Accident
Interventions
30 PAAD treatments
Device
Lead sponsor
Oregon Health and Science University
Other
Eligibility
18 Years to 75 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Beaverton, Oregon
Source: ClinicalTrials.gov public record
Updated Mar 28, 2019 · Synced Jul 21, 2026, 11:23 AM EDT
Active, not recruiting Not applicable Interventional

Home Based Tele-exercise for People With Chronic Neurological Impairments

NCT04564495
View directory record Official record
Conditions
Neurologic Disorder, Stroke, Spinal Cord Injuries, Cerebral Palsy, Hemiplegia, Hemiparesis, Multiple Sclerosis, Parkinson Disease, Trauma, Brain, Transverse Myelitis, Friedreich Ataxia
Interventions
Seated exercise
Behavioral
Lead sponsor
Burke Medical Research Institute
Other
Eligibility
18 Years to 75 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
White Plains, New York
Source: ClinicalTrials.gov public record
Updated Feb 4, 2025 · Synced Jul 21, 2026, 11:23 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

FA Clinical Outcome Measures

NCT03090789
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 3, 2024 · Synced Jul 21, 2026, 11:23 AM 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 Jul 21, 2026, 11:23 AM EDT
Active, not recruiting Phase 3 Interventional

A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

NCT05515536
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 29, 2025 · Synced Jul 21, 2026, 11:23 AM EDT
Completed Phase 1 Interventional

Coenzyme Q10 in Adult-Onset Ataxia

NCT00957216
View directory record Official record
Conditions
Sporadic Ataxia
Interventions
Placebo (sugar pill), Coenzyme Q10
Drug
Lead sponsor
The University of Texas Medical Branch, Galveston
Other
Eligibility
18 Years to 80 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 10, 2018 · Synced Jul 21, 2026, 11:23 AM EDT
No Longer Available No phase listed Expanded access

EPI-743 for Mitochondrial Respiratory Chain Diseases

NCT01370447
View directory record Official record
Conditions
Mitochondrial Diseases
Interventions
EPI-743
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
1 Year and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
16
States / cities
Los Angeles, California • Orange, California • Palo Alto, California + 12 more
Source: ClinicalTrials.gov public record
Updated Jan 22, 2024 · Synced Jul 21, 2026, 11:23 AM EDT
Completed Phase 2 Interventional Results available

Treatment of Fragile-X Associated Tremor/Ataxia Syndrome (FXTAS) With Allopregnanolone

NCT02603926
View directory record Official record
Conditions
Fragile X-associated Tremor/Ataxia Syndrome
Interventions
Allopregnanolone
Drug
Lead sponsor
Randi J. Hagerman, MD
Other
Eligibility
50 Years to 85 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Dec 5, 2018 · Synced Jul 21, 2026, 11:23 AM EDT
Completed No phase listed Observational

Clinical and Molecular Correlations in Spinocerebellar Ataxia Type 10 (SCA10)

NCT00004306
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 5, 2012 · Synced Jul 21, 2026, 11:23 AM EDT
Completed Phase 3 Interventional Accepts healthy volunteers

Diaphragmatic Breathing and Heart Rate Variability Training for Improving Hypertension in Fragile X Associated Tremor/Ataxia

NCT03816540
View directory record Official record
Conditions
Hypertension, FXTAS
Interventions
HRV and respiratory coherence biofeedback
Device
Lead sponsor
University of California, Davis
Other
Eligibility
50 Years to 90 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Oct 21, 2019 · Synced Jul 21, 2026, 11:23 AM EDT
Completed Phase 2Phase 3 Interventional Results available

A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

NCT04577352
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Vatiquinone, Placebo
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
7 Years and older
Enrollment
146 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
5
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 12, 2026 · Synced Jul 21, 2026, 11:23 AM EDT