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Showing 1–24 of 68 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Friedreich Ataxia Clear all
Completed Phase 2 Interventional

Safety and Efficacy Study of A0001 in Subjects With Friedreich's Ataxia

NCT01035671
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
alpha-tocopherolquinone (A0001), placebo
Drug
Lead sponsor
Penwest Pharmaceuticals Co.
Industry
Eligibility
18 Years to 60 Years
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 21, 2011 · Synced Sep 15, 2026, 2:45 PM 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 Sep 15, 2026, 2:45 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 15, 2026, 2:45 PM EDT
Completed Phase 2 Interventional Results available

Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia

NCT03214588
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
TAK-831, TAK-831 Placebo
Drug
Lead sponsor
Neurocrine Biosciences
Industry
Eligibility
18 Years to 55 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
6
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 13, 2021 · Synced Sep 15, 2026, 2:45 PM EDT
Completed Phase 3 Interventional Results available

A Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia

NCT04102501
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
RT001, Placebo
Drug
Lead sponsor
Biojiva LLC
Industry
Eligibility
12 Years to 50 Years
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
6
States / cities
Long Beach, California • Los Angeles, California • Gainesville, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated May 26, 2022 · Synced Sep 15, 2026, 2:45 PM EDT
Completed No phase listed Observational

A Study to Assess Variation in Potential Biomarkers in Friedreich Ataxia

NCT04255680
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 17, 2020 · Synced Sep 15, 2026, 2:45 PM EDT
Completed Phase 1 Interventional

Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia

NCT05573698
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
DT-216, Placebo
Drug
Lead sponsor
Design Therapeutics, Inc.
Industry
Eligibility
18 Years to 55 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 28, 2024 · Synced Sep 15, 2026, 2:45 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

TCA Cycle in the Dentate in Friedreich's Ataxia

NCT03122925
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
MR
Device
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2023
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 26, 2023 · Synced Sep 15, 2026, 2:45 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 15, 2026, 2:45 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study

NCT06016946
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Not listed
Lead sponsor
Friedreich's Ataxia Research Alliance
Other
Eligibility
Not listed
Enrollment
3,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2048
U.S. locations
9
States / cities
Los Angeles, California • Denver, Colorado • Gainesville, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Sep 15, 2026, 2:45 PM EDT
Completed Phase 2 Interventional

EPI-743 in Friedreich's Ataxia Point Mutations

NCT01962363
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
EPI-743
Drug
Lead sponsor
University of South Florida
Other
Eligibility
18 Years to 65 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jun 28, 2016 · Synced Sep 15, 2026, 2:45 PM EDT
Terminated Phase 2Phase 3 Interventional

Pilot Study of Varenicline (Chantix®) in the Treatment of Friedreich's Ataxia

NCT00803868
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
varenicline, placebo
Drug
Lead sponsor
University of South Florida
Other
Eligibility
18 Years to 80 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
2
States / cities
Tampa, Florida • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 23, 2012 · Synced Sep 15, 2026, 2:45 PM EDT
Completed Phase 3 Interventional Results available

Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients

NCT00697073
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Idebenone
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
8 Years to 18 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
2
States / cities
Los Angeles, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 10, 2023 · Synced Sep 15, 2026, 2:45 PM 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 Sep 15, 2026, 2:45 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Determine Range of Tissue Frataxin Concentrations and Other Potential Biomarkers

NCT05028764
View directory record Official record
Conditions
Friedreich Ataxia, Healthy Volunteers
Interventions
Buccal Swabs, Blood Draws and Skin Punch Biopsy
Diagnostic Test
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 23, 2022 · Synced Sep 15, 2026, 2:45 PM EDT
Recruiting Phase 1 Interventional

A Study of SGT-212 Gene Therapy in Friedreich's Ataxia

NCT07180355
View directory record Official record
Conditions
Friedreich's Ataxia (FA)
Interventions
SGT-212
Drug
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
18 Years to 40 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
3
States / cities
Los Angeles, California • Columbus, Ohio • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 16, 2026 · Synced Sep 15, 2026, 2:45 PM EDT
Completed Not applicable Interventional Results available

NAD+ and Exercise in FA

NCT04192136
View directory record Official record
Conditions
Friedreich Ataxia 1
Interventions
Nicotinamide Riboside, Placebo, Exercise Intervention
Dietary Supplement · Other
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
10 Years to 40 Years
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 10, 2026 · Synced Sep 15, 2026, 2:45 PM EDT
Completed No phase listed Observational

Measurement of Frataxin mRNA in Biofluids

NCT06496451
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Spinal tap for CSF collection, Venipuncture for blood sample
Other
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jan 15, 2026 · Synced Sep 15, 2026, 2:45 PM 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 Sep 15, 2026, 2:45 PM EDT
Terminated Phase 1 Interventional

A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia

NCT06681766
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Nomlabofusp, Placebo
Drug
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
2 Years to 17 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Chevy Chase, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 9, 2026 · Synced Sep 15, 2026, 2:45 PM EDT
Completed Phase 2 Interventional Results available

A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia

NCT02255435
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg, Placebo, Omaveloxolone Capsules, 20 mg, Omaveloxolone Capsules, 40 mg, Omaveloxolone Capsules, 80 mg, Omaveloxolone Capsules, 160 mg, Omaveloxolone Capsules, 300 mg, Omaveloxolone Capsules, 150 mg
Drug
Lead sponsor
Biogen
Industry
Eligibility
16 Years to 40 Years
Enrollment
172 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2025
U.S. locations
7
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Sep 15, 2026, 2:45 PM EDT
Completed Phase 2 Interventional Results available

Interferon Gamma-1b in Friedreich Ataxia (FRDA)

NCT01965327
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Interferon Gamma-1b
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
5 Years to 17 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 12, 2021 · Synced Sep 15, 2026, 2:45 PM EDT
Completed Phase 3 Interventional Results available

Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia

NCT02415127
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Interferon γ-1b, Placebo
Drug
Lead sponsor
Amgen
Industry
Eligibility
10 Years to 25 Years
Enrollment
92 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
4
States / cities
Los Angeles, California • Gainesville, Florida • Iowa City, Iowa + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 18, 2024 · Synced Sep 15, 2026, 2:45 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Instrumented Data Exchange for Ataxia Study

NCT04268147
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 4, 2024 · Synced Sep 15, 2026, 2:45 PM EDT