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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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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 2, 2026, 4:27 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 2, 2026, 4:27 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 Jan 22, 2026 · Synced Sep 2, 2026, 4:27 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 2, 2026, 4:27 PM EDT
Completed No phase listed Observational

Genetic and Environmental Determinants That Control Metabolism in Pulmonary Hypertension

NCT02594917
View directory record Official record
Conditions
Iron-sulfur Cluster Deficiency, Pulmonary Hypertension, Friedreich Ataxia
Interventions
Right Heart Catheterization, Blood draw
Procedure · Other
Lead sponsor
University of Pittsburgh
Other
Eligibility
18 Years to 60 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2023
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 21, 2023 · Synced Sep 2, 2026, 4:27 PM EDT
Completed Phase 1 Interventional

Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia

NCT05285540
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
DT-216, DT-216 matching Placebo
Drug
Lead sponsor
Design Therapeutics, Inc.
Industry
Eligibility
18 Years to 55 Years
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
1
States / cities
Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Mar 27, 2023 · Synced Sep 2, 2026, 4:27 PM EDT
Completed Not applicable Interventional

Coronary Artery Disease in Patients With Friedreich's Ataxia

NCT04649866
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 7, 2023 · Synced Sep 2, 2026, 4:27 PM 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 Sep 2, 2026, 4:27 PM 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 Sep 2, 2026, 4:27 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 2, 2026, 4:27 PM EDT
Not yet recruiting Phase 3 Interventional

Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)

NCT07681713
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Vatiquinone
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
7 Years to 21 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
4
States / cities
Los Angeles, California • Tampa, Florida • Iowa City, Iowa + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2026 · Synced Sep 2, 2026, 4:27 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 2, 2026, 4:27 PM EDT
Completed Phase 2 Interventional

Idebenone to Treat Friedreich's Ataxia

NCT00229632
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 17, 2019 · Synced Sep 2, 2026, 4:27 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

NCT05445323
View directory record Official record
Conditions
Friedreich Ataxia, Cardiomyopathy, Secondary
Interventions
Low dose LX2006, Mid Dose LX2006, High Dose LX2006
Genetic
Lead sponsor
Lexeo Therapeutics
Industry
Eligibility
18 Years to 50 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
3
States / cities
Los Angeles, California • Tampa, Florida • Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 22, 2025 · Synced Sep 2, 2026, 4:27 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 2, 2026, 4:27 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 2, 2026, 4:27 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 2, 2026, 4:27 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 2, 2026, 4:27 PM EDT
Completed Phase 3 Interventional Results available

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

NCT00537680
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Idebenone, Placebo
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
8 Years to 17 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
2
States / cities
Los Angeles, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 10, 2023 · Synced Sep 2, 2026, 4:27 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 2, 2026, 4:27 PM EDT
Completed Phase 3 Interventional Results available

Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia

NCT02797080
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
interferon γ-1b
Drug
Lead sponsor
Amgen
Industry
Eligibility
11 Years to 27 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
4
States / cities
Los Angeles, California • Gainesville, Florida • Iowa City, Iowa + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 9, 2024 · Synced Sep 2, 2026, 4:27 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 2, 2026, 4:27 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

A Natural History Study to TRACK Brain and Spinal Cord Changes in Individuals with Friedreich Ataxia (TRACK-FA)

NCT04349514
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Natural history
Other
Lead sponsor
Monash University
Other
Eligibility
5 Years and older
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2025
U.S. locations
3
States / cities
Gainesville, Florida • Minneapolis, Minnesota • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 26, 2024 · Synced Sep 2, 2026, 4:27 PM EDT
Completed Phase 2 Interventional

A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia

NCT05579691
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
CTI-1601, Placebo
Biological · Other
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
28 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 Nov 28, 2024 · Synced Sep 2, 2026, 4:27 PM EDT