Friedreich ataxia trials in United States

In United States 16 Also abroad 24

16 trials · From ClinicalTrials.gov, checked 11 November 2026. Ask the study team whether a trial fits you.

Phase 1Taking people
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Where
Columbus, Los Angeles, Philadelphia
Age
18 years to 40 years
People
10
Run by
Solid Biosciences Inc.
See details and how to joinNCT07180355
No phaseTaking people
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Where
Sioux Falls
People
20,000
Run by
Sanford Health
See details and how to joinNCT01793168
Phase 2Taking people
An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia
Where
Chevy Chase, Columbus, Eatontown, Gainesville, Iowa City, Los Angeles, Philadelphia, Salt Lake City, San Francisco, Tampa
Age
2 years to 60 years
People
85
Run by
Larimar Therapeutics, Inc.
See details and how to joinNCT06447025
No phaseTaking people
Clinical Course Of Disease In Participants With FA-CM
Where
Boston, Cincinnati, Indianapolis, La Jolla, Portland, Rochester, St Louis, Tampa
Age
6 years and over
People
65 worldwide
Run by
Lexeo Therapeutics
See details and how to joinNCT06865482
Phase 2Not yet taking people in United States
GV101 for Friedreich's Ataxia (FA) Treatment
Where
Eatontown
Age
16 years to 40 years
People
48
Run by
Graviton Bioscience Corporation
See details and how to joinNCT07870590
Phase 3Taking people
A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia
Where
Chicago, Dallas, Eatontown, Tampa
Age
12 years to 40 years
People
150 worldwide
Run by
Larimar Therapeutics, Inc.
See details and how to joinNCT07778836 · EU 2025-521628-31-00
No phaseTaking people
Biomarkers in Friedreich's Ataxia
Where
Gainesville
Age
6 years to 70 years
People
203
Run by
University of Florida
See details and how to joinNCT02497534
Phase 1Taking people
Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia
Where
New York
Age
12 years to 50 years
People
25
Run by
Weill Medical College of Cornell University
See details and how to joinNCT05302271
No phaseTaking people
Cardiac Output and Fatigue in Friedreich's Ataxia
Where
New York
People
30
Run by
Scott Barbuto
See details and how to joinNCT07444333
Phase 2Taking people
Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
Where
Tampa
Age
6 years and over
People
26
Run by
Lexeo Therapeutics
See details and how to joinNCT07721025 · EU 2025-524970-40-00
No phaseTaking people
Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
Where
Atlanta, Columbus, Denver, Gainesville, Iowa City, Los Angeles, Memphis, Philadelphia, Tampa
People
3,000 worldwide
Run by
Friedreich's Ataxia Research Alliance
See details and how to joinNCT06016946
Phase 3Not yet taking people in United States
Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
Where
Iowa City, Los Angeles, Philadelphia, Tampa
Age
7 years to 21 years
People
120 worldwide
Run by
PTC Therapeutics
See details and how to joinNCT07681713 · EU 2026-526947-37-00
No phaseTaking people
Characterization of the Cardiac Phenotype of Friedreich's Ataxia (FRDA)
Where
New York
Age
12 years to 50 years
People
100
Run by
Weill Medical College of Cornell University
See details and how to joinNCT02316314
No phaseTaking people
A Study to Learn How BIIB141 (Omaveloxolone) Affects the Health of Participants With Friedrich's Ataxia Who Took it During Pregnancy and/or During Breastfeeding and About the Health of Their Babies
Where
Morrisville
Age
16 years and over
People
10
Run by
Biogen
See details and how to joinNCT06628687
No phaseEnrolling by invitation
GABA and GSH in FRDA
Where
Philadelphia
Age
8 years to 16 years
People
60
Run by
Children's Hospital of Philadelphia
See details and how to joinNCT07635030
No phaseTaking people
A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors
Where
Aurora, Gainesville, Los Angeles, Philadelphia
Age
16 years and over
People
300 worldwide
Run by
Biogen
See details and how to joinNCT06623890