Friedreich ataxia: trials for referral
25 open trials in the registries we read. Each row links to its registry record.
Registry data only. Frontlight does not judge whether a patient can take part: the trial team decides.
Where the open trials are
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25 trials shown
Phase 13 sites
RecruitingUnited States
NCT07180355
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Solid Biosciences Inc.
Registry updated 6 Nov 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingItaly
NCT07095062
Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.
IRCCS Eugenio Medea
Registry updated 5 Nov 2026
Registry record: ClinicalTrials.govPhase 210 sites
RecruitingUnited States
NCT06447025
An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia
Larimar Therapeutics, Inc.
Registry updated 27 Oct 2026
Registry record: ClinicalTrials.govPhase 21 site
Not yet recruitingUnited States
NCT07870590
GV101 for Friedreich's Ataxia (FA) Treatment
Graviton Bioscience Corporation
Registry updated 15 Oct 2026
Registry record: ClinicalTrials.govPhase 35 sites
RecruitingFrance, United States
NCT07778836
A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia
Larimar Therapeutics, Inc.
Registry updated 9 Oct 2026
Registry record: ClinicalTrials.govPhase 11 site
RecruitingUnited States
NCT05302271
Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia
Weill Medical College of Cornell University
Registry updated 28 Sept 2026
Registry record: ClinicalTrials.govPhase 21 site
RecruitingUnited States
NCT07721025
Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
Lexeo Therapeutics
Registry updated 2 Sept 2026
Registry record: ClinicalTrials.govPhase 39 sites
Not yet recruitingBelgium, Brazil, Canada, Spain, France, United States
NCT07681713
Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)
PTC Therapeutics
Registry updated 14 Aug 2026
Registry record: ClinicalTrials.govNo phase1 site
By invitation onlyGermany
NCT07072676
The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.
LMU Klinikum
Registry updated 18 Jul 2025
Registry record: ClinicalTrials.govPhase 1/23 sites
RecruitingAustralia
NCT06874010
A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia
Design Therapeutics, Inc.
Registry updated 18 May 2025
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT01793168
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Sanford Health
Registry updated 28 Oct 2026
Registry record: ClinicalTrials.govNo phase0 sites
Not yet recruiting
NCT07887828
A Study to Learn More About Friedrich's Ataxia in People Living in the Gulf Cooperation Council (GCC) Region of the World
Biogen
Registry updated 21 Oct 2026
Registry record: ClinicalTrials.govNo phase19 sites
RecruitingBrazil, Canada, Czechia, Germany, Spain, France
NCT06865482
Clinical Course Of Disease In Participants With FA-CM
Lexeo Therapeutics
Registry updated 19 Oct 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT02497534
Biomarkers in Friedreich's Ataxia
University of Florida
Registry updated 6 Oct 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT07444333
Cardiac Output and Fatigue in Friedreich's Ataxia
Scott Barbuto
Registry updated 22 Sept 2026
Registry record: ClinicalTrials.govNo phase34 sites
RecruitingAustria, Australia, Belgium, Brazil, Canada, Czechia
NCT06016946
Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
Friedreich's Ataxia Research Alliance
Registry updated 1 Sept 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT02316314
Characterization of the Cardiac Phenotype of Friedreich's Ataxia (FRDA)
Weill Medical College of Cornell University
Registry updated 29 Jul 2026
Registry record: ClinicalTrials.govNo phase1 site
Not yet recruitingUnited Kingdom
NCT07731971
Digital Outcome Assessment Using AI Active Gaming and Motion Capture in Friedreich Ataxia
University of Exeter
Registry updated 28 Jul 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT06628687
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
Biogen
Registry updated 10 Jul 2026
Registry record: ClinicalTrials.govNo phase1 site
By invitation onlyUnited States
NCT07635030
GABA and GSH in FRDA
Children's Hospital of Philadelphia
Registry updated 9 Jun 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingFrance
NCT07013292
Efficacy of Omaveloxolone Treatment for Dysphagia in French Patients With Friedreich's Ataxia
Centre Hospitalier Universitaire de Nice
Registry updated 29 Apr 2026
Registry record: ClinicalTrials.govNo phase14 sites
RecruitingAustria, Belgium, Czechia, Germany, Italy, Netherlands
NCT06623890
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
Biogen
Registry updated 28 Apr 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingFrance
NCT07508631
Friedreich Ataxia Nerve Ultrasund
Centre Hospitalier Universitaire de Nice
Registry updated 2 Apr 2026
Registry record: ClinicalTrials.govNo phase6 sites
RecruitingAustria, Germany, France
NCT05943002
Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia
German Center for Neurodegenerative Diseases (DZNE)
Registry updated 1 Apr 2026
Registry record: ClinicalTrials.govNo phase1 site
By invitation onlyGermany
NCT06605612
Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders
LMU Klinikum
Registry updated 10 Sept 2025
Registry record: ClinicalTrials.gov
The same sites as a list
- United States16 open trials
- France7 open trials
- Germany6 open trials
- Italy4 open trials
- Spain3 open trials
- Brazil3 open trials
- Canada3 open trials
- Belgium3 open trials
- Czechia3 open trials
- Austria3 open trials
- Australia2 open trials
- United Kingdom2 open trials
- Netherlands2 open trials
- Ireland1 open trial
- New Zealand1 open trial
- India1 open trial
- Greece1 open trial
Who treats this
Institutions with trials or publications in this disease (OpenAlex, registries). Institutions only, no people.
The University of Texas Southwestern Medical Center
Dallas, United States
1 trial · 12 publications · ROR
What changed in the last 7 days
Registry facts, one per line, newest first.
Closed for recruitment
Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
NCT06692296 · No phase · Italy
Source: ClinicalTrials.govClosed for recruitment
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
NCT05515536 · Phase 3 · Australia, Brazil, Canada, Germany, Spain, France
Source: ClinicalTrials.govOpened for recruitment
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
NCT05515536 · Phase 3 · Australia, Brazil, Canada, Germany, Spain, France
Source: ClinicalTrials.govStatus changed: Recruiting → Not yet recruiting
NCT07778836 · Phase 3 · France, United States
Source: ClinicalTrials.gov
Results card
Trials that finished or stopped in the last 3 years, with the reason the registry gives and linked publications.
No phaseEnded 14 Jan 2026
Phase 2Ended Dec 2025
Withdrawn before start0 participants
NCT03761511
Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich Ataxia
Reason given: Due to a temporary halt the recruitment start was delayed. A re-evaluation of the sponsor revealed, that the study is no longer feasible due to increased administrative and financial requirements.
Phase 2Ended 19 Dec 2025
Completed172 participants
NCT02255435
A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia
No phaseEnded 1 Nov 2025
No phaseEnded 31 Oct 2025
Phase 1Ended 22 Oct 2025
Withdrawn before start0 participants
NCT06483802
A Study of ASP2016 in Adults Who Have Heart Disease Associated With Friedreich Ataxia
Reason given: Corporate decision to withdraw the trial at this time.
No phaseEnded 7 Aug 2025
Phase 1Ended 27 Apr 2025
Stopped early18 participants
NCT06681766
A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia
Reason given: Review of Cohort 1 data facilitated direct enrollment into open label study and eliminate pause in study drug administration between PK study and open label study.
No phaseEnded Dec 2024
Completed1200 participants
NCT02069509
Patient Registry of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS)
No phaseEnded 1 Dec 2024
Completed120 participants
NCT04346238
Characterization of the Interruptions of the GAA Expansion and Study of Their Influence on the Severity of Friedreich's Ataxia
Phase 2Ended 29 Aug 2024
Completed5 participants
NCT05485987
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
Phase 1/2Ended 25 Jul 2024
No phaseEnded 30 Jun 2024
Phase 1/2Ended 17 Apr 2024
Phase 2Ended 28 Mar 2024
Phase 2Ended 4 Dec 2023
Completed28 participants
NCT05579691
A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia
Not medical advice. fetched 10 Nov 2026