RecruitingPhase 2NCT06447025
Neurological
Friedreich Ataxia
Also known as FRDA, Friedreich's ataxia, frataxin deficiency
Friedreich ataxia is caused by a GAA trinucleotide repeat expansion in the FXN gene, reducing production of frataxin - a mitochondrial protein essential for iron regulation. Without adequate frataxin, iron accumulates in mitochondria, causi
ORPHA:95 ↗Gene FXN (GAA repeat expansion)Prevalence 1-9 per 100,000 (Orphanet)Onset Adolescent, ChildhoodGenetic (autosomal recessive)
19
studies recruiting now
as of 7 Sept 2026
112
studies registered in total
as of 7 Sept 2026
17
countries with a recruiting site
as of 7 Sept 2026
21 Aug 2026
most recent study posted
among recruiting studies
Recruiting trials
RecruitingNCT06865482
Clinical Course Of Disease In Participants With FA-CM
RecruitingPhase 2NCT07721025
Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
RecruitingNCT06016946
Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
RecruitingPhase 3NCT07778836
A Study to Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 19 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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About Friedreich Ataxia
Friedreich ataxia is caused by a GAA trinucleotide repeat expansion in the FXN gene, reducing production of frataxin - a mitochondrial protein essential for iron regulation. Without adequate frataxin, iron accumulates in mitochondria, causing oxidative damage to neurons and heart muscle. It presents in adolescence with progressive ataxia, cardiomyopathy, scoliosis, and diabetes. Omaveloxolone (Skyclarys) became the first approved treatment in 2023.
Common clinical features
Gait ataxiaGait imbalanceDysarthriaLimb ataxiaBabinski signHand muscle atrophyImpaired proprioceptionDysmetria
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
2 approved treatments and 21 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Approved: Idebenone (Raxone)Approved: Omaveloxolone (Skyclarys)
Phase 3Vatiquinone
Phase 3Pioglitazone
Phase 3Interferon Gamma-1b (Actimmune)
Phase 2/3Varenicline
Phase 2Luvadaxistat
Phase 2Vitamin E (Aquasol e)
Phase 2Resveratrol
Phase 2Epoetin Alfa (Abseamed)
+ 13 more in development
Before you apply
Things trial teams commonly ask about for Friedreich Ataxia. Not eligibility rules; those are set by each study.
- GAA repeat length in both alleles and current ambulatory status are standard eligibility criteria
- Cardiac function (LVEF) is a required safety screen in most Friedreich ataxia trials
- The mFARS (modified Friedreich Ataxia Rating Scale) score determines disease stage for enrollment
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).