Recruiting
Who can join AIPeople with Friedreich's ataxia.
What is being studied AITests daily subcutaneous nomlabofusp versus placebo on neurological function.
Study typeTreatment (drug) · Phase 3
CountriesUnited States
Friedreich Ataxia
Browse recruiting and upcoming clinical trials for Friedreich ataxia.
Patient-friendly, AI-assisted summaries based on official ClinicalTrials.gov ↗ records. Last updated: Oct 10, 2026
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ClinicalTrials.gov is the complete and most up-to-date source. Trials may be missing, and trial information may have changed since our last check. Always verify important information in the official registry.
Recruiting
Who can join AIPeople aged ≥6 with genetically confirmed Friedreich ataxia and secondary cardiomyopathy (left ventricular hypertrophy).
What is being studied AITests LX2006 gene therapy versus usual care in Friedreich ataxia cardiomyopathy.
Study typeTreatment (drug) · Phase 2
CountriesUnited States
Recruiting
Who can join AIPeople with genetically confirmed Friedreich's ataxia who can safely ride a stationary bicycle.
What is being studied AITests omaveloxolone plus home aerobic exercise effects on VO2max and fatigue.
Study typeTreatment (other)
CountriesUnited States
Recruiting
Who can join AIAdults 18–40 years with a clinical and genetic diagnosis of Friedreich's ataxia (symptom onset ≤25 years) meeting study staging requirements.
What is being studied AITests the safety and tolerability of SGT-212 gene therapy given by IDN and IV.
Study typeTreatment (drug) · Phase 1
CountriesUnited States
Recruiting
Who can join AIAdults 18–65 with genetically confirmed Friedreich's ataxia (homozygous GAA repeat expansions).
What is being studied AITests safety, tolerability, and pharmacokinetics of multiple ascending doses of DT-216P2.
Study typeTreatment (drug) · Phase 1/2
CountriesAustralia
Recruiting
Who can join AIPeople aged ≥14 with a genetic diagnosis of Friedreich's Ataxia who can stand unsupported and walk at least 10 metres.
What is being studied AITests stabilometric platform plus conventional rehabilitation effects on standing balance in Friedreich's Ataxia.
Study typeTreatment (other)
CountriesItaly
Recruiting
Who can join AIPeople aged 2–60 with Friedreich Ataxia (FRDA) who meet the study's medical criteria.
What is being studied AILong-term safety, PK/PD, frataxin levels, and clinical effects of subcutaneous CTI-1601.
Study typeTreatment (drug) · Phase 2
CountriesUnited States
Recruiting
Who can join AIPeople aged 12–50 with Friedreich's ataxia and evidence of FA-related cardiomyopathy (cardiac hypertrophy). The registry allows myocardial fibrosis of no more than 10% of the left ventricular wall.
What is being studied AITests safety and preliminary effects of IV AAVrh.10hFXN for FA cardiomyopathy.
Study typeTreatment (drug) · Phase 1
CountriesUnited States
Not yet recruiting
Who can join AIHealthy adult volunteers aged 18-60. The drug is being developed for Friedreich ataxia, but people with the condition are not eligible for this first safety study.
What is being studied AITests safety, tolerability, and how ATI-105 is processed in the body.
Study typeTreatment (drug) · Phase 1
CountriesUnited States
Not yet recruiting
Who can join AIPeople aged 7 to 21 with genetically confirmed Friedreich's ataxia who meet specified clinical and mobility scores.
What is being studied AITests vatiquinone's effects on disease measures and safety over 24 months.
Study typeTreatment (drug) · Phase 3
CountriesBelgium, Brazil, Canada +3
Not yet recruiting
Who can join AIHealthy adults 18–45 years (note: the registry lists Friedreich Ataxia among conditions).
What is being studied AISafety, tolerability and pharmacokinetics of single ascending doses of DT-216P2 (SC and IV).
Study typeTreatment (drug) · Phase 1
CountriesAustralia
Enrolling by invitation
Who can join AIPeople aged 65 years with diagnosed neuromuscular diseases (for example: inclusion body myositis, myotonic dystrophy, myasthenia gravis, SMA, Guillain‑Barré).
What is being studied AITests whether a week of adapted assistive gait devices plus training changes fall-risk and walking measures.
Study typeTreatment (procedure/device)
CountriesGermany
Recruiting
Who can join AIPeople aged 8–60 with genetically confirmed Friedreich's Ataxia and a SARA score below 30.
What is being studied AIUses HD-EEG and neuropsychological tests to study brain connectivity and rehabilitation effects.
Study typeOther
CountriesItaly
Recruiting
Friedreich Ataxia Nerve Ultrasund
NCT07508631
Who can join AIAdults 18–70 with genetically confirmed Friedreich's Ataxia who had a peripheral nerve ultrasound between Dec 2025 and Apr 2026 and are followed at the Neurogenetics Competence Centre.
What is being studied AIUses nerve ultrasound to assess intraneural blood flow, nerve size, and internal nerve structure.
Study typeObservational
CountriesFrance
Recruiting
Who can join AIPeople aged ≥16 with genetically confirmed Friedreich's ataxia treated with omaveloxolone for at least 6 months.
What is being studied AICollects six-month swallowing questionnaire scores from Friedreich Ataxia patients treated with omaveloxolone.
Study typeObservational
CountriesFrance
Recruiting
Who can join AIPeople aged 6 years and older with genetically confirmed Friedreich ataxia and cardiomyopathy (left ventricular hypertrophy) with left ventricular ejection fraction ≥30%.
What is being studied AICollects heart imaging and clinical data to describe cardiac disease course in FA-CM.
Study typeObservational
CountriesBrazil, Canada, Czechia +5
Who can join AIFemales aged 16 years or older with Friedreich Ataxia who were exposed to omaveloxolone during pregnancy and/or while breastfeeding.
What is being studied AICollects health data after omaveloxolone (BIIB141/SKYCLARYS) exposure during pregnancy or breastfeeding.
Study typeObservational
CountriesUnited States
Recruiting
Who can join AIPeople aged 12 years and older with genetically confirmed Friedreich Ataxia and SARA score ≤30 who can use a smartphone or tablet.
What is being studied AICollects patient-reported, psychosocial, economic outcomes and mobile‑app usability data in Friedreich Ataxia.
Study typeObservational
CountriesAustria, France, Germany
Recruiting
Biomarkers in Friedreich's Ataxia
NCT02497534
Who can join AIPeople with Friedreich's ataxia, healthy control volunteers, and carriers of the Friedreich's ataxia gene.
What is being studied AICollects cardiac, exercise, neuromuscular measurements and optional tissue samples in Friedreich's ataxia.
Study typeObservational
CountriesUnited States
Recruiting
Who can join AIPeople with Friedreich's ataxia aged 12–50, and matched healthy controls aged 12–30.
What is being studied AICardiac imaging, exercise testing, blood markers and corneal confocal microscopy as disease biomarkers.
Study typeObservational
CountriesUnited States
Not yet recruiting
Who can join AIPeople with Friedreich ataxia and healthy volunteers.
What is being studied AITests whether AI-driven active gaming and markerless motion capture can measure movement in FA.
Study typeObservational
CountriesUnited Kingdom
Enrolling by invitation
GABA and GSH in FRDA
NCT07635030
Who can join AIChildren aged 8 up to (but not including) 16 years with Friedreich's ataxia (FRDA), and neurotypical (control) children.
What is being studied AIMRS measurements of brain GABA and glutathione (GSH) as biomarkers in FRDA.
Study typeObservational
CountriesUnited States
Enrolling by invitation
Who can join AIPeople with one of the neuromuscular disorders listed in the registry (see full list).
What is being studied AIDevelops and validates the FBIndex using timed gait and muscle-function tests to assess fall risk.
Study typeObservational
CountriesGermany
Recruiting
Who can join AIPeople 16 years and older with genetically confirmed Friedreich's Ataxia enrolled in the FA‑GCC UNIFAI who are starting or recently started omaveloxolone.
What is being studied AICollects long-term safety data on omaveloxolone in people with Friedreich's Ataxia.
Study typeRegistry
CountriesAustria, Belgium, Czechia +4
Recruiting
Who can join AIPeople of any age with genetically confirmed Friedreich ataxia, and healthy control participants.
What is being studied AICollects long-term clinical, functional, and patient-reported data on Friedreich ataxia.
Study typeRegistry
CountriesAustralia, Austria, Belgium +14
Recruiting
Who can join AIPeople with rare or undiagnosed conditions, and unaffected carriers of rare or uncommon diseases.
What is being studied AICollects patients' contact, demographic, and health information for a centralized rare-disease registry.
Study typeRegistry
CountriesAustralia, United States