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2026 data Public-data reference. official source

Friedreich Ataxia

Open-data reference.

15 US clinical trials · 7 currently recruiting

Active & Recent Trials

RECRUITING 3,000 participants

Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study

Friedreich's Ataxia Research Alliance

NCT06016946

RECRUITING 300 participants

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

NCT06623890

RECRUITING Phase 3 255 participants

A Study to Learn More About the Effects and Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old (BRAVE)

Biogen

NCT06953583

RECRUITING 65 participants

Clinical Course Of Disease In Participants With FA-CM

Lexeo Therapeutics

NCT06865482

RECRUITING Phase 1 35 participants

A Study to Find Out How BIIB141 (Omaveloxolone) is Processed in the Body and to Learn More About Its Safety in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old

Biogen

NCT06054893

RECRUITING Phase 1 25 participants

Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia

Weill Medical College of Cornell University

NCT05302271

RECRUITING 20 participants

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

NCT06628687

ACTIVE NOT RECRUITING 2,000 participants

FA Clinical Outcome Measures

Friedreich's Ataxia Research Alliance

NCT03090789

ACTIVE NOT RECRUITING 300 participants

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

Monash University

NCT04349514

ACTIVE NOT RECRUITING Phase 3 130 participants

A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

PTC Therapeutics

NCT05515536

ACTIVE NOT RECRUITING Phase 1 8 participants

Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

Lexeo Therapeutics

NCT05445323

COMPLETED 85 participants

Early and Longitudinal Assessment of Neurodegeneration in the Brain and Spinal Cord in Friedreich's Ataxia

University of Minnesota

NCT01776164

COMPLETED Phase 1 32 participants

Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia

Design Therapeutics

NCT05573698

COMPLETED Phase 2 28 participants

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

Larimar Therapeutics

NCT05579691

COMPLETED 10 participants

Measurement of Frataxin mRNA in Biofluids

University of Florida

NCT06496451

Phase Distribution

PhaseTrial count
Early Phase 1 4
Phase 2 1
Phase 3 2

Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.

Reading the Friedreich Ataxia Trial Landscape

ClinicalTrials.gov lists 15 US studies indexed under Friedreich Ataxia, and 7 of those are currently open to recruitment — roughly 47% of the total volume on the registry. That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.

The phase distribution for Friedreich Ataxia shows 2 late-stage studies (Phase 3 and Phase 4 combined) alongside 5 earlier-phase entries (Phase 1 through Phase 2). Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.

Top sponsor activity for Friedreich Ataxia is led by Biogen with 4 indexed trials, alongside 9 other organizations in the top contributor list. The list on this page surfaces up to 15 of the most relevant recent and active entries, ordered with recruiting studies first so practical options are visible. All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation — eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.

Frequently Asked Questions

How many clinical trials are there for Friedreich Ataxia?

PlainTrial tracks 15 US clinical trials for Friedreich Ataxia, of which 7 are currently recruiting participants. Data sourced from ClinicalTrials.gov.

How do I find a recruiting trial for Friedreich Ataxia?

Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.

Is this data current?

Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.

Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2024 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.

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