A Phase 3 study of Omaveloxolone in participants with Friedreich’s Ataxia Ages 2 < 16 years

• Research type

  Research Study

• Full title

  A Phase 3, 2-Part, Randomised, Double-Blind, Placebo-Controlled Study (Part 1) and Open-Label Extension (Part 2) to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Omaveloxolone (BIIB141) in Participants With Friedreich’s Ataxia Aged 2 to < 16 Years.

• IRAS ID

  1012042

• Contact name

  Van Phan

• Contact email

  clinicaltrials@biogen.com

• Sponsor organisation

  Biogen Idec Research Limited

• Clinicaltrials.gov Identifier

  NCT06953583

• Research summary

  This phase 3 study evaluates the safety, effectiveness, and how the body processes the drug Omaveloxolone (BIIB141) in children aged 2 to under 16 years with Friedreich’s ataxia (FA). FA is a rare genetic disease that affects the nervous system and muscles, causing problems with movement, coordination, and sometimes the heart. It is caused by a lack of a protein called frataxin, which is essential for nerve and muscle health. FA usually begins in childhood or adolescence and worsens over time. Omaveloxolone is already approved for use in children with FA aged 16 and older. It works by improving how cells function and reducing damage caused by the lack of frataxin, which may help slow the disease and reduce symptoms.

  This is a 2-part study testing the approved dose of BIIB141 in children under 16. The goal is to see if BIIB141 helps restore muscle function. In Part 1 (Randomised Controlled Trial), participants will take either BIIB141 or placebo as a capsule for 52 weeks, with regular clinic visits. On non-visit days, they will continue taking the assigned capsule. Participants who complete Part 1 can choose to join Part 2 (Open-label Extension), where all will receive BIIB141, including those previously on placebo, for about two years. Those who don’t join Part 2 will have a safety follow-up call about one month later.
  Throughout both parts of the study, participants or their caregivers will record each dose taken using a diary. The study drug can be taken at home.

  Biogen Idec Research Limited, the sponsor of the study, hopes BIIB141 will help participants and provide valuable information to support future treatment development for FA.

• REC name

  North East - York Research Ethics Committee

• REC reference

  25/NE/0144

• Date of REC Opinion

  30 Oct 2025

• REC opinion

  Further Information Favourable Opinion