Clinical Research Alert: Phase 3 Study of Omaveloxolone in Children and Teenagers with Friedreich Ataxia (FA)

Researchers at Biogen are seeking pediatric FA patients to participate in a phase 3 clinical trial to better understand Friedreich ataxia (FA) and to evaluate omaveloxolone, an investigational therapy for children and teenagers with FA.

The study 

Children and teenagers with FA who are 2 to 15 years old may be eligible to participate in a phase 3 clinical trial (BRAVE) to evaluate the efficacy and safety of the investigational therapy omaveloxolone to treat FA. This study is evaluating safety of omaveloxolone and how well it works for the treatment of FA in these younger individuals. Omaveloxolone is approved by the U.S. Food and Drug Administration (FDA), European Medicines Agency and in multiple countries around the world to treat FA in patients aged 16 years and older. It is currently an investigational product for patients ages 2 to 15 years old.

The BRAVE study is a phase 3 study where participants will be randomly assigned to receive omaveloxolone or a placebo control for a year (Part 1). Participants are twice as likely to be assigned to receive omaveloxolone than to receive placebo. The primary objective of the study is to evaluate changes in upright stability – a measure including how well someone can stay balanced while standing and walking – after one year of treatment. Upright stability is identified as a sensitive way to measure disease progression in children living with FA. The effects of the drug will also be evaluated using other scales and measures.

Participants who complete Part 1 of the study may be eligible to continue into Part 2 of BRAVE in which all participants will receive omaveloxolone for an additional duration of 2 years. Participants in Part 1 will engage in a screening visit, followed by clinic visits on day 1, weeks 4, 8 (home or clinic), 12, 26, 39, and 52. Additional visits are expectedPart 2 of the study. The total study duration for participants will be about 3 years. Omaveloxolone will be administered orally (by mouth).

Study criteria 

To be eligible for this study, individuals must be aged 2 to 15 years old and meet the inclusion and exclusion criteria outlined by the study investigators.

Please visit this link for the full listing of inclusion and exclusion criteria.

Interested in participating? 

Travel support will be available for eligible participants.

People interested in learning more can reach out to the BRAVE Patient Navigator by phone at 1-877-223-3576 ext. 57078 or by visiting the sponsor’s website.

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