Phase 3 Study to Assess Safety, Effects, and How the Body Processes Omaveloxolone in Children and Teens Ages 2 to 15 with Friedreichs Ataxia

What this Trial Is About

Researchers are studying omaveloxolone, also known as BIIB141 or SKYCLARYS, to understand its effects and safety in children and teens aged 2 to 15 years with Friedreichs Ataxia FA. Omaveloxolone is already approved for people 16 years and older, but this study focuses on younger participants to see how the drug affects their FA symptoms, overall health, and heart function. The study also examines how the body processes omaveloxolone in this younger age group. Participants will first be screened for up to 4 weeks to determine eligibility. In Part 1, they will be randomly assigned to receive either omaveloxolone or a placebo by mouth once daily for about one year in a double-blind setup. Participants will have up to 9 clinic visits and 1 phone call during this time. Those who complete Part 1 can join Part 2, where all participants will receive omaveloxolone openly for about two years, with up to 8 clinic visits and 1 phone call. After stopping the medication, a follow-up phone call will occur about one month later. Throughout the study, participants will undergo various assessments including the modified Friedreichs Ataxia Rating Scale mFARS, questionnaires on quality of life and daily activities, and tests of muscle strength and nerve function. Researchers will monitor safety by tracking adverse events and heart health through echocardiograms. The total participation time can be up to three years, including all visits and follow-ups, to evaluate long-term effects and safety of omaveloxolone in young people with FA.

A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia