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

To join this study, people need to be between 2 and 15 years old and have a confirmed diagnosis of Friedreich’s Ataxia. For those aged 7 to under 16, there's also a specific test of balance they need to meet.

However, some health conditions might mean someone can't take part. For example, if they have very high blood sugar levels (measured by a test called HbA1c), a particular heart marker (BNP) that's too high, or a heart function (ejection fraction) that's too low. People with serious heart conditions, other than a mild to moderate form often seen in Friedreich’s Ataxia, might also not be able to join.

There might be other reasons, not listed here, why someone can or cannot join. The study doctors will carefully check everyone's health to make sure it's safe for them to take part.

If you decide to join, you'll first have a screening period of up to 4 weeks to check if the study is right for you. Then, for about one year (Part 1), you'll be randomly given either omaveloxolone or a dummy pill that looks the same but has no active medicine. Neither you, your doctor, nor the study staff will know which you are receiving. During this time, you'll have up to 9 visits to the clinic and one phone call. After about a month of finishing this part, you'll have a safety check by phone.

If you complete Part 1, you'll then move to Part 2 for about two more years, where everyone will receive omaveloxolone. You'll have up to 8 more clinic visits and one phone call. About a month after you stop taking the medicine in Part 2, you'll have a final follow-up phone call. In total, you could be in the study for up to 3 years, with a total of up to 17 clinic visits and 3 phone calls.