St. Jude Care & Treatment

Menu

Close

Recruiting

BRAVE: Omaveloxone for Friedreich’s Ataxia

About this study

Friedreich’s ataxia (FA) is a rare disease that affects the brain and other body systems, causing problems with movement, balance, and coordination. While there is no cure for FA, the medicine omaveloxolone (BIIB 141) was approved by the FDA to treat the symptoms of FA in patients over 16 years old.

In this study, we want to find out if omaveloxolone is safe and works well to treat the symptoms of Friedreich’s ataxia in children and teens. Participants will get either the medicine omaveloxolone as a pill, or they will get a pretend pill (placebo). The placebo pill looks the same, but it does not have the medicine omaveloxolone in it.

This study will last for about 3 years. Participants will have a screening visit to see if they can take part. If so, they will get a medical exam; an electrocardiogram (ECG) to check their heart function; and an echocardiogram (ECHO), which takes pictures of their heart using sound waves. They will have blood and urine tests. They will also have balance and other movement tests.

Participants will be randomly put into 1 of 2 groups. They will not know which group they are in. In the first part of the study, one group will get the pill with omaveloxone. The other group will get the placebo. This first part of the study will last for 52 weeks. Each visit during this time will take about 6 hours. Participants will answer questions about their daily life, how well they do things, and how they feel. Only children age 7 and older will answer these questions by themselves.

After this part of the study, participants and their families can choose if they want to go to the second part of the study. In this part, all participants will get omaveloxone. This part of the study will last up to 104 weeks

This study is important because it helps doctors learn if a medicine that works for older teens and adults with Friedreich’s ataxia can also help younger children. If it works and is safe, it could give children with this disease a better chance to feel stronger and do everyday things more easily.

Eligibility overview

  • Has Friedrich’s ataxia
  • 2–15 years old

Request a referral

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.

Overview

Full title:

A Phase 3, 2-Part, Randomized, Double-blind, Placebo-controlled Study (Part 1) and Open-label Extension (Part 2) to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Omaveloxolone (BIIB 141) in Participants with Friedreich’s Ataxia Aged 2 to < 16 Years (296FA301)

Study goal:

To study the use of omaveloxolone for Friedreich’s ataxia symptoms in children and young teens

Diagnosis:

Friedreich’s Ataxia

Age:

2–15 years

Clinical trial categories:

Neurological Disorders Friedreich’s Ataxia

For physicians and researchers

Patients accepted to St. Jude must be referred by a physician or other qualified medical professional. Learn how St. Jude can partner with you to care for your patient.

 

Learn more