The Children’s Hospital of Philadelphia (CHOP) is conducting a study that will investigate dose-safety and pharmacokinetics (PK) of omaveloxolone in a pediatric population. The study is sponsored by Biogen.
Anyone considering participating in a clinical trial should discuss the matter with their physician. FARA does not endorse or recommend any particular studies.
Study Details:
Participants will receive a single dose of omaveloxolone followed by a 15-day monitoring period to study safety and tolerability. Participants will have the option to roll over onto an open label drug extension study.
You can learn more about the study and cohort design by watching the FA Community Conversations webinar recording from May 2, 2024.
Key Inclusion Criteria:
- Have a genetic test confirming a diagnosis of Friedreich’s ataxia, with a copy you can share with the study team.
- Are 2-5 years old
- Reside within the United States or Canada and speak English.
- Are willing and able to swallow capsules or eat capsule contents sprinkled on applesauce.
- Have NO clinically significant cardiac disease (such as hospitalization for heart failure, arrhythmia, or outflow tract obstruction).
- Are willing to have multiple blood draws.
- Are willing and able to stay in Philadelphia for roughly 2 weeks, or travel to Philadelphia multiple times within a 1-month period.
Additional inclusion and exclusion criteria may apply and will be evaluated by a study doctor.
Length of Study Commitment:
- Two Weeks