Study Overview:

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 funded by Reata Pharmaceuticals, Inc., a wholly-owned subsidiary of 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:

As of July 1, 2024, the first cohort of the study has completed enrollment and begun dosing of participants aged 12 to 15 years old. Additional and younger cohorts (between ages 6-11 and 2-5 years) will be recruited separately after safety data from this first cohort is reviewed. You can learn more about the study and cohort design by watching the FA Community Conversations webinar recording from May 2, 2024.

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.

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 12 to 15 years old
  • Reside within the United States 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.

How to participate:

Recruitment information for younger cohorts (between ages 6-11 and 2-5 years) will be announced at a later date.

Additional inclusion and exclusion criteria may apply and will be evaluated by a study doctor.

Length of Study Commitment:

  • Two Weeks

Participating Study Locations

Institution Name and LocationInvestigatorStudy Coordinator Contact InformationStatus

Children’s Hospital of Philadelphia
Philadelphia, PA

David Lynch, MD

McKenzie Wells,
Courney Park,
Medina Keita,
Victoria Profeta,
Katherine Gunther,

Not recruiting

Explore the FA Drug Development Pipeline

FARA believes that there are many different approaches to treating Friedreich’s ataxia, and that it will require a cocktail approach of two or more treatments to slow, stop, reverse, and cure FA. Learn more about the approach behind this potential treatment and explore the other approaches that are in the FA Drug Development pipeline.