Category: Pediatric Movement Disorders
Objective: This phase 3 randomized controlled trial will assess the efficacy and safety of omaveloxolone in children aged 2 to <16 years.
Background: Omaveloxolone is approved for the treatment of patients with Friedreich ataxia (FA) aged ≥16 years based on the registrational MOXIe study and its open-label extension (OLE) (NCT02255435, EudraCT2015-002762-23). However, the early onset and more uniformly rapid progression of FA in children younger than 16 years emphasizes the need for therapies in pediatric populations.
Method: This 2-part phase 3 global clinical trial comprises a randomized, double-blind, placebo-controlled study (Part 1) and OLE (Part 2) to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of omaveloxolone in participants with FA aged 2 to <16 years. This study aims to enroll approximately 255 participants with genetically confirmed FA, randomized 2:1 to receive omaveloxolone or placebo. The primary objective of Part 1 is to evaluate the efficacy of omaveloxolone as measured by change from baseline in upright stability score (USS), a subscale of the modified Friedreich Ataxia Rating Scale (mFARS), at Week 52. Participants who complete Part 1 will be eligible to move on to the OLE (Part 2); the primary objective of Part 2 is to evaluate the safety and tolerability of long-term omaveloxolone use up to 104 weeks.
Results: In the MOXIe study, the mFARS was used as a primary outcome measure for assessing neurological progression in patients with FA aged ≥16 years. However, the utility of the mFARS as a primary outcome measure in the pediatric population is limited by high variability and heterogeneity across subgroups. Therefore, our pediatric study will use the USS, which is supported by findings of less variable and more consistent annual progression based on the natural history data, suggesting upright stability to be an appropriate outcome measure in this population.
Conclusion: Results from this study will inform the efficacy and safety of omaveloxolone in patients with FA aged 2 to <16 years, with the goal of providing expanded approval in this pediatric population with unmet need.
To cite this abstract in AMA style:
D. Lynch, M. Delatycki, M. França, A. Durr, E. Bertini, S. Perlman, A. Arizpe, R. Domingo-Horne, S. Fradette, S. Sinks, M. Murai, N. Folschweiller. Design and Outcome Measures of BRAVE, a Phase 3 Study of Omaveloxolone in Pediatric Patients With Friedreich Ataxia [abstract]. Mov Disord. 2025; 40 (suppl 1). https://www.mdsabstracts.org/abstract/design-and-outcome-measures-of-brave-a-phase-3-study-of-omaveloxolone-in-pediatric-patients-with-friedreich-ataxia/. Accessed October 5, 2025.« Back to 2025 International Congress
MDS Abstracts - https://www.mdsabstracts.org/abstract/design-and-outcome-measures-of-brave-a-phase-3-study-of-omaveloxolone-in-pediatric-patients-with-friedreich-ataxia/