Patient-reported experiences during long-term omaveloxolone treatment in Friedreich ataxia: survey design and planned qualitative analysis

Topic:

Other

Poster Number: 336 T

Author(s):

Susan Perlman, MD, University of California, Los Angeles, Theresa Zesiewicz, MD, MD, University of South Florida, Matthew Lafleur, MA, Bionews, Inc., Pensacola, FL, USA, Libby Schwers, BA, With Love, Libby, Des Moines, Iowa, USA, Jennifer Farmer, MS, Friedreich's Ataxia Research Alliance, Pearl WU, MS, Biogen, Claudia Yang Santos, PhD, Biogen, Inc., Kyle Fowler, MBiolSci, Syneos Health Consulting, Zurich, Switzerland, Fiona Thomas, MBChB, Syneos Health Consulting, London, UK, David Lynch, MD, PhD, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA

Objective: This qualitative survey will explore patient-reported lived experiences and perceptions of functional outcomes among adults (≥18 years) with Friedreich Ataxia (FA) receiving long-term omaveloxolone treatment in the United States.

Background: Omaveloxolone is approved for the treatment of FA in patients aged ≥16 years, with safety and efficacy demonstrated in the MOXIe clinical trial program (NCT02255435) and its open-label extension. There are limited insights available that describe the long-term real-world experiences, perceived benefits, and ongoing challenges faced by individuals receiving omaveloxolone treatment. Collecting patient perspectives is essential for understanding changes in daily functioning, quality of life, and unmet needs.

Methods: This qualitative survey, initiated in November 2025 with an anticipated completion in January 2026, will include structured one-on-one interviews with 20 adults with FA who previously participated in the MOXIe clinical trial and have been on omaveloxolone for ≥3 years. Participants will be recruited in partnership with Friedreich’s Ataxia Research Alliance (FARA) and with support requested from all MOXIe investigators.

Interviews will cover domains such as mobility and independence, fatigue and energy levels, communication, daily functioning, participation in work or education, and social engagement. Interview transcripts will undergo analysis from a Steering Committee that includes medical experts, researchers and patient representatives to identify patterns in patient experience, perceived treatment impact, and persistent unmet needs.

Results: The findings will summarize patient-reported experiences with long-term omaveloxolone treatment and highlight areas that participants deem most impactful.

Conclusions: This survey will offer qualitative insights into the long-term experiences of adults with FA treated with omaveloxolone. By integrating patient perspectives with clinical understanding, the results may inform patient-centered care, refine outcome measures for future FA research, and enhance understanding of long-term treatment experiences.