Journal article
Efficacy of Omaveloxolone in Friedreich's Ataxia: Delayed-Start Analysis of the MOXIe Extension
Movement disorders, Vol.38(2), pp.313-320
02/2023
DOI: 10.1002/mds.29286
PMID: 36444905
Abstract
BACKGROUND MOXIe was a two-part study evaluating the safety and efficacy of omaveloxolone in patients with Friedreich's ataxia, a rare, progressive neurological disease with no proven therapy. MOXIe part 2, a randomized double-blind placebo-controlled trial, showed omaveloxolone significantly improved modified Friedreich's Ataxia Rating Scale (mFARS) scores relative to placebo. Patients who completed part 1 or 2 were eligible to receive omaveloxolone in an open-label extension study. OBJECTIVE The delayed-start study compared mFARS scores at the end of MOXIe part 2 with those at 72 weeks in the open-label extension period (up to 144 weeks) for patients initially randomized to omaveloxolone versus those initially randomized to placebo. METHODS We performed a noninferiority test to compare the difference between treatment groups (placebo to omaveloxolone versus omaveloxolone to omaveloxolone) using a single mixed model repeated measures (MMRM) model. In addition, slopes of the change in mFARS scores were compared between both groups in the open-label extension. RESULTS The noninferiority testing demonstrated that the difference in mFARS between omaveloxolone and placebo observed at the end of placebo-controlled MOXIe part 2 (-2.17 ± 1.09 points) was preserved after 72 weeks in the extension (-2.91 ± 1.44 points). In addition, patients previously randomized to omaveloxolone in MOXIe part 2 continued to show no worsening in mFARS relative to their extension baseline through 144 weeks. CONCLUSIONSThese results support the positive results of MOXIe part 2 and indicate a persistent benefit of omaveloxolone treatment on disease course in Friedreich's ataxia. © 2022 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
Details
- Title: Subtitle
- Efficacy of Omaveloxolone in Friedreich's Ataxia: Delayed-Start Analysis of the MOXIe Extension
- Creators
- David R LynchMelanie P Chin - Reata Pharmaceuticals (United States)Sylvia Boesch - Innsbruck Medical UniversityMartin B Delatycki - Victorian Clinical Genetics ServicesPaola Giunti - University College HospitalAngie Goldsberry - Reata Pharmaceuticals (United States)J Chad Hoyle - The Ohio State UniversityCaterina Mariotti - Fondazione IRCCS Istituto Neurologico Carlo BestaKatherine D Mathews - Roy J. and Lucille A. Carver College of MedicineWolfgang Nachbauer - Innsbruck Medical UniversityMegan O'Grady - Reata Pharmaceuticals (United States)Susan Perlman - University of California, Los AngelesS H Subramony - McKnight Brain InstituteGeorge Wilmot - Emory UniversityTheresa Zesiewicz - University of South FloridaColin J Meyer - Reata Pharmaceuticals (United States)
- Resource Type
- Journal article
- Publication Details
- Movement disorders, Vol.38(2), pp.313-320
- DOI
- 10.1002/mds.29286
- PMID
- 36444905
- NLM abbreviation
- Mov Disord
- ISSN
- 0885-3185
- eISSN
- 1531-8257
- Language
- English
- Electronic publication date
- 11/29/2022
- Date published
- 02/2023
- Academic Unit
- Neurology; Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Neurology (Pediatrics)
- Record Identifier
- 9984321959902771
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