Analysis of variance

Statistics

Ataxia

Surgery

Psychiatry

Placebo

Alternative medicine

Mathematics

Repeated measures design

Physical therapy

Internal medicine

Medicine

Randomized controlled trial

Pathology

Movement Disorders

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. Conclusions These 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.

Efficacy of Omaveloxolone in Friedreich's Ataxia: <scp>Delayed‐Start</scp> Analysis of the <scp>MOXIe</scp> Extension

Efficacy of Omaveloxolone in Friedreich's Ataxia: Delayed‐Start Analysis of the MOXIe Extension