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Designing phase II clinical trials in Friedreich ataxia

Therapeutic approaches in FRDA have consisted of two different approaches: (1) augmenting or restoring frataxin production and (2) modulating a variety of downstream processes related to mitochondrial dysfunction, including reactive oxygen species production, ferroptosis, or Nrf2 activation. In this review, the authors summarize data from major phase II clinical trials in FRDA published between 2015 and 2020 which includes A0001/EPI743, Omaveloxolone, RT001, and Actimmune. A growing number of drug candidates are being tested in phase II clinical trials for FRDA; however, most have not met their primary endpoints, and none have received FDA approval. In this review, the authors aim to summarize completed phase II clinical trials in FRDA, outlining critical lessons that have been learned and that should be incorporated into future trial design to ultimately optimize drug development in FRDA.

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