Last updated on September 2017

RTA 408 Capsules in Patients With Friedreich's Ataxia - MOXIe


Brief description of study

Friedreich's ataxia is an autosomal recessive cerebellar ataxia caused by triplet-repeat expansions. The causative mutation is a trinucleotide (GAA) repeat expansion in the first intron of the frataxin gene, leading to impaired transcription of frataxin. The pathological consequences of frataxin deficiency include a severe disruption of iron-sulfur cluster biosynthesis, mitochondrial iron overload coupled to cellular iron dysregulation, and an increased sensitivity to oxidative stress. A hallmark of Friedreich's ataxia is impairment of antioxidative defense mechanisms, which play a major role in disease progression. Studies have demonstrated that nuclear factor erythroid-derived 2-related factor 2 (Nrf2) signaling is grossly impaired in patients with Friedreich's ataxia. Therefore, the ability of omaveloxolone (RTA 408) to activate Nrf2 and induce antioxidant target genes is hypothesized to be therapeutic in patients with Friedreich's ataxia. This 2-part study will evaluate the efficacy, safety, and pharmacodynamics of omaveloxolone (RTA 408) in the treatment of patients with Friedreich's ataxia. Part 1: The first part of this study will be a randomized, placebo-controlled, double-blind, dose-escalation study to evaluate the safety of omaveloxolone (RTA 408) at various doses in patients with Friedreich's ataxia. Part 2: The second part of this study is a randomized, placebo-controlled, double-blind, parallel-group study to evaluate the safety and efficacy of omaveloxolone (RTA 408) 150 mg in patients with Friedreich's ataxia. Patients enrolled in Part 2 will be randomized 1:1 to receive omaveloxolone (RTA 408) 150 mg or placebo. Extension: The extension will assess long-term safety and tolerability of omaveloxolone (RTA 408) in qualified patients with Friedreich's ataxia following completion of Part 1 or Part 2. Patients will not be unblinded to study treatment in Part 1 or Part 2 upon entering the extension study. Patients will receive open-label omaveloxolone (RTA 408) at 150 mg once daily.

Clinical Study Identifier: NCT02255435

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Susan Perlman, MD

UCLA
Los Angeles, CA United States
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SH Subramony, MD

University of Florida - Neurology
Gainesville, FL United States
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Theresa Zesiewicz, MD

USF Ataxia Research Center
Tampa, FL United States
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George Wilmot, MD

Emory University Hospital - Neurology
Atlanta, GA United States
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Katherine Mathews, MD

University of Iowa Stead Family Children's Hospital
Iowa City, IA United States
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Chad Hoyle, MD

Ohio State University - Neurology
Columbus, OH United States
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David Lynch, MD

Children's Hospital of Philadelphia
Philadelphia, PA United States
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Martin Delatycki, MD

Murdoch Childrens Research Institute
Parkville, Australia
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Sylvia Boesch, MD

Medical University Innsbruck
Innsbruck, Austria
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Paola Giunti, MD

University College of London
London, United Kingdom
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