Clinical Trial: RTA 408 Capsules in Patients With Mitochondrial Myopathy - MOTOR

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Mitochondrial Myopathy (MOTOR)

Brief Summary:

Mitochondrial myopathies are a multisystemic group of disorders that are characterized by a wide range of biochemical and genetic mitochondrial defects and variable modes of inheritance. Currently there are no effective treatments for this disease. Despite the heterogeneous myopathy phenotypes, a unifying feature of mitochondrial myopathies is that the pathogenic mtDNA mutations and/or nuclear mutations of the electron transport chain invariably lead to dysfunctional mitochondrial respiration. This reduction in mitochondrial respiration leads to a reduced ability to produce cellular adenosine triphosphate (ATP), often resulting in muscle weakness, exercise intolerance, and fatigue in patients with mitochondrial myopathies.

RTA 408 is a potent activator of Nrf2 and inhibitor of NF κB (nuclear factor kappa-light-chain-enhancer of activated B cells), and thus induces an antioxidant and anti-inflammatory phenotype. Several lines of evidence suggest that Nrf2 activation can increase mitochondrial respiration and biogenesis. Collectively, available data suggest that the ability of RTA 408 to activate Nrf2 and induce its target genes could potentially improve muscle function, oxidative phosphorylation, antioxidant capacity, and mitochondrial biogenesis in patients with mitochondrial myopathies.

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 mitochondrial myopathies.

Part 2: The second part of this study is a randomized, placebo-controlled, double-blind, parallel study to evaluate the safety, efficacy, and pharmacodynamics of up to 2 dose levels of omaveloxolone (RTA 408) in patients with mitochondrial myopathies. Eligible pati

Detailed Summary:
Sponsor: Reata Pharmaceuticals, Inc.

Current Primary Outcome: Measure the change of peak workload (in watts/kg) during exercise testing [ Time Frame: 12 weeks ]

Original Primary Outcome: Same as current

Current Secondary Outcome: Measure the change in distance walked during a 6-minute walk test [ Time Frame: 12 weeks ]

Original Secondary Outcome: Same as current

Information By: Reata Pharmaceuticals, Inc.

Dates:
Date Received: September 30, 2014
Date Started: April 2015
Date Completion: December 2017
Last Updated: November 14, 2016
Last Verified: November 2016