NCT02255422

RTA 408 Capsules in Patients With Mitochondrial Myopathy - MOTOR

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 53 (actual)
• Sponsor: Biogen · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

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. 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.

Conditions

• MItochondrial Myopathies

Interventions

Type	Name	Description
DRUG	Omaveloxolone capsules, 2.5 mg
DRUG	omaveloxolone capsules, 5 mg
DRUG	omaveloxolone capsules, 10 mg
DRUG	Placebo capsules
DRUG	omaveloxolone capsules, 20 mg
DRUG	omaveloxolone capsules, 40 mg
DRUG	omaveloxolone capsules, 80 mg
DRUG	omaveloxolone capsules, 160 mg

Timeline

• Start date: 2015-05-05
• Primary completion: 2017-11-02
• Completion: 2017-11-30
• First posted: 2014-10-02
• Last updated: 2025-06-05
• Results posted: 2020-09-17

Locations

9 sites across 2 countries: United States, Denmark