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RTA 408 Capsules in Patients With Mitochondrial Myopathy - MOTOR

NCT02255422 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 53

Last updated 2025-06-05

Study results available
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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

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

Sponsors & Collaborators

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-05-05
Primary Completion
2017-11-02
Completion
2017-11-30

Countries

  • United States
  • Denmark

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02255422 on ClinicalTrials.gov