RTA 408 Capsules in Patients With Mitochondrial Myopathy - MOTOR
Public ClinicalTrials.gov record NCT02255422. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Mitochondrial Myopathy (MOTOR)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT02255422
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 53 participants
Conditions and interventions
Conditions
Interventions
- 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 Drug
Drug
Eligibility (public fields only)
- Age range
- 18 Years to 75 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- May 4, 2015
- Primary completion
- Nov 1, 2017
- Completion
- Nov 29, 2017
- Last update posted
- Jun 4, 2025
2015 – 2017
United States locations
- U.S. sites
- 8
- U.S. states
- 5
- U.S. cities
- 7
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| UCLA | Los Angeles | California | 90095 | — |
| Mass General Hospital | Boston | Massachusetts | 02114 | — |
| Akron Children's Hospital | Akron | Ohio | 44308 | — |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| University of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | — |
| Insitute for Exercise & Environmental Medicine | Dallas | Texas | 75231 | — |
| Baylor College of Medicine | Houston | Texas | 77030 | — |
| University of Texas Medical School at Houston | Houston | Texas | 77030 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 1 non-U.S. site.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02255422, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jun 4, 2025 · Synced Sep 6, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT02255422 live on ClinicalTrials.gov.