MItochondrial MyopathiesPhase 2NCT02255422

RTA 408 Capsules in Patients With Mitochondrial Myopathy - MOTOR

A Phase 2 study testing Omaveloxolone capsules, 2.5 mg, omaveloxolone capsules, 5 mg and omaveloxolone capsules, 10 mg in people with MItochondrial Myopathies, run by Biogen at 9 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 18 to 75 years
Conditions the study is for: MItochondrial Myopathies
What is being tested?: Omaveloxolone capsules, 2.5 mg, omaveloxolone capsules, 5 mg and omaveloxolone capsules, 10 mg
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 53 participants
Where: 9 sitesin 2 countries
Study dates: 5 May 2015to 30 Nov 2017
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 75 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Have mitochondrial myopathy as evidenced by the following 2 criteria (must meet both):
Have a history of exercise intolerance with or without weakness and/or progressive exercise intolerance (in which modest exercise typically provokes heaviness, weakness, aching of active muscles, or tachycardia)
Have a known primary mitochondrial DNA mutation or a nuclear DNA defect that is associated with reduced activity of at least 1 mitochondrially encoded respiratory chain complex
Be male or female and ≥18 years of age and ≤75 years of age
Have no changes to exercise regimen within 30 days prior to Study Day 1 and be willing to remain on the same exercise regimen during the 16-week study period
Have the ability to complete maximal exercise testing
Have a peak workload during maximal exercise testing of ≤ 1.5 W/kg
Be able to swallow capsules

Who cannot join

Have uncontrolled diabetes (HbA1c >11.0%)
Have B-type natriuretic peptide level >200 pg/mL
Have a history of clinically significant left-sided heart disease and/or clinically significant cardiac disease
Have known active fungal, bacterial, and/or viral infection, including human immunodeficiency virus or hepatitis virus (B or C)
Have known or suspected active drug or alcohol abuse
Have clinically significant abnormalities of clinical hematology or biochemistry, including but not limited to elevations greater than 1.5 times the upper limit of normal of aspartate aminotransferase, alanine aminotransferase, or creatinine

Show all 12Show fewer

Have any abnormal laboratory test value or serious pre-existing medical condition that, in the opinion of the investigator, would put the patient at risk by study enrollment
Have taken any of the following drugs within 7 days prior to Study Day 1 or plan to take any of these drugs during the time of study participation:
Sensitive substrates for cytochrome P450 2C8 or 3A4 (e.g., repaglinide, midazolam, sildenafil)
Substrates for p-glycoprotein transporter (e.g., ambrisentan, digoxin)
Have participated in any other interventional clinical study within 30 days prior to Study Day 1
Have a cognitive impairment that may preclude ability to comply with study procedures

Trial locations and contacts

9 sites in 2 countries.

Country

City, postcode or hospital

9 sites in 2 countries

United States8 sites

Akron Children's Hospital
Akron, Ohio, 44308
Mass General Hospital
Boston, Massachusetts, 02114
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
UCLA
Los Angeles, California, 90095
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
University of Pittsburgh
Pittsburgh, Pennsylvania, 15224

Other trials in United States

Denmark1 site

Neuromuscular Clinic, Rigshospitalet, University of Copenhagen
Copenhagen, DK-2100

Other trials in Denmark

About this study

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

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
Being tested: Omaveloxolone capsules, 2.5 mg, omaveloxolone capsules, 5 mg, omaveloxolone capsules, 10 mg, Placebo capsules, omaveloxolone capsules, 20 mg
Keywords: omaveloxolone, RTA 408 capsules, mitochondrial myopathies
Medical subject headings: Mitochondrial Myopathies

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Omaveloxolone capsules, 2.5 mgDrug
Also called RTA 408 Capsules 2.5 mg
omaveloxolone capsules, 5 mgDrug
Also called RTA 408 capsules, 5 mg
omaveloxolone capsules, 10 mgDrug
Also called RTA 408, 10 mg
Placebo capsulesDrug
omaveloxolone capsules, 20 mgDrug
Also called RTA 408 capsules, 20 mg
omaveloxolone capsules, 40 mgDrug
Also called RTA 408 capsules, 40 mg
omaveloxolone capsules, 80 mgDrug
Also called RTA 408 capsules, 80 mg
omaveloxolone capsules, 160 mgDrug
Also called RTA 408 capsules, 160 mg

Study groups in NCT02255422
Study group	Role	What they receive
omaveloxolone Capsules 2.5 mg and 5 mg	Experimental	omaveloxolone (RTA 408) Capsules, 2.5 mg taken orally once daily for 2 weeks, then 5 mg taken orally once daily for 10 weeks Receives: Drug: Omaveloxolone capsules, 2.5 mg; Drug: omaveloxolone capsules, 5 mg
omaveloxolone Capsules 10 mg	Experimental	omaveloxolone (RTA 408) capsules, 10 mg taken orally once daily for 12 weeks Receives: Drug: omaveloxolone capsules, 10 mg
Placebo Capsules	Placebo comparator	Placebo capsules taken orally once daily for 12 weeks Receives: Drug: Placebo capsules
omaveloxolone Capsules 20 mg	Experimental	omaveloxolone (RTA 408) Capsules, 20 mg taken orally once daily for 12 weeks. Receives: Drug: omaveloxolone capsules, 20 mg
omaveloxolone Capsules 40 mg	Experimental	omaveloxolone (RTA 408) Capsules, 40 mg taken orally once daily for 12 weeks. Receives: Drug: omaveloxolone capsules, 40 mg
omaveloxolone Capsules 80 mg	Experimental	omaveloxolone (RTA 408) Capsules, 80 mg taken orally once daily for 12 weeks. Receives: Drug: omaveloxolone capsules, 80 mg
omaveloxolone Capsules 160 mg	Experimental	omaveloxolone (RTA 408) Capsules, 160 mg taken orally once daily for 12 weeks. Receives: Drug: omaveloxolone capsules, 160 mg

What is being measured

The primary outcome is the main question the study is designed to answer.

Change of Peak Workload (in Watts/kg) During Exercise Testing
Cycle ergometry using a stationary recumbent bike was used to conduct maximal exercise testing. Peak work is defined as the workload at which patients reach maximal volition (defined as an inability to continue to exercise due to exhaustion). Change of peak workload during exercise testing was measured at baseline, Week 4, and Week 12. Change from baseline at Week 12 reported.
Measured: 12 weeks

Secondary outcomes (1)

Change in 6-minute Walk Test (6MWT) Distance
Patients were instructed to walk as far as they could along a marked path for 6 minutes. Distance walked was measured. If patients used a cane or walking assist device at Screening, the same walking assist device was to be used for all 6MWT assessments.
Measured: 6MWT was assessed at Week 4, Week 8, and Week 12 and compared to baseline

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

5 May 201530 Nov 2017

First posted2 Oct 2014 (estimated)
Study start5 May 2015
Primary completion2 Nov 2017
Study completion30 Nov 2017

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for MItochondrial MyopathiesStudies of the same condition, open and under way.
More trials run by BiogenThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Sequential
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 53 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02255422
Sponsor's protocol ID: RTA 408-C-1403
Status last verified: May 2025
First submitted: 30 Sep 2014
Last updated: 5 Jun 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
Individual data sharing: Yes: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

Publications

Madsen KL, Buch AE, Cohen BH, Falk MJ, Goldsberry A, Goldstein A, Karaa A, Koenig MK, Muraresku CC, Meyer C, O'Grady M, Scaglia F, Shieh PB, Vockley J, Zolkipli-Cunningham Z, Haller RG, Vissing J. Safety and efficacy of omaveloxolone in patients with mitochondrial myopathy: MOTOR trial. Neurology. 2020 Feb 18;94(7):e687-e698. doi: 10.1212/WNL.0000000000008861. Epub 2020 Jan 2.
PubMed 31896620

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All MItochondrial Myopathies trials

Study at a glance

From the registry record, last updated by the sponsor on 5 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Mitochondrial Myopathy (MOTOR)
ClinicalTrials.gov ID: NCT02255422
Acronym / sponsor ID: RTA 408-C-1403
Conditions: MItochondrial Myopathies
Keywords: omaveloxolone, RTA 408 capsules, mitochondrial myopathies
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 53 participants (actual)
Lead sponsor: BiogenIndustry (pharma, biotech, devices)
Collaborators: AbbVie
Study start: 5 May 2015 (actual)
Primary completion: 2 Nov 2017 (actual)
Study completion: 30 Nov 2017 (actual)
First posted: 2 Oct 2014 (estimated)
Results: Posted 17 Sep 2020

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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 design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Sequential
Masking: Double blind (participant, investigator)
Number of arms: 7

Arm	Type	Receives
omaveloxolone Capsules 2.5 mg and 5 mg	Experimental	Drug: Omaveloxolone capsules, 2.5 mg, Drug: omaveloxolone capsules, 5 mgomaveloxolone (RTA 408) Capsules, 2.5 mg taken orally once daily for 2 weeks, then 5 mg taken orally once daily for 10 weeks
omaveloxolone Capsules 10 mg	Experimental	Drug: omaveloxolone capsules, 10 mgomaveloxolone (RTA 408) capsules, 10 mg taken orally once daily for 12 weeks
Placebo Capsules	Placebo comparator	Drug: Placebo capsulesPlacebo capsules taken orally once daily for 12 weeks
omaveloxolone Capsules 20 mg	Experimental	Drug: omaveloxolone capsules, 20 mgomaveloxolone (RTA 408) Capsules, 20 mg taken orally once daily for 12 weeks.
omaveloxolone Capsules 40 mg	Experimental	Drug: omaveloxolone capsules, 40 mgomaveloxolone (RTA 408) Capsules, 40 mg taken orally once daily for 12 weeks.
omaveloxolone Capsules 80 mg	Experimental	Drug: omaveloxolone capsules, 80 mgomaveloxolone (RTA 408) Capsules, 80 mg taken orally once daily for 12 weeks.
omaveloxolone Capsules 160 mg	Experimental	Drug: omaveloxolone capsules, 160 mgomaveloxolone (RTA 408) Capsules, 160 mg taken orally once daily for 12 weeks.

Who may qualify

Ages: 18 to 75 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (8 listed)

Have mitochondrial myopathy as evidenced by the following 2 criteria (must meet both):
Be male or female and ≥18 years of age and ≤75 years of age
Have no changes to exercise regimen within 30 days prior to Study Day 1 and be willing to remain on the same exercise regimen during the 16-week study period
Have the ability to complete maximal exercise testing
Have a peak workload during maximal exercise testing of ≤ 1.5 W/kg

Key exclusion criteria (12 listed)

Have uncontrolled diabetes (HbA1c >11.0%)
Have B-type natriuretic peptide level >200 pg/mL
Have a history of clinically significant left-sided heart disease and/or clinically significant cardiac disease
Have known active fungal, bacterial, and/or viral infection, including human immunodeficiency virus or hepatitis virus (B or C)
Have known or suspected active drug or alcohol abuse

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Omaveloxolone capsules, 2.5 mgAlso: RTA 408 Capsules 2.5 mg	Drug	omaveloxolone Capsules 2.5 mg and 5 mg
omaveloxolone capsules, 5 mgAlso: RTA 408 capsules, 5 mg	Drug	omaveloxolone Capsules 2.5 mg and 5 mg
omaveloxolone capsules, 10 mgAlso: RTA 408, 10 mg	Drug	omaveloxolone Capsules 10 mg
Placebo capsules	Drug	Placebo Capsules
omaveloxolone capsules, 20 mgAlso: RTA 408 capsules, 20 mg	Drug	omaveloxolone Capsules 20 mg
omaveloxolone capsules, 40 mgAlso: RTA 408 capsules, 40 mg	Drug	omaveloxolone Capsules 40 mg
omaveloxolone capsules, 80 mgAlso: RTA 408 capsules, 80 mg	Drug	omaveloxolone Capsules 80 mg
omaveloxolone capsules, 160 mgAlso: RTA 408 capsules, 160 mg	Drug	omaveloxolone Capsules 160 mg

Primary outcome

Change of Peak Workload (in Watts/kg) During Exercise Testing
Time frame: 12 weeks
Cycle ergometry using a stationary recumbent bike was used to conduct maximal exercise testing. Peak work is defined as the workload at which patients reach maximal volition (defined as an inability to continue to exercise due to exhaustion). Change of peak workload during exercise testing was measured at baseline, Week 4, and Week 12. Change from baseline at Week 12 reported.

1 secondary outcome

Change in 6-minute Walk Test (6MWT) Distance
Time frame: 6MWT was assessed at Week 4, Week 8, and Week 12 and compared to baseline

Results and safety

Results posted to the registry on 17 Sep 2020, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Placebo	13	13
Omaveloxolone Capsules 2.5 and 5 mg	6	6
Omaveloxolone Capsules 10 mg	6	5
Omaveloxolone Capsules 20 mg	6	6
Omaveloxolone Capsules 40 mg	6	6
Omaveloxolone Capsules 80 mg	6	6
Omaveloxolone Capsules 160 mg	10	8

Primary outcome: Change of Peak Workload (in Watts/kg) During Exercise Testing

Time frame: 12 weeks. Least squares mean (Standard Error). Unit: Watts/kg

Measure	Placebo n = 12	Omaveloxolone Capsules 2.5 and 5 mg n = 6	Omaveloxolone Capsules 10 mg n = 6	Omaveloxolone Capsules 20 mg n = 5	Omaveloxolone Capsules 40 mg n = 6	Omaveloxolone Capsules 80 mg n = 6	Omaveloxolone Capsules 160 mg n = 9
Value	0.0090 (0.0388)	0.020 (0.0544)	-0.0300 (0.0544)	0.1050 (0.0596)	-0.0520 (0.0544)	-0.0660 (0.0544)	0.0020 (0.0465)

Placebo vs Omaveloxolone Capsules 2.5 and 5 mg vs Omaveloxolone Capsules 10 mg vs Omaveloxolone Capsules 20 mg vs Omaveloxolone Capsules 40 mg vs Omaveloxolone Capsules 80 mg vs Omaveloxolone Capsules 160 mg: LS mean difference (net): -0.015 (95% CI -0.1051 to 0.0743), p = 0.7321, Mixed Models Analysis

Population analysed: Three patients out of the 53 total were excluded from the analysis set. One each from the 20 mg arm and the 160 mg arm were excluded because they did not have post-baseline efficacy assessments. One patient from the placebo arm was excluded from analysis because their baseline maximal exercise test duration \< 4 min and was not considered valid.

1 secondary outcome is reported in the full results.

Adverse events

Time frame: 16 weeks. Other adverse events reported at 2% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Placebo	1 of 13 (7.7%)	11 of 13 (84.6%)	0 of 13
Omaveloxolone Capsules 2.5 and 5 mg	0 of 6 (0%)	4 of 6 (66.7%)	0 of 6
Omaveloxolone Capsules 10 mg	1 of 6 (16.7%)	6 of 6 (100%)	0 of 6
Omaveloxolone Capsules 20 mg	0 of 6 (0%)	6 of 6 (100%)	0 of 6
Omaveloxolone Capsules 40 mg	1 of 6 (16.7%)	6 of 6 (100%)	0 of 6
Omaveloxolone Capsules 80 mg	1 of 6 (16.7%)	4 of 6 (66.7%)	0 of 6
Omaveloxolone Capsules 160 mg	1 of 10 (10%)	10 of 10 (100%)	0 of 10

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 5 Jun 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 31896620Madsen KL, Buch AE, Cohen BH, Falk MJ, Goldsberry A, Goldstein A, Karaa A, Koenig MK, Muraresku CC, Meyer C, O'Grady M, Scaglia F, Shieh PB, Vockley J, Zolkipli-Cunningham Z, Haller RG, Vissing J. Safety and efficacy of omaveloxolone in patients with mitochondrial myopathy: MOTOR trial. Neurology. 2020 Feb 18;94(7):e687-e698. doi: 10.1212/WNL.0000000000008861. Epub 2020 Jan 2.
Individual participant dataSharing planned: Yes. In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

9 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 5 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in United States, Denmark. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 17 Sep 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change of Peak Workload (in Watts/kg) During Exercise Testing (time frame: 12 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02255422, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jun 2025.