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New drug aims to help mitochondrial disease patients walk farther

NCT ID NCT05162768

Disease control Sponsor: Stealth BioTherapeutics Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This Phase 3 trial tested a daily injection called elamipretide in 102 adults with primary mitochondrial myopathy, a genetic condition that causes muscle weakness and fatigue. Participants received either the drug or a placebo for 48 weeks. The main goal was to see if the drug could improve how far they could walk in six minutes.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
elamipretide (a drug given as a daily injection)
What this could lead to
If it works, this could point toward a treatment that improves muscle function and daily activity in people with mitochondrial disease.
What could go wrong
This is a completed Phase 3 trial, but results are not yet public. The drug requires daily injections, and its benefits may be modest or not outweigh the burden of treatment.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

102 people

The number who actually took part.

Started

Apr 2022

Finished

Dec 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 70 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: A subject must meet all of the following inclusion criteria at the Screening and Baseline Visit (unless otherwise specified) to be eligible for inclusion in the SPIMD-301 trial: 1. Willing and able to provide a signed informed consent form (ICF) prior to participation in any trial-related procedures. 2. Agrees and is able to adhere to the trial requirements for the length of the trial, including administration of assigned treatment. 3. Is ≥18 years and ≤ 70 years of age at the time of screening. 4. Diagnosed with nPMD with a predominant clinical manifestation of myopathy, which must include progressive external ophthalmoplegia (PEO) and exercise intolerance and/or skeletal muscle weakness, with genetic confirmation of either: 1. Nuclear DNA mutation of the mitochondrial replisome (replisome-related mutations), which include the following genes: * POLG 1/2 * TWINKLE (C10ORF2) * TYMP * DGUOK * TK2 * RRM2B * RNASEH1 * SSBP * MGME1 * DNA2 * ANT1 (SLC25A4) * SUCLG1 * SUCLA2 * MPV17 or 2. Other pathogenic mutations specific to nuclear DNA. 5. Women of childbearing potential must agree to use one of the following methods of birth control from the date they sign the ICF until 28 days after the last dose of IMP: 1. Abstinence, when it is in line with the preferred and usual lifestyle of the subject. Subject agrees to use a highly effective method of contraception should they become sexually active. 2. Relationships with male partners who have been surgically sterilized by vasectomy (the vasectomy procedure must have been conducted at least 60 days prior to the Screening Visit). 3. Barrier method (e.g., condom or occlusive cap) with spermicidal foam/gel/film/cream AND either hormonal contraception (oral, implanted, or injectable) or an intrauterine device or system. Note: Non-childbearing potential is defined as surgical sterilization (e.g., bilateral oophorectomy, hysterectomy, or tubal ligation) or postmenopausal (defined as permanent cessation of menstruation for at least 12 consecutive months prior to the Screening Visit). 6. Male subjects with female partners of childbearing potential must be willing to use a highly effective method of contraception from the date they sign the ICF until 28 days after the last dose of IMP. Exclusion Criteria: 1. Is unable to perform the 6MWT, 3TUG, or 5XSST functional tests. The use of a gait assist device is allowed; however, use should remain consistent for the entire duration of the trial. 2. Female subjects who are pregnant, planning to become pregnant, or breastfeeding/lactating. 3. Walks \< 150 meters or \> 450 meters during the 6MWT (Screening Visit only). 4. The estimated glomerular filtration rate (eGFR) is \< 30 mL/min/1.73 m2, using the Modification of Diet in Renal Disease (MDRD) Study equation (Screening Visit only). 5. Has undergone an in-patient hospitalization within 30 days prior to screening or has a planned hospitalization or a surgical procedure during the trial, unless, in the opinion of the Investigator, it is concluded that it will not impact the outcome measurements of the trial. 6. Has clinically significant respiratory disease and/or cardiac disease that would interfere with trial assessments, in the opinion of the Investigator. 7. Has had any prior interventional cardiac procedure (e.g., cardiac catheterization, angioplasty/percutaneous coronary intervention, balloon valvuloplasty, etc.) within 3 months prior to screening. 8. Has history of or current severe neurologic impairment, severe epilepsy, severe ataxia, or severe neuropathy that may interfere with their ability to complete all trial requirements, in the opinion of the Investigator. 9. Active malignancy or any other cancer from which the subject has been disease-free for \< 2 years. Localized squamous or non-invasive basal cell skin carcinomas are allowed, if appropriately treated prior to screening. 10. Has had a solid organ transplant. 11. Has been previously diagnosed with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection. 12. Has a history of a systemic eosinophilic illness and/or an eosinophil count \>1,000 cells x106/L at the Screening Visit. 13. Is currently participating or has participated in an interventional clinical trial (i.e., investigational product or device, stem cell therapy, gene therapy) within 30 days prior to current trial; or is currently enrolled in a non-interventional clinical trial that, in the opinion of the Investigator, may be potentially confounding to the results of the current trial (e.g., exercise therapy trial). 14. Has received elamipretide (MTP-131) within the past one year of the Screening Visit. 15. Has a history of active substance abuse during the year prior, in the opinion of the Investigator.

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Conditions

The condition(s) this trial relates to.

inborn mitochondrial metabolism disorder inborn mitochondrial myopathy mitochondrial complex I deficiency mitochondrial disease mitochondrial DNA depletion syndrome Muscle Weakness progressive external ophthalmoplegia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Mitochondrial complex I deficiency Mitochondrial diseases Mitochondrial DNA deletion Mitochondrial DNA depletion Mitochondrial DNA mutation Mitochondrial metabolism defect Mitochondrial myopathies Mitochondrial pathology

Contacts and locations

Locations

  • Akron Children's Hospital

    Akron, Ohio, 44308, United States

  • Azienda Ospedaliero Universitaria Policlinico G. Martino

    Messina, 98125, Italy

  • Azienda Ospedaliero Universitario Pisana, Dipartimento Ambientale di Neuroscienze

    Pisa, 56126, Italy

  • Calvary Health Care Bethlehem

    Parkdale, Victoria, 3162, Australia

  • Columbia University Medical Center College of Physician and Surgeon

    New York, New York, 10032, United States

  • Department of Neurology, University Clinics Munich

    Munich, Bavaria, 80336, Germany

  • Helse Bergen HF

    Bergen, 5021, Norway

  • Hospital Clinic de Barcelona

    Barcelona, 8036, Spain

  • Hospital Universitario 12 de Octubre

    Madrid, 28041, Spain

  • Hospital de la Sta Creu i Sant Pau

    Barcelona, 8025, Spain

  • Hospital la Fe de Valencia

    Valencia, 46026, Spain

  • IRCCS Institute of Neorological Sciences of Bologna Bellaria Hospital

    Bologna, Italy

  • Istituto Nazionale Neurologico Carlo Besta

    Milan, 20133, Italy

  • Istituto di Neurologia, Fondazione Policlinico Universitario A. Gemelli, Università Cattolica del Sacro Cuore

    Rome, Lazio, 00168, Italy

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Newcastle upon Tyne Hospitals Freeman Hospital

    Newcastle, NE77DN, United Kingdom

  • Queen Square Centre for Neuromuscular Diseases The National Hospital for Neurology and Neurosurgery

    London, WC1N 3BG, United Kingdom

  • Radboud University Medical Center

    Nijmegen, 6525, Netherlands

  • Rare Disease Research, LLC

    Atlanta, Georgia, 30329, United States

  • Royal North Shore Hospital Neurology

    Sydney, New South Wales, 2065, Australia

  • Semmelweis Egyetem Genomikai Medicina es Ritka Betegsegek

    Budapest, 1083, Hungary

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • UT Health,Center for the Treatment of Pediatric Neurodegenerative Disease

    Houston, Texas, 77030, United States

  • Univ of Tubingen, Hertie Institute for Clinical Brain Research

    Tübingen, Germany

  • Universitaetsklinikum Carl Gustav Carus Dresden Neurologie

    Dresden, Germany

  • University of Auckland - Auckland City Hospital, Neurology Department

    Auckland, 1023, New Zealand

  • University of Brescia, NeMO Clinical Center for Neuromuscular Diseases

    Gussago, Brescia, 25064, Italy

  • University of California, San Diego

    San Diego, California, 92093, United States

  • University of Cambridge, Department of Clinical Neurosciences

    Cambridge, CB2 0QQ, United Kingdom

  • University of Pittsburgh School of Medicine Children's Hospital of Pittsburgh of UPMC Department of Genetics

    Pittsburgh, Pennsylvania, 15224, United States

  • University of Pécs, Department of Neurology Klinikai Kozpont - neurologiai Klinika

    Pécs, 7624, Hungary

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

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