New drug aims to help mitochondrial disease patients walk farther
NCT ID NCT05162768
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.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for MITOCHONDRIAL MYOPATHIES are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
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