A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension

Stopped early · Phase 3 · Has a placebo group

Conditions studied: Primary Mitochondrial Myopathy

In brief

This is a multicenter phase 3 randomized, double-blind, parallel-group, placebo-controlled trial to evaluate the safety and efficacy of daily subcutaneous injections of elamipretide in subjects with primary mitochondrial myopathy. This will be followed by an open-label treatment extension.

Key facts

Study ID

NCT03323749

Run by

Stealth BioTherapeutics Inc.

People needed

218

Starts

2017-10-09

Expected to finish

2020-02-10

Last updated by the study team

2022-01-24

Who can join

Age: 16 and older, up to 80. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Willing and able to provide a signed informed consent form prior to participation in any trial-related procedures
• Agrees to adhere to the trial requirements for the length of the trial, including the use of the elamipretide delivery system
• Subject is ≥ 16 and ≤ 80 years of age
• Diagnosed with PMM in the opinion of the investigator and confirmed by an Adjudication Committee
• Woman of childbearing potential must agree to use a highly effective method of birth control

You may not qualify if…

• Subject has myopathic signs and or/symptoms due to a neuropathic process or gait problem that would interfere with the 6 minute walk test (6MWT), in the opinion of the Investigator
• Female who are pregnant, planning to become pregnant, or breastfeeding/lactating
• At Screening, the estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m\^2
• Subject has undergone an in-patient hospitalization within the 30 days prior to the Baseline Visit or has a planned hospitalization or a surgical procedure during the trial.
• Subject has clinically significant cardiac disease or prior interventional procedure and/or respiratory disease (medical history or current clinical findings) within 3 months of the Baseline Visit, in the opinion of the Investigator.
• Subject has QTc elongation (using the correction factor utilized at the clinical site) defined as a QTc >450 msec in male subjects and >480 msec in female subjects.
• ECG evidence of acute ischemia, atrial fibrillation, or active conduction system abnormalities with the exception of any of the following:
• First degree Atrioventricular bock (AV-block)
• Second degree AV-block Type 1 (Mobitz Type 1 / Wenckebach type)
• Right bundle branch block
• Subject has severe vision impairment that, in the opinion of the Investigator, may interfere with their ability to complete all trial requirements
• Subject has a seizure disorder that, in the opinion of the Investigator, may interfere with their ability to complete all trial requirements.
• Active malignancy or any other cancer from which the subject has been disease-free for < 2 years.
• Subject has a solid organ transplant and/or is currently receiving treatment with therapy for immunosuppression, in the opinion of the Investigator.
• Subject has been previously diagnosed with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection.
• Subject has a history of a systemic eosinophilic illness and/or an eosinophil count >1,000 cells x10\^6/L at the Screening Visit.
• Subject 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 of the Baseline Visit; or is currently enrolled in a non-interventional clinical trial (except for SPIMM-300) at the Baseline Visit which, in the opinion of the Investigator, may be potentially confounding with results of the current trial (e.g., exercise therapy trial).
• Subject has previously received elamipretide (MTP-131), for any reason.
• Subject has a history of active substance abuse during the year before the Baseline Visit, in the opinion of the Investigator.
• Subject has any prior or current medical condition that, in the judgment of the Investigator, would prevent the subject from safely participating in and/or completing all trial requirements.
• PART 2:
• Continuation Criteria:
• Subjects must continue to be able and willing to adhere to the trial requirements.
• Subject is appropriate to continue in Part 2 (i.e. subject was compliant in Part 1), in the opinion of the Investigator.
• Subject has not had a serious adverse event (SAE)/serious adverse device effect (SADE) attributed to the elamipretide delivery system.

Where it is running

• University of California San Diego — La Jolla, California, United States
• Stanford University — Palo Alto, California, United States
• Children's Hospital Colorado — Aurora, Colorado, United States
• Rare Disease Research, LLC — Atlanta, Georgia, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Columbia University Medical Center — New York, New York, United States
• Akron Children's Hospital — Akron, Ohio, United States
• Cleveland Clinical Neurological Institute — Cleveland, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Children's Hospital of Pittsburgh of UPMC — Pittsburgh, Pennsylvania, United States
• Baylor College of Medicine/Texas Children's Hospital — Houston, Texas, United States
• University of Texas Health Science Center — Houston, Texas, United States
• Seattle Children's Hospital — Seattle, Washington, United States
• Adult Metabolic Diseases Clinic — Vancouver, British Colombia, Canada
• McMaster University Medical Center — Hamilton, Ontario, Canada
• Copenhagen Neuromuscular Center — Copenhagen, Denmark
• University Hospital of Bonn — Bonn, Germany
• Klinikum der Universität München, Friedrich-Baur Institute — Munich, Germany
• Institute of Genomic Medicine and Rare Disorders — Budapest, Hungary
• IRCCS Institute of Neorological Sciences of Bologna, Bellaria Hospital — Bologna, Italy
• Azienda Ospedaliero Universitaria Policlinico G. Martino — Messina, Italy
• Istituto Nazionale Neurologico Carlo Besta — Milan, Italy
• Dipartimento Ambientale di Neuroscienze — Pisa, Italy
• Ospedale Pediatrico Bambin Gesù — Rome, Italy
• Istituto di Neurologia, Fondazione Policlinico Universitario A. Gemelli — Rome, Italy

Full record on ClinicalTrials.gov

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