A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondrial Myopathy Followed by an Open-Label Treatment Extension

Stealth BioTherapeutics Inc.0 sites218 target enrollmentFebruary 16, 2018

ConditionsPrimary Mitochondrial Myopathy MedDRA version: 20.0Level: PTClassification code 10027710Term: Mitochondrial myopathySystem Organ Class: 10010331 - Congenital, familial and genetic disorders Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Primary Mitochondrial Myopathy
Sponsor: Stealth BioTherapeutics Inc.
Enrollment: 218
Status: Active, not recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

February 16, 2018

End Date

February 10, 2020

Last Updated

last year

Study Type

Interventional clinical trial of medicinal product

Sex

All

Investigators

Sponsor

Stealth BioTherapeutics Inc.

Eligibility Criteria

Inclusion Criteria

•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 the use of the elamipretide delivery system.
•3\. Subject is \=16 and \=80 years of age. In Germany, subjects must be \=18 years of age.
•4\. Enrolled (signed ICF) in SPIMM\-300 or have prior approval from the Sponsor to enroll without SPIMM\-300 participation.
•5\. Diagnosed with PMM in the opinion of the Investigator, consisting of:
•a. Molecular genetic abnormality of the mitochondrial respiratory chain, and
•b. Subject reported symptoms (i.e., exercise intolerance, fatigue, muscle weakness) or physical examination findings of myopathy that are the predominant symptoms of the subject’s mitochondrial respiratory chain disorder.
•6\. The subject’s molecular genetic abnormality is consistent with PMM as confirmed by the Adjudication Committee.
•7\. Women of childbearing potential must agree to use 1 of the following methods of birth control from the date they sign the ICF until 28 days after the last dose of IMP:
•a. 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.

Exclusion Criteria

•1\. Subject has myopathic signs and/or symptoms due to a neuropathic process (i.e. cerebellar dysfunctions and peripheral neuropathies) or a gait problem that would interfere with the 6MWT, in the opinion of the Investigator.
•2\. Female subjects who are pregnant, planning to become pregnant, or breastfeeding/lactating.
•3\. Walks \< 100 meters or \> 450 meters during the 6MWT at either the Screening Visit OR Baseline Visit.
•4\. At the Baseline Visit, the estimated glomerular filtration rate (eGFR) \< 30 mL/min/1\.73 m2, using the Screening Visit value with the Modification of Diet in Renal Disease (MDRD) Study equation.
•5\. 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.
•6\. Subject has clinically significant respiratory disease and/or cardiac disease (medical history or current clinical findings), in the opinion of the Investigator, or prior interventional cardiac procedure (e.g., cardiac catheterization, angioplasty/percutaneous coronary intervention, balloon valvuloplasty, etc.) within 3 months of the Baseline Visit.
•7\. Subject has a pacemaker, implantable cardioverter\-defibrillator, or cardiac resynchronization therapy device OR 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.
•Note: At the initial electrocardiogram (ECG), if QTc exceeds these parameters, the ECG may be repeated 2 more times (during the same visit), and the average of the 3 QTc values used to determine the subject’s eligibility.
•8\. ECG evidence of acute ischemia, atrial fibrillation, or active conduction system abnormalities with the exception of any of the following:
•a. First degree AV\-block