A Phase 3 Randomized, 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
Overview
- Phase
- Phase 3
- Intervention
- Not specified
- Conditions
- Primary Mitochondrial Myopathy
- Sponsor
- Stealth BioTherapeutics Inc.
- Enrollment
- 218
- Locations
- 27
- Primary Endpoint
- Total Fatigue Score on the on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
- Status
- Terminated
- Last Updated
- 4 years ago
Overview
Brief Summary
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.
Detailed Description
Part 11 is a 24-week, randomized, double-blind, parallel-group, placebo-controlled assessment of the efficacy and safety of single daily subcutaneous (SC) doses of 40 mg elamipretide (vs placebo) administered with the elamipretide delivery system as a treatment for subjects with primary mitochondrial myopathy (PMM). Part 2 was to assess the long-term safety and tolerability of single daily SC doses of 40 mg elamipretide administered with the elamipretide delivery system for up to 144 weeks.
Investigators
Eligibility Criteria
Inclusion Criteria
- •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
Exclusion Criteria
- •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
Outcomes
Primary Outcomes
Total Fatigue Score on the on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
Time Frame: Baseline to 24 weeks
Change from Baseline in Total fatigue score on the on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) by visit. Each individual item score ranges from 1 (none) to 4 (severe). The total fatigue score ranges from 4-16. Lower values represent a better outcome. The total fatigue score is the sum of question 1 through question 4 on the Primary Mitochondrial Myopathy Symptom Assessment.
Six-minute Walk Test (6MWT)
Time Frame: Baseline to 24 weeks
Change From Baseline in Distance Walked (meters) on the Six-Minute Walk Test by Visit
Secondary Outcomes
- Change From Baseline in the Most Bothersome Symptom Score on the Primary Mitochondrial Myopathy Symptoms Assessment(Baseline to 24 weeks)
- Fatigue During Activities Score on the Primary Mitochondrial Disease Symptom Assessment (PMMSA).(Baseline to 24 weeks)
- Neuro-QoL Fatigue Activities of Daily Living(Baseline to 24 weeks)
- Neuro-QoL Fatigue Short Form Score(24 Weeks)