Status:
TERMINATED
A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension
Lead Sponsor:
Stealth BioTherapeutics Inc.
Conditions:
Primary Mitochondrial Myopathy
Eligibility:
All Genders
16-80 years
Phase:
PHASE3
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 p...
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) admi...
Eligibility Criteria
Inclusion
- PART 1:
- 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
- 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.
- Subject has not permanently discontinued the elamipretide delivery system.
Key Trial Info
Start Date :
October 9 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 10 2020
Estimated Enrollment :
218 Patients enrolled
Trial Details
Trial ID
NCT03323749
Start Date
October 9 2017
End Date
February 10 2020
Last Update
January 24 2022
Active Locations (27)
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1
University of California San Diego
La Jolla, California, United States, 92093
2
Stanford University
Palo Alto, California, United States, 94304
3
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
4
Rare Disease Research, LLC
Atlanta, Georgia, United States, 30318