Phase 3 trial to evaluate subcutaneous elamipretide injections for PMM
Research type
Research Study
Full title
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
IRAS ID
239210
Contact name
Gráinne Gorman
Contact email
Sponsor organisation
Stealth BioTherapeutics Inc.
Eudract number
2017-002447-15
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
123,553, IND Number:
Duration of Study in the UK
3 years, 10 months, 29 days
Research summary
This is a follow-up interventional-research study in patients with primary mitochondrial myopathy (PMM) from the SPIMM-300 non-interventional study.
Primary mitochondrial diseases (PMD) are genetic disorders of the mitochondria, which are the part of cells responsible for respiration, producing the energy required for normal cellular function. PMM is a subtype of PMD affecting predominantly, but not exclusively, skeletal muscle. PMD has been estimated to affect approximately 1 in 4,300-8,000 persons with varying degrees of disability. There are currently no approved treatments for PMM.
The study drug elamipretide (MTP-131) has been shown to improve the function of mitochondria, which is dysfunctional in PMM. The purpose of this study is to gather information about how well elamipretide dosed daily as a subcutaneous injection (SC; under the skin) with an investigational pen injector device (‘Elamipretide pen injector’) works in patients with PMM and to learn more about long-term safety.
Approximately 200 participants aged 16 to 80 years will take part in this research study at approximately 30 different research sites around the world.
Total participation in the study is expected to last approximately 3 years and 6 months. Part 1 is a 6-month treatment period in which participants will be randomly assigned to either study drug or placebo daily (50% chance of receiving study drug). There will be up to 6 visits to the study centre in Part 1. Part 2 is a long-term follow-up period where all participants will receive elamipretide and lasts approximately 3 years. There will be up to 9 visits to the study centre and 4 phone calls from the study team in Part 2. Study tests include blood and urine testing, physical examinations, electrocardiograms, a timed walking test to examine the extent of exercise tolerance and fatigue (6 Minute Walk Test; 6MWT) and completion of questionnaires.
REC name
HSC REC B
REC reference
18/NI/0084
Date of REC Opinion
22 Jun 2018
REC opinion
Further Information Favourable Opinion