Phase 3 Randomized Trial to Evaluate Subcutaneous Elamipretide Injections for PMD
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 Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD)
IRAS ID
1004666
Contact name
Hess Suh
Contact email
Sponsor organisation
Stealth BioTherapeutics Inc.
Eudract number
2021-003907-16
ISRCTN Number
ISRCTN92370847
Clinicaltrials.gov Identifier
Research summary
This is a randomised, double-blind, parallel-group, placebo-controlled trial that will enrol approximately 130 participants In the whole Clinical Trial. Stealth BioTherapeutics Inc. have begun a study of an investigational drug called elamipretide as a possible treatment for nPMD (Nuclear DNA Primary Mitochondrial Disease). Elamipretide is a molecule which targets the inner lining (membrane) of mitochondria where energy production occurs and normalises its structure and function leading to overall improvement in the function of the cell and organ. The main goal of this study is to learn how well the study drug works and how safe the study drug is compared with placebo. A placebo is an inactive material that looks like the study drug but does not have any active study drug within it.
REC name
East Midlands - Leicester Central Research Ethics Committee
REC reference
22/EM/0058
Date of REC Opinion
3 May 2022
REC opinion
Further Information Favourable Opinion