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Compound Evidence InstituteEvidence synthesis · established 2023Graded assessments of compounds, trials, methods and supply
Document set current to 30 July 2026
Indication assessment · §3

Primary mitochondrial myopathy and bioenergetic disorders — trial records

Trial records in the Institute's register bearing on primary mitochondrial myopathy and bioenergetic disorders.

Document identifier
CEI-IN-31/3
Series
Indication assessment
Version
1.0
Published
07 Dec 2025
Last reviewed
07 Dec 2025
Next review
07 Dec 2027
Identifier
10.71829/cei.ind.31
Certainty
Not rated
Cycle
2025 Q4
ICD-11
8C73
Category
Neuromuscular

§3Trials in this indication

9 trial records in the Institute's register bear on this indication, of which 2 are published trials.

Table 2. Trial records bearing on primary mitochondrial myopathy and bioenergetic disorders.

RecordCompoundPhaseDesignRandomisedYear
MMPOWER-2SS-31 (elamipretide)Phase 2Randomised crossover302018
MMPOWER-3SS-31 (elamipretide)Phase 3Randomised, double-blind, placebo-controlled2182022
MMPOWER-2-ELDERLYprogramme recordSS-31 (elamipretide)Phase 2Randomised, double-blind, placebo-controlled2018
MMPOWER-3-OLEprogramme recordSS-31 (elamipretide)Phase 3Single-arm open-label2022
MMPOWER-2-OLEprogramme recordSS-31 (elamipretide)Phase 2Single-arm open-label2018
MMPOWER-2-ADHERENCEprogramme recordSS-31 (elamipretide)Phase 2Observational2018
MMPOWER-3-BIOMARKERprogramme recordSS-31 (elamipretide)Phase 3Randomised, double-blind, placebo-controlled2022
MMPOWER-2-BIOMARKERprogramme recordSS-31 (elamipretide)Phase 2Randomised, double-blind, placebo-controlled2018
MMPOWER-3-ADHERENCEprogramme recordSS-31 (elamipretide)Phase 3Observational2022
This facet in the trial register.

References cited on this page

References are numbered in order of first citation in this document. Each superscript in the text links to its entry below.

  1. Szeto HH. First-in-class cardiolipin-protective compound as a therapeutic agent to restore mitochondrial bioenergetics. British Journal of Pharmacology 2014;171(8):2029–2050. doi:10.1111/bph.12461 · PMID 24117165

Identifiers are reproduced only where the Institute holds them. Where a digital object identifier or PubMed identifier is not shown, the Institute has recorded the journal and year and has not constructed an identifier.

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