NCT05162768

Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)

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) NuPower

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 102 (actual)
• Sponsor: Stealth BioTherapeutics Inc. · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

SPIMD-301 is a 48-week, randomized, double-blind, parallel-group, placebo-controlled trial to assess efficacy and safety of single daily subcutaneous (SC) administration of elamipretide as a treatment for subjects with primary mitochondrial myopathy associated with nuclear DNA mutations (nPMD).

Detailed description

This 48-week randomized, double-blind, parallel-group, placebo-controlled trial will enroll approximately 130 subjects, consisting of 90 subjects who have nPMD associated with pathogenic mutations of the mitochondrial replisome("replisome-related mutations") for primary analysis and an additional subset of up to 40 subjects who have nPMD associated with other non-replisome-related pathogenic mutations specific to the nuclear DNA. Efficacy and safety of single daily SC doses of elamipretide administered as a treatment for subjects who have primary mitochondrial myopathy associated with nPMD will be determined. Subjects will be randomized 1:1 to 60mg Elamipretide or matching placebo groups.

Conditions

• Mitochondrial Myopathies
• Mitochondrial Pathology
• Mitochondrial DNA Mutation
• Mitochondrial Diseases
• Mitochondrial DNA Deletion
• Mitochondrial DNA Depletion
• Mitochondrial Metabolism Defect
• Mitochondrial Complex I Deficiency

Interventions

Type	Name	Description
DRUG	Elamipretide	60 mg of elamipretide administered as once daily 0.75 mL subcutaneous injections for 48 weeks
DRUG	Placebo	Placebo administered as once daily 0.75 mL subcutaneous injections for 48 weeks

Timeline

• Start date: 2022-04-29
• Primary completion: 2024-09-30
• Completion: 2024-12-04
• First posted: 2021-12-17
• Last updated: 2025-10-24

Locations

32 sites across 10 countries: United States, Australia, Germany, Hungary, Italy, Netherlands, New Zealand, Norway, Spain, United Kingdom

Regulatory

• FDA-regulated drug study