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

NCT05162768 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 102

Last updated 2025-10-24

No results posted yet for this study

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).

Conditions

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

Interventions

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

Sponsors & Collaborators

  • Stealth BioTherapeutics Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-04-29
Primary Completion
2024-09-30
Completion
2024-12-04
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Germany
  • Hungary
  • Italy
  • Netherlands
  • New Zealand
  • Norway
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05162768 on ClinicalTrials.gov