Elamipretide for Adult Patients with Primary Mitochondrial Disease



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The goal of this study is to evaluate the safety and efficacy of an investigational drug called "Elamipretide" in adult patients with mitochondrial disease. "Investigational" means the study drug has not been approved by the U.S. Food and Drug Administration (FDA) and its use in this study is experimental.

The study will last for approximately 13 months and will include at least 7 outpatient visits at CHOP, where participants will be provided the study drug (randomized to active drug or placebo) and monitored for safety.  The clinical trial also involves blood and urine tests, cardiac tests, physical exercise tests, and other study procedures. For more information: https://clinicaltrials.gov/ct2/show/NCT05162768

You will also receive travel support and reimbursement for related expenses.

Who Do I Contact?

If you are interested in participating in the study or want to learn more please contact our study team at nguyens2 [at] chop.edu or 267-426-0225.

Eligibility & Criteria

IRB #:
Official 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)
Study Phase:
Phase III
Eligible Age Range:
18 - 70 Years
Study Categories:

Visit Criteria

The study will require at least 7 outpatient visits at CHOP; procedures include:

  • Medical history review; physical examinations
  • Cardiac tests
  • Physical exercise tests
  • Blood and urine tests
  • Study questionnaires
  • Randomized to receive daily study drug (elamipretide) or placebo


Marni Falk, MD

Amy Goldstein