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Elamipretide for Adult Patients with Primary Mitochondrial Disease
This study is now recruiting.
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.
Eligibility & 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