EPI-743 for Mitochondrial Respiratory Chain Diseases
No longer available · NCT01370447
Conditions: Mitochondrial Diseases
At a glance
- Research payments reported, 2025
- $20,659
- U.S. investigators named
- 2
- Sites paid
- 2
About the trial
- Sponsor
- PTC Therapeutics
- Tested
- EPI-743 (drug)
- Results posted
- Not yet
This study will evaluate the safety and efficacy of EPI-743 in participants with severe mitochondrial respiratory chain diseases who are considered to be within 90 days of end-of-life care.
From ClinicalTrials.gov, last updated 2024-01-23. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Ptc Therapeutics, Inc. reported $20,659 in research payments for this study in 2025 (3 payments).
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Gregory M. Enns, M.D. Medical Genetics · Palo Alto, CA
- Hilary J. Vernon, M.D., PH.D. Pediatrics · Baltimore, MD
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Johns Hopkins University $12,293
- The Board of Trustees of the Leland Stanford Junior University $8,366
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.