A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
Completed · Phase 3 · NCT05218655
Conditions: Inherited Mitochondrial Disease
At a glance
- Research payments reported, 2025
- $622,478
- U.S. investigators named
- 14
- Sites paid
- 14
- Planned or actual enrollment
- 101
About the trial
- Sponsor
- PTC Therapeutics
- Tested
- Vatiquinone (drug)
- Dates
- Started 2022-06-22; completion 2025-04-15
- Results posted
- Yes
The primary objective of this study is to assess the safety of vatiquinone in participants with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron sponsored (previously Edison) clinical study or treatment plan. The study will continue until vatiquinone becomes commercially available or the program is terminated.
From ClinicalTrials.gov, last updated 2025-12-22. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Ptc Therapeutics, Inc. reported $622,478 in research payments for this study in 2025 (75 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
- Richard H. Haas, M.D. Child Neurology · San Diego, CA
- Michele Spencer-manzon, M.D. Medical Genetics · New Haven, CT
- John M. Schreiber, MD Child Neurology · Washington, DC
- James B. Renfroe, MD Child Neurology · Gulf Breeze, FL
- Hilary J. Vernon, M.D., PH.D. Pediatrics · Baltimore, MD
- Michio Hirano, M.D. Neurology · New York, NY
- Bruce H. Cohen, M.D. Child Neurology · Akron, OH
- Sumit Parikh, MD Child Neurology · Cleveland, OH
- David R. Lynch Child Neurology · Philadelphia, PA
- Thomas K. Koch, MD Child Neurology · Charleston, SC
- Mary Kay Koenig, M.D. Child Neurology · Houston, TX
- Fernando Scaglia, MD Medical Genetics · Houston, TX
- Russ P. Saneto Child Neurology · Seattle, WA
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Baylor College of Medicine $184,204
- Children's Hospital of Philadelphia, PA $86,225
- Seattle Children's Hospital, WA $85,422
- Akron Children's Hospital, OH $66,038
- University of Texas Health Science Center at Houston $51,458
- The Board of Trustees of the Leland Stanford Junior University $28,678
- Childrens Research Institute $26,322
- The Trustees of Columbia University in The City of New York $18,991
- Johns Hopkins University $18,040
- Regents of the University of California $15,299
- Medical University of South Carolina $15,045
- Northwest Florida Clinical Research Group LLC $11,869
- Yale University $11,788
- Cleveland Clinic, OH $3,101
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.