A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
Terminated · Phase 2, Phase 3 · NCT04378075
Conditions: Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome, Mitochondrial Encephalopathy (MELAS), Pontocerebellar Hypoplasia Type 6 (PCH6), Alpers Disease, Alpers Syndrome
At a glance
- Research payments reported, 2025
- $20,215
- U.S. investigators named
- 2
- Sites paid
- 2
- Planned or actual enrollment
- 68
About the trial
- Sponsor
- PTC Therapeutics
- Tested
- Vatiquinone (drug), Placebo (other)
- Dates
- Started 2020-09-28; completion 2023-12-27
- Results posted
- Yes
This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase. After completion of the randomized, placebo-controlled phase, participants may enter a 48-week, long-term, extension phase during which they will receive open-label treatment with vatiquinone.
From ClinicalTrials.gov, last updated 2026-03-31. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Ptc Therapeutics, Inc. reported $20,215 in research payments for this study in 2025 (2 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
- Hilary J. Vernon, M.D., PH.D. Pediatrics · Baltimore, MD
- Russ P. Saneto Child Neurology · Seattle, WA
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Johns Hopkins University $16,312
- Seattle Children's Hospital, WA $3,903
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.