TerminatedPHASE2, PHASE3NCT04378075
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
Studying Leigh syndrome
Last synced from ClinicalTrials.gov
ℹ
Clinical trial records are synced from ClinicalTrials.gov through automated extraction.
Report missing dataKey facts
- Sponsor
- PTC Therapeutics
- Principal Investigator
- Vinay Penematsa, MDPTC Therapeutics
- Intervention
- Vatiquinone(drug)
- Enrollment
- 68 enrolled
- Eligibility
- 20 years · All sexes
- Timeline
- 2020 – 2023
Study locations (27)
- University of California, San Diego, California, United States
- Stanford University, Stanford, California, United States
- Yale School of Medicine, New Haven, Connecticut, United States
- Children's National Medical Center - Department Of Neurology, Washington D.C., District of Columbia, United States
- John Hopkins Medicine, Baltimore, Maryland, United States
- Pediatric Genetics Clinic (Main MGH Hospital), Boston, Massachusetts, United States
- Boston Children Hospital, Boston, Massachusetts, United States
- Children's of Minnesota, Minneapolis, Minnesota, United States
- Akron Children's Hospital, Akron, Ohio, United States
- Baylor College of Medicine, Houston, Texas, United States
- University of Texas Health Science, Houston, Texas, United States
- Seattle Children's hospital, Seattle, Washington, United States
- Alberta Children's Hospital, University of Calgary, Calgary, Canada
- CHU d'Angers - Service de génétique, Angers, France
- CHU de Montpellier - Hôpital Gui de Chauliac - Département de neuropédiatrie, Montpellier, France
- +12 more locations on ClinicalTrials.gov
Primary source
Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.
Open NCT04378075 on ClinicalTrials.govOther trials for Leigh syndrome
Additional recruiting or active studies for the same condition.
- ENROLLING BY INVITATIONPHASE2NCT06843811Sirolimus for Leigh SyndromeMatthew Demczko
- RECRUITINGNCT06967831Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural CellsCharite University, Berlin, Germany
- RECRUITINGNANCT03137355The International Registry for Leigh SyndromeThe University of Texas Health Science Center, Houston
- RECRUITINGNCT01780168The NIH MINI Study: Metabolism, Infection, and Immunity in Inborn Errors of MetabolismNational Human Genome Research Institute (NHGRI)