A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy

PHASE2/PHASE3TerminatedINTERVENTIONAL
Enrollment

68

Participants

Timeline

Start Date

September 28, 2020

Primary Completion Date

March 18, 2023

Study Completion Date

December 27, 2023

Conditions
Mitochondrial DiseasesDrug Resistant EpilepsyLeigh DiseaseLeigh SyndromeMitochondrial Encephalopathy (MELAS)Pontocerebellar Hypoplasia Type 6 (PCH6)Alpers DiseaseAlpers Syndrome
Interventions
DRUG

Vatiquinone

Vatiquinone will be administered per the treatment arm description.

OTHER

Placebo

Vatiquinone-matching placebo will be administered per the treatment arm description

Trial Locations (27)

20010

Children's National Medical Center - Department Of Neurology, Washington D.C.

20133

UOC Neuropsichiatria Infantile, Istituto Neurologico Carlo Besta-Fondazione IRCCS, Milan

21287

John Hopkins Medicine, Baltimore

28034

Hospital Ruber Internacional, Neurology Department, Epilepsy Program, Madrid

28041

Hospital Universitario 12 de Octubre, Madrid

34295

CHU de Montpellier - Hôpital Gui de Chauliac - Département de neuropédiatrie, Montpellier

44308

Akron Children's Hospital, Akron

49933

CHU d'Angers - Service de génétique, Angers

55404

Children's of Minnesota, Minneapolis

67200

CHU de Strasbourg - Hôpital de Hautepierre - Service de Neuropédiatrie, Strasbourg

75015

A.P.H.P - Hôpital Necker-Enfants Malades - Service de Neurologie pédiatrique, Paris

77030

Baylor College of Medicine, Houston

University of Texas Health Science, Houston

92123

University of California, San Diego

94305

Stanford University, Stanford

98105

Seattle Children's hospital, Seattle

06520

Yale School of Medicine, New Haven

02114-2696

Pediatric Genetics Clinic (Main MGH Hospital), Boston

02115

Boston Children Hospital, Boston

T3B 6A8

Alberta Children's Hospital, University of Calgary, Calgary

00165

U.O.C. Malattie Muscolari e Neurodegenerative, Dipartimento di Scienze Neurologiche e Psichiatriche, Ospedale Pediatrico Bambino Gesù, Roma

Unknown

PTC Clinical Site, Multiple Locations

04-730

Instytut Pomnik-Centrum Zdrowia Dziecka, Centrum Wsparacia Pediatrycznych Badań Klinicznych, Warsaw

08950

Hospital Sant Joan de Déu, Barcelona

S-171 76

Karolinska University hospital, Astrid Lindgrens Children Hospital, Stockholm

WC1N 3JH

Great Ormond Street Hospital for Children NHS Foundation Trust, London

NE1 4LP

The Newcastle Upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne

Sponsors

Lead Sponsor

All Listed Sponsors
lead

PTC Therapeutics

INDUSTRY

NCT04378075 - A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy | Biotech Hunter | Biotech Hunter