A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

PHASE3CompletedINTERVENTIONAL
Enrollment

102

Participants

Timeline

Start Date

June 21, 2022

Primary Completion Date

April 15, 2025

Study Completion Date

April 15, 2025

Conditions
Inherited Mitochondrial Disease
Interventions
DRUG

Vatiquinone

Vatiquinone will be administered per dose and schedule specified in the arm description.

Trial Locations (26)

10032

Columbia University Medical Center - CUMC, New York

19104

Children's Hospital of Philadelphia - CHOP, Philadelphia

20010

Children's National, Washington D.C.

21205

The Johns Hopkins University, Baltimore

28003

Hospital Universitario 12 de Octubre, Madrid

28034

Hospital Ruber Internacional, Madrid

29425

Medical University of South Carolina - MUSC, Charleston

32561

Child Neurology Center of Northwest Florida, Gulf Breeze

34295

CHU Montpellier - Hopital Saint-Eloi, Montpellier

44195

Cleveland Clinic, Cleveland

44308

Akron Children's Hospital, Akron

49933

Chu Angers, Angers

67000

Hôpital de Hautepierre - Hôpitaux Universitaires de Strasbourg, Strasbourg

75015

Hopital Necker-Enfants Malades, Paris

77030

Baylor College of Medicine, Houston

UT Health The University of Texas, Houston

92037

University of California, San Diego Altman Clinical and Translational Research Institute, La Jolla

94305

Stanford University, Stanford

98105

Seattle Children Hospital, Seattle

06511

Yale Medicine, New Haven

00165

Ospedale Pediatrico Bambino Gesù, Roma

Unknown

PTC Clinical Site, Japanese City

04-730

Instytut Pomnik - Centrum Zdrowia Dziecka, Warsaw

08950

Hospital Sant Joan de Déu, Barcelona

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

NCT05218655 - A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease | Biotech Hunter | Biotech Hunter