157
Participants
Start Date
September 30, 2021
Primary Completion Date
April 3, 2023
Study Completion Date
May 3, 2023
PTC923
PTC923 powder for oral use will be suspended in water or apple juice prior to administration.
Placebo
Placebo matching to PTC923
Pediatric Surgery Center, Tbilisi
Cukurova Üniversity Balcali Hospital Health Application and Research Center, Adana
Westmead Hospital, Westmead
Royal Melbourne Hospital, Melbourne
Grupo Médico Camino SC, Benito Juárez
Hacettepe University Medical Faculty, Altındağ
Gazi Üniversitesi Tıp Fakültesi, Yenimahalle
Hospital Sant Joan de Déu, Barcelona
UPMC Children's Hospital of Pittsburgh, Pittsburgh
The Children's Hospital of Philadelphia, Philadelphia
University Children's Hospital Hamburg Eppendorf (Kinder-UKE) Klinik für Kinder- und Jugendmedizin (Kinder-UKE), Hamburg
Hospital Universitario Ramón y Cajal, Madrid
UF College of Medicine, Department of Pediatrics Division of Genetics and Metabolism, Gainesville
İstanbul Üniversitesi Cerrahpaşa Tıp Fakültesi, Fatih
Ege University Faculty of Medicine Children Hospital, Bornova
Division of Inherited Metabolic Diseases, Azienda Ospedaliera-Università Padova, Padua
Bretonneau Hospital - CHRU de Tours, Tours
CHRU de Tours- Hôpital Pédiatrique de Clocheville, Tours
PanAmerican Clinical Research, Guadalajara
Indiana University School of Medicine, Indianapolis
Universitätsklinikum Münster, Münster
Medical College of Wisconsin, Milwaukee
Universitätsklinikum Heidelberg / Zentrum für Kinder- und Jugendmedizin / Sektion für Neuropädiatrie & Stoffwechselmedizin, Heidelberg
Children's Medical Center Dallas, Dallas
University of Texas Health Science Center of Texas, Houston
University of Colorado and the Children's Hospital CO, Aurora
University of Utah, Division of Medical Genetics (pediatric and adult clinic), Salt Lake City
Stanford University Center for Academic Medicine, Stanford
Boston Children's Hospital, Boston
Icahn School of Medicine at Mount Sinai (ISMMS), New York
PARC Clinical Research, Adelaide
Hospital de clinicas de Porto Alegre, Porto Alegre
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo, Ribeirão Preto
Metabolics and Genetics in Calgary (MAGIC) Clinic, Ltd., Calgary
The Hospital for Sick Children University of Toronto, Adult Clinic: The Fred A Litwin Family Centre in Genetic Medicine University Health Network & Mt. Sinai Hospital, Toronto
Copenhagen University Hospital, Rigshospitalet, Copenhagen
Policlinico Umberto I, Rome
UMCG Beatrix Children's Hospital, Groningen
Centro Hospitalar Universitário Do Porto, Epe, Porto
CENTRO HOSPITALAR UNIVERSITÁRIO LISBOA NORTE Hospital de Santa Maria,, Lisbon
CENTRO HOSPITALAR UNIVERSITÁRIO LISBOA NORTE Hospital de Santa Maria, Lisbon
Birmingham Children's Hospital NHS Foundation Trust, Birmingham
Great Ormond Street Hospital, London
Lead Sponsor
PTC Therapeutics
INDUSTRY