A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

PHASE3RecruitingINTERVENTIONAL
Enrollment

200

Participants

Timeline

Start Date

February 14, 2022

Primary Completion Date

April 30, 2026

Study Completion Date

April 30, 2026

Conditions
Phenylketonuria
Interventions
DRUG

PTC923

PTC923 powder for oral use will be suspended in water or apple juice prior to administration.

Trial Locations (45)

1000

RECRUITING

Univerzitetni klinicni center Ljubljana, Ljubljana

2145

RECRUITING

Westmead Hospital, Westmead

3052

RECRUITING

Royal Children's Hospital, Parkville

10009

RECRUITING

Icahn School of Medicine at Mount Sinai (ISMMS), New York

15224

RECRUITING

UPMC Children's Hospital of Pittsburgh, Pittsburgh

20246

RECRUITING

University Children's Hospital Hamburg Eppendorf (Kinder-UKE) Klinik für Kinder- und Jugendmedizin (Kinder-UKE), Hamburg

28034

RECRUITING

Hospital Universitario Ramón y Cajal, Madrid

32610

RECRUITING

UF College of Medicine, Department of Pediatrics Division of Genetics and Metabolism, Gainesville

34098

RECRUITING

İstanbul Üniversitesi Cerrahpaşa Tıp Fakültesi, Fatih

35100

RECRUITING

Ege University Faculty of Medicine Children Hospital, Bornova

35129

RECRUITING

Azienda Ospedaliera-Universita Padova, Padua

41013

RECRUITING

Hospital Universitario Virgen del Rocío, Seville

44670

RECRUITING

PanAmerican Clinical Research, Guadalajara

46202

RECRUITING

Indiana University School of Medicine Department of Medical and Molecular Genetics, Indianapolis

48149

RECRUITING

Universitätsklinikum Münster, Münster

69120

RECRUITING

Universitätsklinikum Heidelberg / Zentrum für Kinder- und Jugendmedizin / Sektion für Neuropädiatrie & Stoffwechselmedizin, Heidelberg

75015

RECRUITING

Hôpital Necker-Enfants Malades - Centre de Référence des Maladies Héréditaires du Métabolisme, Paris

80045

RECRUITING

Children's Hospital Colorado, Aurora

84108

RECRUITING

University of Utah, Salt Lake City

02115

ACTIVE_NOT_RECRUITING

Boston Children's Hospital, Boston

SA 5000

RECRUITING

PARC Clinical Research, Adelaide

90035-903

RECRUITING

Hospital de clinicas de Porto Alegre, Porto Alegre

14051-140

RECRUITING

Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo, Ribeirão Preto

T2E 7Z4

RECRUITING

Metabolics and Genetics in Calgary (MAGIC) Clinic, Ltd., Calgary

L8N 3Z5

RECRUITING

McMaster Children's Hospital Hamilton Health Sciences, Hamilton

M5G 1X8

RECRUITING

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

100 34

RECRUITING

Fakultní nemocnice Královské Vinohrady Klinika dětí a dorostu Ambulance pro léčbu PKU a HPA, Prague

DK-2100

RECRUITING

Copenhagen University Hospital, Rigshospitalet, Copenhagen

0159

RECRUITING

Medical Genetics and Laboratory Diagnostics Center, Tbilisi

00185

RECRUITING

Department of Human Neuroscience, Child and Adolescent Neuropsychiatry, Policlinico Umberto I, Rome

Unknown

RECRUITING

PTC Clinical Site 1, Multiple Locations

RECRUITING

PTC Clinical Site 2, Multiple Locations

RECRUITING

PTC Clinical Site 3, Multiple Locations

RECRUITING

UNIVERSITY HOSPITALS BIRMINGHAM NHS Foundation Trust, Birmingham

9713 GZ

RECRUITING

UMCG Beatrix Children's Hospital, Groningen

71-252

RECRUITING

Pomorski Uniwersytet Medyczny w Szczecinie Centrum Wsparcia Badań Klinicznych, Szczecin

1649-035

RECRUITING

CENTRO HOSPITALAR UNIVERSITÁRIO LISBOA NORTE Hospital de Santa Maria,, Lisbon

RECRUITING

CENTRO HOSPITALAR UNIVERSITÁRIO LISBOA NORTE Hospital de Santa Maria, Lisbon

4099-001

RECRUITING

Centro Hospitalar Universitário Do Porto, Epe, Porto

08950

RECRUITING

Hospital Sant Joan de Déu, Barcelona

06230

RECRUITING

Hacettepe University Medical Faculty, Altındağ

06500

RECRUITING

Gazi Üniversitesi Tıp Fakültesi, Yenimahalle

01330

RECRUITING

Çukurova Üniversitesi Tıp Fakültesi Balcalı Hastanesi, Adana

B4 6NH

RECRUITING

Birmingham Children's Hospital NHS Foundation Trust, Birmingham

WC1N 3JH

RECRUITING

Great Ormond Street Hospital, London

Sponsors

Lead Sponsor

All Listed Sponsors
lead

PTC Therapeutics

INDUSTRY

NCT05166161 - A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria | Biotech Hunter | Biotech Hunter