New PKU treatment could ease strict diet rules
NCT ID NCT05166161
Summary
This study is testing the long-term safety of an investigational medication called PTC923 for people with phenylketonuria (PKU). It involves 200 participants and aims to see if the treatment allows people to safely eat more protein while keeping their blood phenylalanine levels in a safe range. The study also measures quality of life and how easy the medication is to take.
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Contacts and locations
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Locations
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Azienda Ospedaliera-Universita Padova
Padua, Veneto, 35129, Italy
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Birmingham Children's Hospital NHS Foundation Trust
Birmingham, B4 6NH, United Kingdom
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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CENTRO HOSPITALAR UNIVERSITÁRIO LISBOA NORTE Hospital de Santa Maria
Lisbon, Estremadura, 1649-035, Portugal
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CENTRO HOSPITALAR UNIVERSITÁRIO LISBOA NORTE Hospital de Santa Maria,
Lisbon, Estremadura, 1649-035, Portugal
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Centro Hospitalar Universitário Do Porto, Epe
Porto, 4099-001, Portugal
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Copenhagen University Hospital, Rigshospitalet
Copenhagen, DK-2100, Denmark
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Department of Human Neuroscience, Child and Adolescent Neuropsychiatry, Policlinico Umberto I
Rome, 00185, Italy
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Ege University Faculty of Medicine Children Hospital
Bornova, İzmir, 35100, Turkey (Türkiye)
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Fakultní nemocnice Královské Vinohrady Klinika dětí a dorostu Ambulance pro léčbu PKU a HPA
Prague, 100 34, Czechia
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Gazi Üniversitesi Tıp Fakültesi
Yenimahalle, Ankara, 06500, Turkey (Türkiye)
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Great Ormond Street Hospital
London, WC1N 3JH, United Kingdom
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Hacettepe University Medical Faculty
Altındağ, Ankara, 06230, Turkey (Türkiye)
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Hospital Sant Joan de Déu
Barcelona, Esplugues de Llobregat, 08950, Spain
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Hospital Universitario Ramón y Cajal
Madrid, 28034, Spain
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Hospital Universitario Virgen del Rocío
Seville, 41013, Spain
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Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo
Ribeirão Preto, São Paulo, 14051-140, Brazil
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Hospital de clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, 90035-903, Brazil
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Hôpital Necker-Enfants Malades - Centre de Référence des Maladies Héréditaires du Métabolisme
Paris, 75015, France
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Icahn School of Medicine at Mount Sinai (ISMMS)
New York, New York, 10009, United States
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Indiana University School of Medicine Department of Medical and Molecular Genetics
Indianapolis, Indiana, 46202, United States
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McMaster Children's Hospital Hamilton Health Sciences
Hamilton, Ontario, L8N 3Z5, Canada
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Medical Genetics and Laboratory Diagnostics Center
Tbilisi, 0159, Georgia
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Metabolics and Genetics in Calgary (MAGIC) Clinic, Ltd.
Calgary, Alberta, T2E 7Z4, Canada
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PARC Clinical Research
Adelaide, South Australia, SA 5000, Australia
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PTC Clinical Site 1
Multiple Locations, Japan
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PTC Clinical Site 2
Multiple Locations, Japan
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PTC Clinical Site 3
Multiple Locations, Japan
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PanAmerican Clinical Research
Guadalajara, Jalisco, 44670, Mexico
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Pomorski Uniwersytet Medyczny w Szczecinie Centrum Wsparcia Badań Klinicznych
Szczecin, 71-252, Poland
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Royal Children's Hospital
Parkville, Victoria, 3052, Australia
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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, Ontario, M5G 1X8, Canada
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UF College of Medicine, Department of Pediatrics Division of Genetics and Metabolism
Gainesville, Florida, 32610, United States
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UMCG Beatrix Children's Hospital
Groningen, 9713 GZ, Netherlands
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UNIVERSITY HOSPITALS BIRMINGHAM NHS Foundation Trust
Birmingham, United Kingdom
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UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
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University Children's Hospital Hamburg Eppendorf (Kinder-UKE) Klinik für Kinder- und Jugendmedizin (Kinder-UKE)
Hamburg, 20246, Germany
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University of Utah
Salt Lake City, Utah, 84108, United States
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Universitätsklinikum Heidelberg / Zentrum für Kinder- und Jugendmedizin / Sektion für Neuropädiatrie & Stoffwechselmedizin
Heidelberg, 69120, Germany
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Universitätsklinikum Münster
Münster, 48149, Germany
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Univerzitetni klinicni center Ljubljana
Ljubljana, 1000, Slovenia
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Westmead Hospital
Westmead, New South Wales, 2145, Australia
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Çukurova Üniversitesi Tıp Fakültesi Balcalı Hastanesi
Adana, 01330, Turkey (Türkiye)
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İstanbul Üniversitesi Cerrahpaşa Tıp Fakültesi
Fatih, Istanbul, 34098, Turkey (Türkiye)
Conditions
Explore the condition pages connected to this study.