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

Locations

  • Azienda Ospedaliera-Universita Padova

    Padua, Veneto, 35129, Italy

  • Birmingham Children's Hospital NHS Foundation Trust

    Birmingham, B4 6NH, United Kingdom

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • CENTRO HOSPITALAR UNIVERSITÁRIO LISBOA NORTE Hospital de Santa Maria

    Lisbon, Estremadura, 1649-035, Portugal

  • CENTRO HOSPITALAR UNIVERSITÁRIO LISBOA NORTE Hospital de Santa Maria,

    Lisbon, Estremadura, 1649-035, Portugal

  • Centro Hospitalar Universitário Do Porto, Epe

    Porto, 4099-001, Portugal

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Copenhagen University Hospital, Rigshospitalet

    Copenhagen, DK-2100, Denmark

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

    Rome, 00185, Italy

  • Ege University Faculty of Medicine Children Hospital

    Bornova, İzmir, 35100, Turkey (Türkiye)

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

    Prague, 100 34, Czechia

  • Gazi Üniversitesi Tıp Fakültesi

    Yenimahalle, Ankara, 06500, Turkey (Türkiye)

  • Great Ormond Street Hospital

    London, WC1N 3JH, United Kingdom

  • Hacettepe University Medical Faculty

    Altındağ, Ankara, 06230, Turkey (Türkiye)

  • Hospital Sant Joan de Déu

    Barcelona, Esplugues de Llobregat, 08950, Spain

  • Hospital Universitario Ramón y Cajal

    Madrid, 28034, Spain

  • Hospital Universitario Virgen del Rocío

    Seville, 41013, Spain

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

    Ribeirão Preto, São Paulo, 14051-140, Brazil

  • Hospital de clinicas de Porto Alegre

    Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

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

    Paris, 75015, France

  • Icahn School of Medicine at Mount Sinai (ISMMS)

    New York, New York, 10009, United States

  • Indiana University School of Medicine Department of Medical and Molecular Genetics

    Indianapolis, Indiana, 46202, United States

  • McMaster Children's Hospital Hamilton Health Sciences

    Hamilton, Ontario, L8N 3Z5, Canada

  • Medical Genetics and Laboratory Diagnostics Center

    Tbilisi, 0159, Georgia

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

    Calgary, Alberta, T2E 7Z4, Canada

  • PARC Clinical Research

    Adelaide, South Australia, SA 5000, Australia

  • PTC Clinical Site 1

    Multiple Locations, Japan

  • PTC Clinical Site 2

    Multiple Locations, Japan

  • PTC Clinical Site 3

    Multiple Locations, Japan

  • PanAmerican Clinical Research

    Guadalajara, Jalisco, 44670, Mexico

  • Pomorski Uniwersytet Medyczny w Szczecinie Centrum Wsparcia Badań Klinicznych

    Szczecin, 71-252, Poland

  • Royal Children's Hospital

    Parkville, Victoria, 3052, Australia

  • 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

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

    Gainesville, Florida, 32610, United States

  • UMCG Beatrix Children's Hospital

    Groningen, 9713 GZ, Netherlands

  • UNIVERSITY HOSPITALS BIRMINGHAM NHS Foundation Trust

    Birmingham, United Kingdom

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

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

    Hamburg, 20246, Germany

  • University of Utah

    Salt Lake City, Utah, 84108, United States

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

    Heidelberg, 69120, Germany

  • Universitätsklinikum Münster

    Münster, 48149, Germany

  • Univerzitetni klinicni center Ljubljana

    Ljubljana, 1000, Slovenia

  • Westmead Hospital

    Westmead, New South Wales, 2145, Australia

  • Çukurova Üniversitesi Tıp Fakültesi Balcalı Hastanesi

    Adana, 01330, Turkey (Türkiye)

  • İstanbul Üniversitesi Cerrahpaşa Tıp Fakültesi

    Fatih, Istanbul, 34098, Turkey (Türkiye)

Conditions

Explore the condition pages connected to this study.