New pill for PKU shows promise in Long-Term safety trial
NCT ID NCT05166161
First seen Feb 19, 2026 · Last updated May 15, 2026 · Updated 12 times
Summary
This study tests the long-term safety of an experimental drug called PTC923 (sepiapterin) in 200 people with phenylketonuria (PKU), a genetic condition that makes it hard to process the amino acid phenylalanine. Participants take the drug and researchers track side effects and changes in how much phenylalanine they can eat while keeping safe blood levels. The goal is to see if PTC923 can help people with PKU safely manage their diet over time.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for PHENYLKETONURIA are added.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
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
-
The Queensland Children's Hospital
South Brisbane, Brisbane, Australia
-
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.