A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
NCT ID: NCT05166161
Last Updated: 2025-05-08
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
200 participants
INTERVENTIONAL
2022-02-14
2026-04-30
Brief Summary
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Detailed Description
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Feeder participants: those who have completed a Phase 3 PTC Therapeutics (PTC) sponsored feeder study.
Non-feeder controlled participants: those who have not completed a feeder study and have blood Phe levels \<360 μmol/L at study entry.
Non-feeder uncontrolled participants: those who have not completed a feeder study and have blood Phe levels ≥360 μmol/L at study entry.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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PTC923
Participants will receive PTC923 7.5 mg/kg (participants 0 to \<6 months of age), 15 mg/kg (participants 6 to \<12 months of age), 30 mg/kg (participants 12 months to \<2 years of age), or 60 mg/kg (participants ≥2 years of age) orally once daily for a minimum of 12 months or until participant experiences lack of efficacy, adverse events (AEs) that lead to discontinuation, withdraws from treatment, or PTC923 is authorized and commercially available in the specific country.
PTC923
PTC923 powder for oral use will be suspended in water or apple juice prior to administration.
Interventions
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PTC923
PTC923 powder for oral use will be suspended in water or apple juice prior to administration.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Women of childbearing potential must have a negative pregnancy test at screening and agree to abstinence or the use of at least one highly effective form of contraception for the duration of the study, and for up to 90 days after the last dose of the study drug.
* Males who are sexually active with women of childbearing potential who have not had a vasectomy must agree to use a barrier method of birth control during the study and for up to 90 days after the last dose of study drug. Males must also refrain from sperm donations during this time period.
* Willing to continue current diet unchanged while participating in the study (unless specifically instructed to change diet during the study by the investigator).
Exclusion Criteria
* A female who is pregnant or breastfeeding, or considering pregnancy.
* Serious neuropsychiatric illness (for example, major depression) not currently under medical control, that in the opinion of the investigator or PTC, would interfere with the participant's ability to participate in the study or increase the risk of participation for that participant.
* Past medical history and/or evidence of renal impairment and/or condition including moderate/severe renal insufficiency (glomerular filtration rate \[GFR\] \<60 milliliters \[mL\]/minute \[min\] min as estimated most recently during qualifying participation in a feeder study) and/or under care of a nephrologist.
* Any other condition that in the opinion of the investigator or PTC, would interfere with the participant's ability to participate in the study or increase the risk of participation for that participant.
* Requirement for concomitant treatment with any drug known to inhibit folate synthesis (for example, methotrexate).
* Concomitant treatment with tetrahydrobiopterin (BH4) supplementation (for example, sapropterin dihydrochloride, KUVAN) or pegvaliase-pqpz (PALYNZIQ).
Additional criteria for non-feeder participants who did not participate in a feeder study:
* Gastrointestinal disease (such as irritable bowel syndrome, inflammatory bowel disease, chronic gastritis, and peptic ulcer disease, etc) that could affect the absorption of study drug.
* History of gastric surgery, including Roux-en-Y gastric bypass surgery or an antrectomy with vagotomy, or gastrectomy.
* History of allergies or adverse reactions to synthetic BH4 or sepiapterin.
* Any clinically significant laboratory abnormality as determined by the investigator.
* Any abnormal physical examination and/or laboratory findings indicative of signs or symptoms of renal disease, including calculated GFR \<60 milliliters (mL)/minute/1.73 square meter (m\^2).
Confirmed diagnosis of a primary BH4 deficiency as evidenced by biallelic pathogenic mutations in 6-pyruvoyltetrahydropterin synthase, recessive GTP cyclohydrolase I, sepiapterin reductase, quinoid dihydropteridine reductase, or pterin-4-alphacarbinolamine dehydratase genes.
ALL
No
Sponsors
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PTC Therapeutics
INDUSTRY
Responsible Party
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Locations
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Children's Hospital Colorado
Aurora, Colorado, United States
UF College of Medicine, Department of Pediatrics Division of Genetics and Metabolism
Gainesville, Florida, United States
Indiana University School of Medicine Department of Medical and Molecular Genetics
Indianapolis, Indiana, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Icahn School of Medicine at Mount Sinai (ISMMS)
New York, New York, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
University of Utah
Salt Lake City, Utah, United States
Westmead Hospital
Westmead, New South Wales, Australia
PARC Clinical Research
Adelaide, South Australia, Australia
Royal Children's Hospital
Parkville, Victoria, Australia
Hospital de clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, Brazil
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo
Ribeirão Preto, São Paulo, Brazil
Metabolics and Genetics in Calgary (MAGIC) Clinic, Ltd.
Calgary, Alberta, Canada
McMaster Children's Hospital Hamilton Health Sciences
Hamilton, Ontario, Canada
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, Canada
Fakultní nemocnice Královské Vinohrady Klinika dětí a dorostu Ambulance pro léčbu PKU a HPA
Prague, , Czechia
Copenhagen University Hospital, Rigshospitalet
Copenhagen, , Denmark
Hôpital Necker-Enfants Malades - Centre de Référence des Maladies Héréditaires du Métabolisme
Paris, , France
Medical Genetics and Laboratory Diagnostics Center
Tbilisi, , Georgia
University Children's Hospital Hamburg Eppendorf (Kinder-UKE) Klinik für Kinder- und Jugendmedizin (Kinder-UKE)
Hamburg, , Germany
Universitätsklinikum Heidelberg / Zentrum für Kinder- und Jugendmedizin / Sektion für Neuropädiatrie & Stoffwechselmedizin
Heidelberg, , Germany
Universitätsklinikum Münster
Münster, , Germany
Azienda Ospedaliera-Universita Padova
Padua, Veneto, Italy
Department of Human Neuroscience, Child and Adolescent Neuropsychiatry, Policlinico Umberto I
Rome, , Italy
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, Mexico
UMCG Beatrix Children's Hospital
Groningen, , Netherlands
Pomorski Uniwersytet Medyczny w Szczecinie Centrum Wsparcia Badań Klinicznych
Szczecin, , Poland
CENTRO HOSPITALAR UNIVERSITÁRIO LISBOA NORTE Hospital de Santa Maria,
Lisbon, Estremadura, Portugal
CENTRO HOSPITALAR UNIVERSITÁRIO LISBOA NORTE Hospital de Santa Maria
Lisbon, Estremadura, Portugal
Centro Hospitalar Universitário Do Porto, Epe
Porto, , Portugal
Univerzitetni klinicni center Ljubljana
Ljubljana, , Slovenia
Hospital Sant Joan de Déu
Barcelona, Esplugues de Llobregat, Spain
Hospital Universitario Ramón y Cajal
Madrid, , Spain
Hospital Universitario Virgen del Rocío
Seville, , Spain
Hacettepe University Medical Faculty
Altındağ, Ankara, Turkey (Türkiye)
Gazi Üniversitesi Tıp Fakültesi
Yenimahalle, Ankara, Turkey (Türkiye)
İstanbul Üniversitesi Cerrahpaşa Tıp Fakültesi
Fatih, Istanbul, Turkey (Türkiye)
Ege University Faculty of Medicine Children Hospital
Bornova, İzmir, Turkey (Türkiye)
Çukurova Üniversitesi Tıp Fakültesi Balcalı Hastanesi
Adana, , Turkey (Türkiye)
Birmingham Children's Hospital NHS Foundation Trust
Birmingham, , United Kingdom
UNIVERSITY HOSPITALS BIRMINGHAM NHS Foundation Trust
Birmingham, , United Kingdom
Great Ormond Street Hospital
London, , United Kingdom
Countries
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Central Contacts
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Other Identifiers
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2021-000497-28
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
2023-509229-31-00
Identifier Type: OTHER
Identifier Source: secondary_id
PTC923-MD-004-PKU
Identifier Type: -
Identifier Source: org_study_id
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