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A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
NCT05166161 · View on ClinicalTrials.gov ↗
Study Summary
The main purpose of this study is to evaluate the long-term safety of PTC923 in participants with phenylketonuria, and to evaluate the changes from baseline in dietary phenylalanine (Phe)/protein consumption.
Conditions Studied
Interventions
- DRUG PTC923
Study Locations (20)
Other
- Fakultní nemocnice Královské Vinohrady Klinika dětí a dorostu Ambulance pro léčbu PKU a HPA — Prague
- Copenhagen University Hospital, Rigshospitalet — Copenhagen
- Hôpital Necker-Enfants Malades - Centre de Référence des Maladies Héréditaires du Métabolisme — Paris
- Medical Genetics and Laboratory Diagnostics Center — Tbilisi
- University Children's Hospital Hamburg Eppendorf (Kinder-UKE) Klinik für Kinder- und Jugendmedizin (Kinder-UKE) — Hamburg
Ontario
- McMaster Children's Hospital Hamilton Health Sciences — Hamilton
- 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
Colorado
- Children's Hospital Colorado — Aurora
Florida
- UF College of Medicine, Department of Pediatrics Division of Genetics and Metabolism — Gainesville
Indiana
- Indiana University School of Medicine Department of Medical and Molecular Genetics — Indianapolis
Massachusetts
- Boston Children's Hospital — Boston
New York
- Icahn School of Medicine at Mount Sinai (ISMMS) — New York
Pennsylvania
- UPMC Children's Hospital of Pittsburgh — Pittsburgh
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 200 participants |
| Start Date | 2022-02-14 |
| Est. Completion | 2026-06-30 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05166161
The ClinicalTrials.gov registry entry for NCT05166161 describes a study currently listed as active not recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 200 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is PTC Therapeutics, which has 15 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Phenylketonuria appearing as the primary indexed condition, and to 1 intervention — of which PTC923 is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05166161 reports 20 study locations spanning 15 distinct geographic areas — top geographies include Other, Ontario, Colorado. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT05166161 about?
NCT05166161 is a clinical study titled "A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria". The main purpose of this study is to evaluate the long-term safety of PTC923 in participants with phenylketonuria, and to evaluate the changes from baseline in dietary phenylalanine (Phe)/protein consumption.
What is the current status of trial NCT05166161?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 200 participants. The study started on 2022-02-14. Estimated completion is 2026-06-30.
What conditions does trial NCT05166161 study?
This clinical trial studies the following conditions: Phenylketonuria. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05166161?
The interventions under investigation include: PTC923 (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05166161?
This trial is sponsored by PTC Therapeutics, which has 15 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT05166161 being conducted?
This trial has 20 study locations across Colorado, Florida, Indiana, Massachusetts, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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