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NCT05166161 · ClinicalTrials.gov registry record · Phase 3
A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
A Phase 3 study of Phenylketonuria, sponsored by PTC Therapeutics.
- Active
- Registry status
- Phase 3
- Development phase
- 200
- Enrollment target
- 20
- Study locations
NCT05166161: Active Phase 3 study of Phenylketonuria, sponsored by PTC Therapeutics.
NCT05166161 is a Phase 3 study of Phenylketonuria that is active but no longer recruiting, run by PTC Therapeutics. The registered enrollment target is 200 participants, above the 82-participant average among 12 other Phenylketonuria trials with a reported enrollment target (144% higher). The trial reports 20 study locations across 15 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05166161, a Phase 3 study of Phenylketonuria, is active but no longer recruiting, sponsored by PTC Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 200 participants
- Enrollment target
- 20
- Study locations
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.
Primary Outcome
A TEAE is any unfavorable or unintended sign (including an abnormal laboratory finding), symptom or disease in a study participant who is administered study drug in this study
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 |
What the registry record for NCT05166161 still lists
NCT05166161 is an interventional study that assigns participants to a tested intervention. The registered 200 participants enrollment target is mid-sized for trials with a published cap, above the 82-participant average among 12 other Phenylketonuria trials with a reported enrollment target (144% higher).
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.
NCT05166161 names 20 study sites across 15 states, led by Other, Ontario, Colorado.
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.
What interventions are being tested in trial NCT05166161?
The interventions under investigation include: PTC923 (DRUG).
Who is sponsoring clinical trial NCT05166161?
This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
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Where NCT05166161's enrollment target sits among peer trials
200 2nd of 12 higher than 10 of 12 other Phenylketonuria trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phenylketonuria trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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