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NCT05270837 · ClinicalTrials.gov registry record · Phase 3
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
A Phase 3 study of Phenylketonuria (PKU), sponsored by BioMarin Pharmaceutical.
- Active
- Registry status
- Phase 3
- Development phase
- 55
- Enrollment target
- 16
- Study locations
NCT05270837: Active Phase 3 study of Phenylketonuria (PKU), sponsored by BioMarin Pharmaceutical.
NCT05270837 is a Phase 3 study of Phenylketonuria (PKU) that is active but no longer recruiting, run by BioMarin Pharmaceutical. The registered enrollment target is 55 participants, below the 173-participant average among 6 other Phenylketonuria (PKU) trials with a reported enrollment target (68% lower). The trial reports 16 study locations across 14 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05270837, a Phase 3 study of Phenylketonuria (PKU), is active but no longer recruiting, sponsored by BioMarin Pharmaceutical.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 55 participants
- Enrollment target
- 16
- Study locations
Study Summary
This is a Phase 3 open-label randomized controlled study enrolling approximately 54 adolescents with PKU. The study is designed to assess the safety and efficacy of pegvaliase injections.
Primary Outcome
The blood Phe measurement following 72 weeks on study is defined as the average of the Week 69 and Week 73 blood Phe measurements. Change from baseline in blood Phe will be compared between participants in the active (pegvaliase) and the control (diet-only) treatment arms. Baseline blood Phe concentration at Part 1 was defined as the average of 3 pre-treatment measurements collected between Screening/Run-in (2) and Day 1 pre-dose (1).
Conditions Studied
Interventions
- DRUG Pegvaliase
- OTHER Diet Only
Study Locations (16)
Other
- Charité - Universitätsmedizin Berlin - Berlin
- Universitätsklinikum Hamburg-Eppendorf - Hamburg
- Universitat Mainz - Mainz
Arizona
- Phoenix Children's Hospital - Phoenix
Arkansas
- Arkansas Children's Hospital - Little Rock
Colorado
- Children's Hospital of Colorado - Aurora
Florida
- University of South Florida - Tampa
Illinois
- Ann and Robert H. Lurie Children's Hospital of Chicago - Chicago
Indiana
- IU Health University Hospital - Indianapolis
Kentucky
- University of Kentucky College of Medicine - Lexington
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 55 participants |
| Start Date | 2022-06-17 |
| Est. Completion | 2027-10 |
| Phase | Phase 3 |
What the registry record for NCT05270837 still lists
NCT05270837 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 55 participants, a relatively small participant target, below the 173-participant average among 6 other Phenylketonuria (PKU) trials with a reported enrollment target (68% lower).
The record links to 1 condition, with Phenylketonuria (PKU) appearing as the primary indexed condition, and to 2 interventions - of which Pegvaliase is the first listed.
NCT05270837 lists 16 locations in 14 states (Other, Arizona, Arkansas).
Frequently Asked Questions
What is clinical trial NCT05270837 about?
NCT05270837 is a clinical study titled "Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria". This is a Phase 3 open-label randomized controlled study enrolling approximately 54 adolescents with PKU. The study is designed to assess the safety and efficacy of pegvaliase injections.
What is the current status of trial NCT05270837?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 55 participants. The study started on 2022-06-17. Estimated completion is 2027-10.
What conditions does trial NCT05270837 study?
This clinical trial studies the following conditions: Phenylketonuria (PKU).
What interventions are being tested in trial NCT05270837?
The interventions under investigation include: Pegvaliase (DRUG), Diet Only (OTHER).
Who is sponsoring clinical trial NCT05270837?
This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05270837 being conducted?
This trial has 16 study locations across Arizona, Arkansas, Colorado, Florida, Illinois. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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