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NCT04480567 · ClinicalTrials.gov registry record · Phase 1
AAV Gene Therapy Study for Subjects with PKU
A Phase 1 study of Phenylketonuria (PKU), sponsored by BioMarin Pharmaceutical.
- Active
- Registry status
- Phase 1
- Development phase
- 100
- Enrollment target
- 3
- Study locations
NCT04480567: Active Phase 1 study of Phenylketonuria (PKU), sponsored by BioMarin Pharmaceutical.
NCT04480567 is a Phase 1 study of Phenylketonuria (PKU) that is active but no longer recruiting, run by BioMarin Pharmaceutical. The registered enrollment target is 100 participants, below the 166-participant average among 6 other Phenylketonuria (PKU) trials with a reported enrollment target (40% lower). The trial reports 3 study locations across 3 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04480567, a Phase 1 study of Phenylketonuria (PKU), is active but no longer recruiting, sponsored by BioMarin Pharmaceutical.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 100 participants
- Enrollment target
- 3
- Study locations
Study Summary
This is a Phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of BMN 307 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of BMN 307 and will be followed for safety and efficacy.
Conditions Studied
Interventions
- DRUG BMN 307
Study Locations (3)
Florida
- University of South Florida - Tampa
New Jersey
- Morristown Medical Center - Morristown
Other
- University Hospital Birmingham NHS Foundation Trust - Birmingham
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 100 participants |
| Start Date | 2020-09-24 |
| Est. Completion | 2027-12 |
| Phase | Phase 1 |
What the registry record for NCT04480567 still lists
NCT04480567 is an interventional study that assigns participants to a tested intervention. The registered 100 participants enrollment target is mid-sized for trials with a published cap, below the 166-participant average among 6 other Phenylketonuria (PKU) trials with a reported enrollment target (40% lower).
The record links to 1 condition, with Phenylketonuria (PKU) appearing as the primary indexed condition, and to 1 intervention - of which BMN 307 is the first listed.
NCT04480567 reports a single indexed study location in Florida, New Jersey, Other.
Frequently Asked Questions
What is clinical trial NCT04480567 about?
NCT04480567 is a clinical study titled "AAV Gene Therapy Study for Subjects with PKU". This is a Phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of BMN 307 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of BMN 307 and will be followed for safety and efficacy.
What is the current status of trial NCT04480567?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 100 participants. The study started on 2020-09-24. Estimated completion is 2027-12.
What conditions does trial NCT04480567 study?
This clinical trial studies the following conditions: Phenylketonuria (PKU).
What interventions are being tested in trial NCT04480567?
The interventions under investigation include: BMN 307 (DRUG).
Who is sponsoring clinical trial NCT04480567?
This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04480567 being conducted?
This trial has 3 study locations across Florida, New Jersey. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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