Clinical Trial

AAV Gene Therapy Study for Subjects with PKU

Active, Not Recruiting Phase 1/2
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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.
Protocol Amendment History 5 amendments
This ClinicalTrials.gov record has been amended 5 times since 2020-07-20; most recent amendment 2024-12-09.
Status change: Recruiting → Active, Not Recruiting 2021-10-06
Trial Details
NCT Number NCT04480567
Lead Sponsor BioMarin Pharmaceutical
Conditions Phenylketonuria (PKU)
Enrollment 100 participants
Start Date 2020-09-24
Primary Completion 2027-12 (estimated)
Study Completion 2027-12 (estimated)
Updated on ClinicalTrials.gov 2024-12-12