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ClinicalTrials.gov public records Last synced Sep 13, 2026, 8:43 PM EDT

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Showing 1–1 of 1 matching trial from the live ClinicalTrials.gov search.
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Active, not recruiting Phase 1Phase 2 Interventional

AAV Gene Therapy Study for Subjects with PKU

NCT04480567
Conditions
Phenylketonuria (PKU)
Interventions
BMN 307
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
15 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
2
States / cities
Tampa, Florida • Morristown, New Jersey