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Clinical trial

A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects with Phenylketonuria

NCT04480567 is registered in ClinicalTrials.gov with BioMarin Pharmaceutical as lead sponsor.

NCT NCT04480567
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Registry record

As filed in ClinicalTrials.gov.

Title
A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects with Phenylketonuria
Lead sponsor
BioMarin Pharmaceutical
Phase
Phase 1/2
Overall status
Active, not recruiting
Start date
September 24, 2020
Completion date
December 31, 2027
Enrollment
100 (estimated)
Sites
3

Conditions

As the registry lists them; a condition with its own page is linked.

phenylketonuria (pku)