A Phase 1/2 study to evaluate the safety and efficacy of BMN 307 gene therapy in patients with phenylketonuria

Registry ID

EUCTR2019-001878-28

Source registry

EUCTR

Status

GB - no longer in EU/EEA

Phase

PHASE2

Sponsor

BioMarin Pharmaceutical Inc.

Start date

2019-12-17

Last update

2026-08-16

Conditions

• Phenylketonuria

Summary

- Determine safe, effective, and tolerable dose of BMN 307 - Determine the efficacy of a single administration of BMN 307

Locations

• , , UK

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