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AAV Gene Therapy Study for Subjects with PKU

StatusActive, Not Recruiting
PhasePhase 1/2
Started
View on ClinicalTrials.gov ↗

Record History

  1. Study sites: 4 to 3v6
  2. Trial status: Recruiting to Active, Not Recruiting
    • Site details revised at 4 of 4 sites
    v5
  3. Study sites: 3 to 4v4
  4. Primary and study completion dates: Dec 2026 to Dec 2027
    • Study sites: 1 to 3
    • Start date changed from estimate of Jul 2020 to Sep 24, 2020
    v3
  5. Re-verified, no change to tracked fieldsv2
  6. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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.
Trial Details
NCT Number NCT04480567
Lead Sponsor BioMarin Pharmaceutical
Conditions
(from sponsor)		 Phenylketonuria (PKU)
Related terms Phenylketonurias
Enrollment 100 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov