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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

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Trial Profile

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

Status: Suspended
Phase of Trial: Phase I/II

Latest Information Update: 17 Dec 2024

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At a glance

  • Drugs Domofenogene zalfaparvovec (Primary)
  • Indications Phenylketonuria
  • Focus First in man; Pharmacokinetics
  • Acronyms PHEARLESS
  • Sponsors BioMarin Pharmaceutical

Most Recent Events

  • 17 Feb 2022 According to a BioMarin Pharmaceutical media release, the FDA has requested data from additional non-clinical studies to assess the theoretical oncogenic risk to human study participants, which is expected to take several quarters.The company will communicate next steps for the program when available.
  • 17 Feb 2022 According to a BioMarin Pharmaceutical media release, the Company received additional requests from the Food and Drug Administration (FDA) for information needed to resolve the clinical hold of the PHEARLESS Phase 1/2 study of BMN 307 issued in September 2021.
  • 05 Sep 2021 According to a BioMarin Pharmaceutical media release, the company will work with the Data Review Board and Principal Investigators to further evaluate the study participants who have been dosed and will continue to monitor them over the long-term. Enrollment will be paused until the investigation is completed. The company is working with the FDA and other health authorities and will communicate next steps for the program when available.

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