A Four-Part, Phase 3, Randomized, Double-Blind, Placebo- Controlled, Four-Arm, Discontinuation Study to Evaluate the Efficacy and Safety of Subcutaneous Injections of BMN 165 Self-Administered by Adults With Phenylketonuria (PKU)

Trial Profile

A Four-Part, Phase 3, Randomized, Double-Blind, Placebo- Controlled, Four-Arm, Discontinuation Study to Evaluate the Efficacy and Safety of Subcutaneous Injections of BMN 165 Self-Administered by Adults With Phenylketonuria (PKU)

Active, no longer recruiting
Phase of Trial: Phase III

Latest Information Update: 02 Jan 2018

At a glance

  • Drugs Pegvaliase (Primary)
  • Indications Phenylketonuria
  • Focus Adverse reactions; Registrational
  • Acronyms PRISM-2; Prism302
  • Sponsors BioMarin Pharmaceutical
  • Most Recent Events

    • 22 Dec 2017 According to a BioMarin Pharmaceutical media release, the U.S. Food and Drug Administration (FDA) will require additional time to complete its review of the Biologics License Application (BLA).In a notice received from the FDA, the Prescription Drug User Fee Act (PDUFA) Goal Date for pegvaliase has been extended by three months to May 28, 2018. Due to the Memorial Day weekend, the Action Goal Date will be May 25, 2018.
    • 29 Nov 2017 Planned End Date changed from 1 Nov 2017 to 31 Mar 2019.
    • 29 Nov 2017 Planned primary completion date changed from 1 Jun 2017 to 31 Dec 2018.
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