An Open Label Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate (GPB; RAVICTI) in Pediatric Subjects Under Two Years of Age With Urea Cycle Disorders (UCDs)

Trial Profile

An Open Label Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate (GPB; RAVICTI) in Pediatric Subjects Under Two Years of Age With Urea Cycle Disorders (UCDs)

Completed
Phase of Trial: Phase IV

Latest Information Update: 23 Oct 2017

At a glance

  • Drugs Glycerol phenylbutyrate (Primary)
  • Indications Inborn urea cycle disorders
  • Focus Therapeutic Use
  • Sponsors Horizon Pharma
  • Most Recent Events

    • 15 Aug 2017 Status changed from active, no longer recruiting to completed.
    • 01 May 2017 According to a Horizon Pharma media release, the US FDA has approved the company's supplemental New Drug Application (sNDA) to expand the age range for RAVICTI (glycerol phenylbutyrate) Oral Liquid to people two months of age and older who have urea cycle disorders (UCDs) that cannot be managed by dietary protein restriction and/or amino acid supplementation alone. The approval was based on this and two other studies.
    • 12 Jan 2017 Status changed from recruiting to active, no longer recruiting.
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