NCT02246218

A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders

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)

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 27 (actual)
• Sponsor: Amgen · Industry
• Sex: All
• Age: 1 Day – 2 Years
• Healthy volunteers: Not accepted

Summary

This is an open-label study consisting of a transition period to RAVICTI, followed by a safety extension period for at least 6 months and up to 24 months of treatment with RAVICTI, depending on age at enrollment. It is designed to capture information important for evaluating safety, pharmacokinetics and efficacy in young children. Subjects who are followed by or referred to the Investigator for management of their UCD. Subjects eligible for this study will include patients ranging from newborn to \< 2 years of age with either a diagnosed or clinically suspected UCD.

Detailed description

Study acquired from Horizon in 2024.

Conditions

• Urea Cycle Disorder

Interventions

Type	Name	Description
DRUG	RAVICTI

Timeline

• Start date: 2014-12-31
• Primary completion: 2016-10-17
• Completion: 2017-07-17
• First posted: 2014-09-22
• Last updated: 2024-07-01
• Results posted: 2019-03-05

Locations

17 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study