NCT00992459

Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders

A Phase 3, Randomized, Double-Blind, Cross-Over, Active-Controlled Study of the Efficacy and Safety of HPN-100, Glyceryl Tri-(4-phenylbutyrate), for the Treatment of Adults With Urea Cycle Disorders (Help UCD)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 46 (actual)
• Sponsor: Amgen · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This was a randomized, active-controlled, double-blind, cross-over study designed to enroll subjects with UCDs who are being treated with NaPBA.

Detailed description

This was a randomized, active-controlled, double-blind, cross-over study designed to enroll subjects with UCDs who are being treated with NaPBA. Subjects were randomly assigned to receive either HPN-100 + NaPBA placebo or NaPBA + HPN 100 placebo for 2 weeks, and then crossed over to receive the other treatment for 2 weeks. Venous ammonia was the primary outcome measure. Subjects were admitted to the clinical research unit for 24 hours of pharmacokinetic (PK) blood and urine sampling (including an overnight stay) at the end of each treatment period, by which time the study drug had reached steady state. Subjects followed a stable diet throughout the study as prescribed by the investigator and dietician. Throughout the study, diet diaries were completed by the subject and dietary protein intake were assessed by a dietician based on completed dietary diaries and consultation with the subject. Subjects who completed this study and met the study entry criteria, were offered the opportunity to enroll in the HPN-100 open-label safety protocol (HPN-100-007). Study acquired from Horizon in 2024.

Conditions

• Urea Cycle Disorders

Interventions

Type	Name	Description
DRUG	HPN-100	HPN-100 is a triglyceride that has a similar mechanism of action as NaPBA. It is a liquid with minimal taste and odor. Three teaspoons of HPN-100 (\~17.4mL) delivers equivalent amount of PBA in 40 tablets of NaPBA.
DRUG	Buphenyl (NaPBA)	Buphenyl (NaPBA) will be the comparator drug to HPN-100 in this study.

Timeline

• Start date: 2009-10-01
• Primary completion: 2010-09-01
• Completion: 2010-09-01
• First posted: 2009-10-09
• Last updated: 2024-07-09
• Results posted: 2013-08-12

Locations

22 sites across 2 countries: United States, Canada