CI

At a glance

ClinicalIndex Comparison Record
Phase 3Completed· 23 enrolled
Drug / intervention
HPN-100drug
Likely dose
HPN-100 17.4 mLfrom record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT01347073
NCT01347073Phase 3Completed
CompareCT.gov

A Switch-Over, Open-Label Study of the Safety, Pharmacokinetics, and Efficacy of HPN-100, Followed by Long-Term Treatment With HPN-100, in Pediatric Subjects Under 6 Years of Age With Urea Cycle Disorders (UCDs)

Amgen·interventional·Posted May 4, 2011·Updated Jul 11, 2024

In Brief

A Phase 3 clinical trial evaluating HPN-100 for Urea Cycle Disorders. Completed, enrolled 23 participants across 8 sites.

Detailed Summary

This non-randomized, open-label study was approximately one year in duration and consisted of a short term NaPBA to HPN-100 switchover part involving two overnight stays followed by a 12-month long term treatment period involving monthly visits.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsUrea Cycle Disorders
CountriesUnited States
Collaborators--

Timeline

Phase 3CompletedFinished
2011201220132014201520162017201820192020202120222023202420252026
First PostedMay 4, 2011
Enrollment StartJul 1, 2011
Primary CompletionFeb 1, 2013
Study CompletionMar 1, 2013
TodayJul 2, 2026
Enrollment to primary: 1.6 yearsPosted 15.2 years ago

Interventions

HPN-100drug

HPN-100 is a pro-drug of PAA that combines with glutamine to provide an alternative vehicle for waste nitrogen elimination. It is a liquid with minimal taste and odor. Approximately three teaspoons of HPN-100 (\~17.4 mL) delivers an equivalent amount as PBA that 40 tablets of NaPBA.