Phase 3
N=23
Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)
Urea Cycle Disorders
Bottom Line
View on ClinicalTrials.gov: NCT01347073 ↗Enrolled (actual)
23
Serious AEs
47.8%
Results posted
May 2015
Primary outcome: Primary: Adverse Events — 0; 6 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- HPN-100 (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Amgen
- Primary completion
- Feb 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Adverse Events |
23 | — |
| PRIMARY Adverse Events |
23 | — |
| SECONDARY Blood Ammonia |
914.43; 647.63 | — |
| SECONDARY Frequency of Ammonia Levels Greater Than the Upper Limit of Normal (ULN) on HPN-100 Compared With NaPBA |
22; 8 | — |
| SECONDARY Hyperammonemic Crisis |
29; 12 | — |
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.
Eligibility Criteria
Inclusion Criteria
- Male and female subjects 29 days to 100 μmol/L and signs and symptoms indicative of hyperammonemia; subjects may be rescreened after their ammonia is controlled and they are clinically stable, at the discretion of the investigator
- Use of any investigational drug within 30 days of Day 1
- Active infection (viral or bacterial) or any other condition that may increase ammonia levels
- Any clinical or laboratory abnormality of Grade 3 or greater severity according to the Common Terminology Criteria for Adverse Events (CTCAE) v4.03, except Grade 3 elevations in ammonia and liver enzymes, defined as levels 5-20 times ULN (upper limit of normal)in alanine aminotransferase (ALT), aspartate aminotransferase (AST), or gamma glutamyl transpeptidase (GGT) in a clinically stable subject
- Any clinical or laboratory abnormality or medical condition that, at the discretion of the investigator, may put the subject at increased risk by participating in this study
- Known hypersensitivity to PAA or PBA
- Liver transplant, including hepatocellular transplant
- Currently treated with Carbaglu® (carglumic acid)
Data sourced from ClinicalTrials.gov (NCT01347073). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.