Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)

• Status: Completed
• Trial end date: 2013-03-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Horizon Pharma Ireland, Ltd., Dublin Ireland
• Treatments: 4-phenylbutyric acid; Glycerol

Criteria

Inclusion Criteria:

• Male and female subjects 29 days to < 6 years old. If the subject is born prematurely, calculation of the lower age limit begins at the corrected gestational age of 40 weeks.

• Signed informed consent by the subject's legally acceptable representative

• Suspected or confirmed UCD diagnosis of any subtype, except NAGS deficiency

• On stable dose of NaPBA powder for at least 5 days before Day 1

• Not receiving sodium benzoate for at least 5 days before Day 1

• No concomitant illness which would preclude safe participation as judged by the investigator

• Able to receive medication orally

• Has not undergone liver transplantation, including hepatocellular transplantation

• Judged sufficiently stable and compliant with diet and treatment to be suitable for enrollment

Exclusion Criteria:

• Screening ammonia level > 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)