Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders
Status:
Completed
Trial end date:
2011-08-01
Target enrollment:
Participant gender:
Summary
Protocol HPN-100-005 was the first study of HPN-100 in pediatric subjects with urea cycle
disorders (UCDs) and was a fixed-sequence, open-label, switch over study of HPN-100 with a
long-term (12 month) safety extension designed to assess the safety of HPN-100 and to
prospectively assess its ability to control blood ammonia as compared with Sodium
Phenylbutyrate (NaPBA). Upon DSMB review of the first ten subjects who completed the switch
over part of the study, and with DSMB approval, up to an additional 20 subjects were enrolled
into the safety extension part of the study. 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 an equivalent amount of PBA to 40 tablets of NaPBA.