Phase 2 comparison of a novel ammonia scavenging agent with sodium phenylbutyrate in patients with urea cycle disorders: safety, pharmacokinetics and ammonia …

B Lee, W Rhead, GA Diaz, BF Scharschmidt… - Molecular genetics and …, 2010 - Elsevier
Glycerol phenylbutyrate (glyceryl tri (4-phenylbutyrate))(GPB) is being studied as an
alternative to sodium phenylbutyrate (NaPBA) for the treatment of urea cycle disorders
(UCDs). This phase 2 study explored the hypothesis that GPB offers similar safety and
ammonia control as NaPBA, which is currently approved as adjunctive therapy in the chronic
management of UCDs, and examined correlates of 24-h blood ammonia. METHODS: An
open-label, fixed sequence switch-over study was conducted in adult UCD patients taking …