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

@article{Lee2010Phase2C,
  title={Phase 2 comparison of a novel ammonia scavenging agent with sodium phenylbutyrate in patients with urea cycle disorders: safety, pharmacokinetics and ammonia control.},
  author={Brendan Lee and William James Rhead and George A Diaz and Bruce F. Scharschmidt and A. Mian and Oleg A. Shchelochkov and J. F. Marier and Martin Beliveau and Joseph Mauney and Klara Dickinson and Antonia Mart{\'i}nez and Sharron Gargosky and Masoud Mokhtarani and Susan A Berry},
  journal={Molecular genetics and metabolism},
  year={2010},
  volume={100 3},
  pages={221-8}
}
UNLABELLED 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… CONTINUE READING