Ammonia Control in Children with Urea Cycle Disorders (UCDs); Phase 2 Comparison of Sodium Phenylbutyrate and Glycerol Phenylbutyrate

Twenty four hour ammonia profiles and correlates of drug effect were examined in a phase 2 comparison of sodium phenylbutyrate (NaPBA) and glycerol phenylbutyrate (GPB or HPN-100), an investigational drug being developed for urea cycle disorders (UCDs).

Study Design: Protocol HPN-100-005 involved open label fixed-sequence switch-over from the prescribed NaPBA dose to a PBA-equimolar GPB dose with controlled diet.

Author(s): Uta Lichter-Konecki, George Diaz, J. Lawrence Merritt, Annette Feigenbaum, Claudia Jomphe, Jean-Francois Marier, Martin Beliveau, Joseph Mauney, Klara Dickinson, Antonia Martinez, Masoud Mokhtarani, Bruce Scharschmidt, William Rhead

Year: August 1, 2011