PHENOPTIN TO INCREASE PHENYLALANINE TOLERANCE IN CHILDREN WITH PKU

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The primary objective of the study: " To evaluate the ability of Phenoptin to increase the tolerance in BH4 responsive children with phenylketonuria who are a Phe-restricted diet. The secondary objective of the study are: " To evaluate the ability of Phenoptin to reduce blood Phe levels in children with phenylketonuria that are following a Phe-restricted diet. " To compare the ability of Phenoptin versus placebo to increase Phe tolerance in children with phenylketonuria who are following a Phe-restricted diet " To explore the potential reduction in the cost of medical foods and Phe-free formulas " Safety Objective: " To evaluate the safety of Phenoptin as compared with placebo in this subject population.