PHENOPTIN TO INCREASE PHENYLALANINE TOLERANCE IN CHILDREN WITH PKU

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目及 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者的研究机构。 本研究的主要目的： " 评估苯那普汀增加苯丙酮尿症患儿对苯丙氨酸限制饮食的BH 4反应耐受性的能力。 本研究的次要目的为： " 评价苯那普汀降低苯丙酮尿症儿童血苯丙氨酸水平的能力，这些儿童遵循苯丙氨酸限制饮食。 " 比较Phenoptin与安慰剂增加苯丙酮尿症儿童苯丙氨酸耐受性的能力，这些儿童遵循苯丙氨酸限制饮食 " 探索降低医疗食品和无苯丙氨酸配方食品成本的可能性 " 安全目标： " 在该受试者人群中评价Phenoptin与安慰剂相比的安全性。

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.