Clinical Trial: Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional




Official Title: A Multicenter, Double-Blind, Placebo-Controlled, Randomized, 2-Arm Phase IIa Pilot Trial Assessing the Effect of Sapropterin on Cognitive Abilities in Young Adults With Phenylket

Brief Summary: This is a Phase 2a multicenter, double-blind, placebo-controlled, randomized, 2-arm pilot trial designed to assess the effect of sapropterin on cognitive abilities in young adults with Phenylketonuria (PKU) over a 26-week treatment period.