Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria

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.

Timeline

Start

2014-02

Primary completion

2014-11

Completion

2014-11

Drugs

Indications