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.
Detailed Summary:
Sponsor: Merck KGaA
Current Primary Outcome: Number of Subjects With Adverse Events (AEs), Serious AEs (SAEs), AEs Leading to Death and AEs Leading to Discontinuation [ Time Frame: Screening up to 24 weeks + 4-week follow-up ]
Original Primary Outcome: Number of subjects with adverse event [ Time Frame: baseline up to 30 weeks ]
Current Secondary Outcome:
Original Secondary Outcome:
Information By: Merck KGaA
Dates:
Date Received: October 31, 2013
Date Started: February 2014
Date Completion:
Last Updated: January 14, 2016
Last Verified: January 2016