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 ]

An AE was defined as any new untoward medical occurrences/worsening of pre-existing medical condition without regard to possibility of causal relationship. A SAE was an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect.


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