Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria (SIGNAL)
This is a Phase IIa 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. This pilot trial is conducted to detect trends and generate hypotheses, relating to variability and the possible effect size on executive function, attention and processing speed. As this trial is exploratory in nature, no statistical endpoints were defined as primary or secondary.
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
|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 Phenylketonuria|
- Number of subjects with adverse event [ Time Frame: baseline up to 30 weeks ] [ Designated as safety issue: Yes ]
|Study Start Date:||February 2014|
|Study Completion Date:||September 2014|
|Primary Completion Date:||September 2014 (Final data collection date for primary outcome measure)|
Sapropterin tablets will be administered orally once daily at a dose of 20 milligram per kilogram (mg/kg) during both the 2-week response test period and 24-week study period.
Other Name: Kuvan®
|Placebo Comparator: Placebo||
Placebo tablets matching to Sapropterin will be administered orally once daily during the 24-week study period.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01977820
|Santiago de Compostela, Spain|
|Study Director:||Medical Responsible||Merck KGaA|