Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria (SIGNAL)
|ClinicalTrials.gov Identifier: NCT01977820|
Recruitment Status : Terminated (Recruitment challenges)
First Posted : November 7, 2013
Results First Posted : February 12, 2016
Last Update Posted : February 22, 2018
|Condition or disease||Intervention/treatment||Phase|
|Phenylketonuria||Drug: Sapropterin Drug: Placebo||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||2 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Investigator)|
|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|
|Study Start Date :||February 2014|
|Actual Primary Completion Date :||November 2014|
|Actual Study Completion Date :||November 2014|
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||
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 Placebo tablets matching to sapropterin will be administered orally once daily during the 24-Week study period.
- 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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01977820
|Study Director:||Medical Responsible||BioMarin Pharmaceutical|