Conditions: Phenylketonuria
Keywords: Phenylketonuria Sapropterin Placebo Cognitive Abilities Kuvan®
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 2
Healthy volunteers: no

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 description:

Arms:
arm 1: None arm 2: None

Interventions: intervention 1: Sapropterin intervention 2: Placebo
Intervention descriptions: intervention 1: 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. intervention 2: 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.

Protocol document: