NCT01114737.

Methods	Allocation: randomized Endpoint classification: safety and efficacy trial Intervention model: parallel assignment Masking: double blind (participant, caregiver, investigator, outcome assessor) Primary purpose: treatment
Participants	People with PKU. 8 years to 65 years
Interventions	Oral sapropterin dihydrochloride (20 mg/kg/day) versus placebo
Outcomes	Primary outcome measures Evaluate the therapeutic effects of sapropterin dihydrochloride on the symptoms of ADHD and on global function compared to placebo, in subjects with a blood phe level reduction after treatment ADHD change will be measured as a change in ADHD from baseline to week 13 using the Attention‐Deficit Hyperactivity Disorder Rating Scale and Adult ADHD Self‐Report Scale (ADHD RS/ASRS) measurement Global function will be measured as a change in global function using the Clinical Global Impression‐Improvement (CGI‐I) scale rating compared from baseline to week 13. Secondary outcome measures Evaluate the therapeutic effects of sapropterin dihydrochloride on the symptoms of anxiety and depression compared to placebo, in subjects with a blood phe level reduction after treatment Evaluate the durability of any therapeutic effects of sapropterin dihydrochloride on neuropsychiatric symptoms and global function of subjects who have a blood phe level reduction after treatment. Determine if sapropterin dihydrochloride has a therapeutic effect on neuropsychiatric symptoms in PKU people who do not have a blood phe reduction after treatment Assess the safety of sapropterin dihydrochloride when administered as therapy to these participants
Notes	Estimated completion date: January 2013. Estimated enrolment: 200 participants ClinicalTrials.gov Identifier: NCT01114737