NCT01114737

Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients

A Double-blind, Placebo-controlled, Randomized Study to Evaluate the Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Subjects With Phenylketonuria

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 206 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 8 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This double-blind, placebo-controlled, randomized study is designed to evaluate the safety and therapeutic effects of sapropterin dihydrochloride on neuropsychiatric symptoms in subjects with PKU.

Detailed description

Phenylketonuria (PKU) results from deficient phenylalanine hydroxylase (PAH) activity and leads to toxic phenylalanine (Phe) accumulation in patients with PKU causing mental retardation, microcephaly, delayed speech, seizures, psychiatric symptoms and behavioral abnormalities. Although for most PKU patients early initiation of dietary treatment prevents severe complications, discontinuation of dietary restrictions at an early age is associated with poor cognitive development and neuropsychiatric disorders are present even in early-treated and well controlled PKU patients. This study, PKU-016, will be conducted in PKU patients to evaluate the therapeutic effects of sapropterin dihydrochloride on the symptoms of attention deficit hyperactivity disorder (ADHD), depression, and anxiety.

Conditions

• Phenylketonuria

Interventions

Type	Name	Description
DRUG	Sapropterin dihydrochloride	A dose of 20 mg/kg/day will be administered. Route of administration is oral (intact).
DRUG	Placebo	Placebo (tablet without active ingredient) is dosed once/day for the first 13 weeks of the study.

Timeline

• Start date: 2010-08-01
• Primary completion: 2013-03-01
• Completion: 2013-03-01
• First posted: 2010-05-03
• Last updated: 2016-02-01
• Results posted: 2016-02-01

Locations

36 sites across 2 countries: United States, Canada