Phenylketonuria Clinical Trial
Official title:
A Double-blind, Placebo-controlled, Randomized Study to Evaluate the Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Subjects With Phenylketonuria
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.
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.
;
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
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