Clinical Trial: Kuvan®'s Effect on the Cognition of Children With Phenylketonuria
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: A Phase 4 Open-Label, Single-Cohort Study of the Long-Term Neurocognitive Outcomes in 4 to 5 Year-Old Children With Phenylketonuria Treated With Sapropterin Dihydrochlorid
Brief Summary: The study is a single-cohort, interventional, open-label trial to evaluate long-term neurocognitive (NC) outcomes in children aged 4 to 5 years with phenylketonuria (PKU) treated with Kuvan® and Phenylalanine-restricted diet over a period of 7 years.
Detailed Summary:
Sponsor: BioMarin Pharmaceutical
Current Primary Outcome: Mean Full Scale Intelligence Quotient (FSIQ) Score of the Wechsler Intelligence Scale for Children (WISC)-IV [ Time Frame: Year 7 ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Height compared to the World Health Organization (WHO) Growth Standards [ Time Frame: up to 7 years ]
- Weight compared to the World Health Organization (WHO) Growth Standards [ Time Frame: up to 7 years ]
- Blood levels of tyrosine, tryptophan, pre-albumin and methylmalonic acid [ Time Frame: up to 7 years ]
- Intelligence Quotient (IQ) score and sub-scores such as, verbal IQ, performance IQ, Full Scale Intelligence Quotient (FSIQ) of Wechsler Preschool and Primary Scale of Intelligence (WPPSI)-III [ Time Frame: Baseline ]
- Intelligence Quotient (IQ) score and sub-scores such as verbal comprehension, perceptual reasoning, working memory, and processing speed indexes and Full Scale Intelligence Quotient (FSIQ) of the Wechsler Intelligence Scale for Children (WISC)-IV [ Time Frame: up to 7 years ]
- Change from Baseline in FSIQ score at 2, 4 and 7 years [ Time Frame: Baseline, and Year 2, 4 and 7 ]
- Dietary Phenylalanine tolerance [ Time Frame: up to 7 years ]
- Phenylalanine levels [ Time Frame: up to 7 years ]
- Index of Dietary Control (IDC) [ Time Frame: up to 7 years ]
- Percentage of tablets taken to assess treatment compliance [ Time Frame: up to 7 years ]
- Distribution of phenylalanine hydroxylase (PAH) genotype [ Time Frame: Baseline ]
- Number of subjects with Adverse Events and Serious Adverse Events [ Time Frame: up to 7 years ]
Original Secondary Outcome: Same as current
Information By: BioMarin Pharmaceutical
Dates:
Date Received: October 16, 2013
Date Started: October 2013
Date Completion: December 2022
Last Updated: May 10, 2016
Last Verified: May 2016