Kuvan®'s Effect on the Cognition of Children With Phenylketonuria

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Disease Information

Descriptive Information
Brief Title † Kuvan®'s Effect on the Cognition of Children With Phenylketonuria
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 Dihydrochloride (Kuvan®) for 7 Years
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 Description
Study Phase Phase 4
Study Type † Interventional
Study Design † Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary Outcome Measure † Mean Full Scale Intelligence Quotient (FSIQ) Score of the Wechsler Intelligence Scale for Children (WISC)-IV
Secondary Outcome Measure † Height compared to the World Health Organization (WHO) Growth Standards
Condition † Phenylketonuria
Intervention † DrugKuvan®
Study Arms / Comparison Groups Kuvan®
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Drug
Estimated Enrollment † 50
Start Date † October 2013
Completion Date December 2022
Primary Completion Date November 2022
Eligibility Criteria † Inclusion Criteria: - Male or female outpatients, 4 to 5 years of age (greater than or equal to 4 and less than 6) at the time of informed consent form signature by parent(s) or guardian(s) - Confirmed clinical and biochemical diagnosis of PKU, including at least two separate blood phenylalanine levels greater than or equal to 400 micromole per liter (mcmol/L) - Defined pre-Kuvan®/-tetrahydrobiopterin (BH4) dietary phenylalanine tolerance consistent with the diagnosis of PKU - Responsive to Kuvan®/BH4: - For subjects currently treated with Kuvan®/BH4 at Screening: subject is a responder as per Investigator judgment based on documented effect of Kuvan®/BH4 on phenylalanine levels and/or phenylalanine tolerance - For subjects not treated with Kuvan®/BH4 at Screening: a response test has been performed during Screening or is available from the subject's medical records and satisfies the 3 following criteria: a decrease in blood phenylalanine levels of at least 30 percent was observed after at least 24 hours with a dose of at least 10 mg/kg/day - Intelligence Quotient (IQ) greater than or equal to 70, as assessed with the Wechsler Preschool and Primary Scale of Intelligence (WPPSI)-III, 2nd part - Good adherence with dietary treatment (including prescribed dietary phenylalanine restriction and prescribed amounts of phenylalanine-free protein supplements and low-phenylalanine foods), as assessed by the Investigator - Well-controlled phenylalanine levels, as assessed by a minimum of 75 percent of phenylalanine levels within the target recommended in each centre during the previous 3 months - Low phenylalanine diet started within the first 3 weeks of life - Parent(s) or guardian(s) willing to comply with all study procedures, maintain strict adherence with the diet, and willing and able to provide written, signed informed consent before any trial-related activities are carried out, as well as ability of child to comply with trial procedures Exclusion Criteria: - Known hypersensitivity to Kuvan® or its excipients - Known hypersensitivity to other approved or non-approved formulations of BH4 - Previous diagnosis of BH4 deficiency - Current use of methotrexate, trimethoprim or other dihydrofolate reductase inhibitors - Current use of medications that are known to affect nitric oxide synthesis, metabolism or action - Current use of experimental or unregistered drugs (other than sapropterin/BH4) that may affect the study outcomes or use of such agents within 30 days prior to Screening - Concurrent use of levodopa - Concurrent disease or condition that would induce repeatedly catabolic situations, or interfere with the trial participation, diet, or NC development, as assessed by the Investigator - Any condition that, in the view of the Investigator, renders the subject at high risk for failure to comply with treatment or to complete the trial - Participation in a clinical trial investigating any other agent than Kuvan® within the past 30 days
Gender Both
Ages 4 Years - 5 Years
Accepts Healthy Volunteers No
Contacts †† Merck KGaA Communication Center, +49 6151 72 5200,
Location Countries † Germany
Administrative Information
NCT ID † NCT01965912
Organization ID EMR 700773-002
Secondary IDs †† 2009-015844-41
Responsible Party Sponsor
Study Sponsor † Merck KGaA
Collaborators ††
Investigators † Study Director: Medical Responsible, Merck KGaA
Information Provided By
Verification Date August 2015
First Received Date † October 16, 2013
Last Updated Date August 11, 2015
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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