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Kuvan® in Phenylketonuria Patients Less Than 4 Years Old

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Disease Information

Descriptive Information
Brief Title † Kuvan® in Phenylketonuria Patients Less Than 4 Years Old
Official Title † A Phase IIIb, Multicentre, Open-Label, Randomized, Controlled Study of the Efficacy, Safety, and Population Pharmacokinetics of Sapropterin Dihydrochloride (Kuvan®) in Phenylketonuria (PKU) Patients
Brief Summary This is a Phase 3b, multicenter, open-label, randomized, controlled study to evaluate efficacy, safety and population pharmacokinetics of sapropterin dihydrochloride (Kuvan®) in less than 4 year-old infants and children with Phenylketonuria (PKU).
Detailed Description
Study Phase Phase 3
Study Type † Interventional
Study Design † Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary Outcome Measure † Dietary Phe tolerance after 6 months (26 weeks) of treatment with Kuvan® + a Phe -restricted diet versus just a Phe-restricted diet alone
Secondary Outcome Measure † Levels of blood Phe
Condition † Phenylketonuria
Intervention † DrugKuvan®
Study Arms / Comparison Groups Kuvan® + Phe-restricted diet Subjects will be treated with Kuvan® tablets once daily + Phe-restricted diet therapy Phe-restricted diet alone Subjects will follow a Phe-restricted diet alone
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 † 56
Start Date † June 2011
Completion Date February 2017
Primary Completion Date July 2014
Eligibility Criteria † Inclusion Criteria: - Male or female PKU infants and young children =) 400 mcmol/L obtained on 2 separate occasions - Previously responded, as assessed by the Investigator, to a tetrahydrobiopterin (BH4) test, if all 3 of the following criteria are satisfied: 1. The BH4 dose was 20 mg/kg/day 2. The duration of the test was at least for 24 hours 3. A 30% decrease in blood Phe levels. - Defined level of dietary Phe tolerance consistent with the diagnosis of PKU - Good adherence to dietary treatment, including prescribed dietary Phe restriction and prescribed amounts of Phe-free protein supplements and low-Phe foods - Maintenance of blood Phe levels within the therapeutic target range of 120-360 mcmol/L (defined as >=120 to )30 days - Known hypersensitivity to Kuvan® or its excipients - Known hypersensitivity to other approved or non-approved formulations of tetrahydrobiopterin - 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 levodopa - Current use of experimental/other investigational or unregistered drugs that may affect the study outcomes - Inability to comply with study procedures - Inability to tolerate oral intake - History of organ transplantation - Concurrent disease or condition that would interfere with study participation or increase the risk for adverse events, including seizure disorders, corticosteroid administration, active malignancy, diabetes mellitus, severe congenital heart disease, renal or hepatic failure - Other significant disease that in the Investigator's opinion would exclude the subject from the trial - Any condition that, in the view of the Principal Investigator renders the subject at high risk for failure to comply with treatment or to complete the study
Gender Both
Ages N/A - 4 Years
Accepts Healthy Volunteers No
Contacts ††
Location Countries † Austria
Administrative Information
NCT ID † NCT01376908
Organization ID EMR 700773-003
Secondary IDs †† 2009-015768-33
Responsible Party Sponsor
Study Sponsor † Merck KGaA
Collaborators ††
Investigators † Study Director: Medical Responsible, Merck Serono, a division of Merck KGaA, Darmstadt, Germany
Information Provided By
Verification Date August 2014
First Received Date † June 17, 2011
Last Updated Date August 13, 2014
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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