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

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Trial Status Completed

Trial Identifier

NCT01965912

Condition

Phenylketonuria

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial 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.

Eligibility Criteria

Birth Sex

All

Age

4 Years to 5 Years

Healthy Volunteers

No

Drug/Treatment:

Kuvan®

Phase:

Phase 4

Study Type:

Interventional

Number of Participants:

34

Study Started:

2013-10

Study Updated:

2023-04-24

Trial Locations

  • Research site

    Munich, , Germany

  • Research site

    Münster, , Germany

  • Research site

    Bologna, , Italy

  • Research site - Bambino Gesu

    Roma, , Italy

  • Research site - La Sapienza

    Roma, , Italy

  • Research Site

    Barcelona, , Spain

  • Research site

    Murcia, , Spain

  • Research site

    Santiago de Compostela, , Spain

  • Research site

    Birmingham, , United Kingdom

  • Research site

    Bristol, , United Kingdom

  • Research Site - Evelina

    London, , United Kingdom

  • Research site - GOSH

    London, , United Kingdom

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 day

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