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Kuvan® in Phenylketonuria Patients Less Than 4 Years Old

NCT01376908 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 56

Last updated 2017-09-15

Study results available
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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).

Conditions

Interventions

DRUG

Kuvan®

Kuvan® (sapropterin dihydrochloride) tablets will be administered orally at the dose of 10 mg/kg/day and will be escalated to 20 mg/kg/day if after 4 weeks a subject's Phe tolerance is not increased by at least 20% versus baseline.

OTHER

Phenylalanine (Phe)-restricted diet

Phe intake will be adjusted every 2 weeks, based on the mean Phe levels of the previous 2 weeks using pre-defined Phe adjustment criteria.

Sponsors & Collaborators

Principal Investigators

  • Ignacio Alvarez, MD · BioMarin Pharmaceutical

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Max Age
4 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-06-30
Primary Completion
2014-07-31
Completion
2017-02-17

Countries

  • Austria
  • Belgium
  • Czechia
  • Germany
  • Italy
  • Netherlands
  • Slovakia
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

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Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01376908 on ClinicalTrials.gov