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Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU

NCT00838435 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 95

Last updated 2020-12-22

Study results available
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Summary

This multicenter, open label study is designed to evaluate the safety of Kuvan® and its effect on neurocognitive function, blood Phe concentration, and growth in children with PKU who are 0-6 years old.

Conditions

Interventions

DRUG

sapropterin dihydrochloride

A dose of 20 mg/kg will be administered dissolved in water or apple juice, based on subject's age and ability, and taken orally once daily with food.

Sponsors & Collaborators

Principal Investigators

  • Joshua Lilienstein, M.D. · BioMarin Pharmaceutical

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2009-02-28
Primary Completion
2018-09-14
Completion
2018-09-14

Countries

  • United States
  • Canada

Study Locations

More Related Trials

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 NCT00838435 on ClinicalTrials.gov