Effect of sapropterin on variations of blood phenylalanine and tyrosine over 24 hours and from day to day in patients with phenylketonuria
- Conditions
- phenylketonuriaPKU10021605
- Registration Number
- NL-OMON44967
- Lead Sponsor
- niversitair Medisch Centrum Groningen
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 13
- Males and females from 4 to 12 years of age incl. and 18 years and above.
- Diagnosed with phenylketonuria by newborn screening.
- Use of sapropterin as part of the treatment.
- Under good metabolic control; defined as 2/3 or 67% of the blood phenylalanine levels within target ranges during the last year.
- Concomitant disease which may preclude the participation in the study in the judgment of the investigator.
- Intercurrent illness which might influence the blood phenylalanine levels.
- Concomitant medication as mentioned in the Kuvan® SPC.
- Known hypersensitivity to Kuvan® or its excipients.
- Known hypersensitivity to other approved or non-approved formulations of tetrahydrobiopterin.
- Non-compliance with study procedures in the judgement of the investigator.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The mean standard deviations of the blood phenylalanine concentrations measured<br /><br>four times a day of 2 consecutive days and once a day on the 6 consecutive days<br /><br>of all participants compared between the sapropterin + diet treatment period<br /><br>and the diet alone treatment period.</p><br>
- Secondary Outcome Measures
Name Time Method