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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 31 October 2016
Main ID:  EUCTR2010-021343-41-NL
Date of registration: 30/11/2011
Prospective Registration: Yes
Primary sponsor: University Medical Center Groningen
Public title: Variations of blood phenylalanine and tyrosine in children with phenylketonuria under sapropterin
Scientific title: Effect of sapropterin on variations of blood phenylalanine and tyrosine over 24 hours and from day to day in children with phenylketonuria
Date of first enrolment: 21/03/2014
Target sample size:
Recruitment status: Authorised-recruitment may be ongoing or finished
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-021343-41
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: no Randomised: yes Open: yes Single blind: no Double blind: no Parallel group: no Cross over: yes Other: no If controlled, specify comparator, Other Medicinial Product: Placebo: Other:  
Phase:  Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): yes
Countries of recruitment
Netherlands
Contacts
Name: Independent expert   
Address:  Hanzeplein 1 9713 GZ Groningen Netherlands
Telephone: 00310503614147
Email:
Affiliation:  University Medical Center Groningen
Name: Independent expert   
Address:  Hanzeplein 1 9713 GZ Groningen Netherlands
Telephone: 00310503614147
Email:
Affiliation:  University Medical Center Groningen
Key inclusion & exclusion criteria
Inclusion criteria:
- Males and females 4-12 years of age.
- Diagnosed with phenylketonuria by newborn screening.
- Tested to be sapropterin responsive.
- Under good metabolic control; defined as 2/3 or 67% of the blood phenylalanine levels within target ranges during the last year.

Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
- 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 KuvanĀ® SPC.
- Known hypersensitivity to KuvanĀ® 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.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Phenylketonuria (PKU) is an autosomal recessive metabolic genetic disorder by a mutation in the gene for the enzyme phenylalanine hydroxylase (PAH), rendering it nonfunctional. Left untreated, the disease will result in high concentrations of phenylalanine (Phe) in blood and tissues, likely resulting in severe mental retardation and behavioural problems. Treatment focusus on the restriction of dietary phenylalanine intake with supplementation of a synthetic phenylalaninefree amino acid mixture.
Intervention(s)

Trade Name: Kuvan
Pharmaceutical Form: Soluble tablet

Primary Outcome(s)
Primary end point(s): The mean standard deviations of the blood phenylalanine concentrations measured four times a day of 2 consecutive days and once a day on 8 consecutive days of all participants compared between the sapropterin + diet treatment period and the diet alone treatment period.
Timepoint(s) of evaluation of this end point: In both study periods blood sampling is scheduled for the first six days once a day (7-8 am) and four times daily (7-8 am, 12-1 pm, 5-6 pm and bedtime) on the two days thereafter, in total 28 samples per patient.
Secondary Objective: - To measure the effect of sapropterin on diurnal and day to day variations of blood tyrosine concentrations.
- To measure the effect of sapropterin on diurnal and day to day variations of blood phenylalanine/tyrosine ratios.
Main Objective: To measure the effect of sapropterin on diurnal and day to day variations of blood phenylalanine concentrations.
Secondary Outcome(s)
Timepoint(s) of evaluation of this end point: In both study periods blood sampling is scheduled for the first six days once a day (7-8 am) and four times daily (7-8 am, 12-1 pm, 5-6 pm and bedtime) on the two days thereafter, in total 28 samples per patient.
Secondary end point(s): - The mean standard deviations of the blood tyrosine concentrations measured once a day on 8 consecutive days of all participants compared between the sapropterin + diet treatment period and the diet alone treatment period.
- The mean standard deviations of the blood tyrosine concentrations measured four times a day of 2 consecutive days of all participants compared between the sapropterin + diet treatment period and the diet alone treatment period.
- The mean standard deviations of the blood phenylalanine/tyrosine ratios measured once a day on 8 consecutive days of all participants compared between the sapropterin + diet treatment period and the diet alone treatment period.
- The mean standard deviations of the blood phenylalanine/tyrosine concentrations measured four times a day of 2 consecutive days of all participants compared between the sapropterin + diet treatment period and the diet alone treatment period.
Secondary ID(s)
ME040
Source(s) of Monetary Support
Merck KGaA
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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