FindTrial
Sign In

A Phase IV, prospective, open-label, uncontrolled, single-centre cohort trial to be conducted in Norway to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 7 days. - Response to Kuvan® in subjects with PKU from a single centre in Norway

A Phase IV, prospective, open-label, uncontrolled, single-centre cohort trial to be conducted in Norway to assess the responsiveness of subjects with phenylketonuria (PKU) to treatment with Kuvan® 20 mg/kg/day for 7 days. - Response to Kuvan® in subjects with PKU from a single centre in Norway

Status
Active, not recruiting
Phases
Phase 4
Study type
Interventional
Source
EU CTR
Registry ID
EUCTR2009-012978-12-NO
Enrollment
Unknown
Registered
2009-06-02
Start date
Unknown
Completion date
Unknown
Last updated
2017-10-02

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hyperphenylalaninemia (HPA) in adults and children >=16 years of age with phenylketonuri (PKU) (classic or mild PKU, or mild hyperphenylalaninemia (HPA)). MedDRA version: 9.1 Level: LLT Classification code 10034872 Term: Phenylketonuria MedDRA version: 9.1 Level: LLT Classification code 10034873 Term: Phenylketonuria (PKU) MedDRA version: 9.1 Level: PT Classification code 10034872 Term: Phenylketonuria MedDRA version: 9.1 Level: LLT Classification code 10034871 Term: Phenylalaninemia

Interventions

Trade Name: Kuvan® (sapropterin dihydrochloride) Pharmaceutical Form: Soluble tablet INN or Proposed INN: sapropterin Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentr

Sponsors

Merck Serono, an affiliate of E. Merck AB
Lead Sponsor

Eligibility

Sex/Gender
All

Inclusion criteria

Inclusion criteria: Subjects will be included if they meet the following requirements: • aged between =16 years and 65 years at the screening visit • diagnosed with PKU (classic PKU, mild PKU or mild HPA) • previously untreated with sapropterin dihydrochloride • baseline blood PHE measurement =400 µmol/L • agreed to comply with study procedures • provided written informed consent. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes 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

Exclusion criteria: Main exclusion criteria are: • documented BH4 deficiency • pregnant, planning pregnancy or breastfeeding.

Design outcomes

Primary

MeasureTime frame
Main Objective: The primary objective is to identify subjects with PKU responsive to Kuvan® (=30% reduction from baseline in blood PHE level) in a single centre in Norway using the 24-hour BH4 loading test.;Secondary Objective: The secondary objectives are to compare the response to Kuvan® in subjects with PKU after the 24-hour BH4 loading test with the response to Kuvan® after 7 days of treatment, and to compare the response to Kuvan® between subjects with differing PKU genotypes and phenotypes.;Primary end point(s): The primary endpoint is the proportion of subjects with a =30% reduction from baseline in blood PHE level (responders) to 24 hours after treatment with Kuvan® 20 mg/kg (the BH4- loading test).—

Countries

Norway

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026