Trials / Completed
CompletedNCT01965912
Kuvan®'s Effect on the Cognition of Children With Phenylketonuria
A Phase 4 Open-Label, Single-Cohort Study of the Long-Term Neurocognitive Outcomes in 4 to 5 Year-Old Children With Phenylketonuria Treated With Sapropterin Dihydrochloride (Kuvan®) for 7 Years
- Status
- Completed
- Phase
- Phase 4
- Study type
- Interventional
- Enrollment
- 34 (actual)
- Sponsor
- BioMarin Pharmaceutical · Industry
- Sex
- All
- Age
- 4 Years – 5 Years
- Healthy volunteers
- Not accepted
Summary
The study is a single-cohort, interventional, open-label trial to evaluate long-term neurocognitive (NC) outcomes in children aged 4 to 5 years with phenylketonuria (PKU) treated with Kuvan® and Phenylalanine-restricted diet over a period of 7 years.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Kuvan® | Kuvan® oral soluble tablet will be administered once daily along with Phenylalanine-restricted diet for a period of 7 years. Dosage of Kuvan® could range from 5 to 20 milligram per kilogram per day (mg/kg/day), as per Summary of Product Characteristics (SPC). |
Timeline
- Start date
- 2013-10-01
- Primary completion
- 2023-01-04
- Completion
- 2023-01-04
- First posted
- 2013-10-18
- Last updated
- 2023-04-24
Locations
12 sites across 4 countries: Germany, Italy, Spain, United Kingdom
Source: ClinicalTrials.gov record NCT01965912. Inclusion in this directory is not an endorsement.