drugset / Trial / NCT01965912
Kuvan®'s Effect on the Cognition of Children With Phenylketonuria
NaSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2013-10
- Primary completion
- 2023-01-04
- Completion
- 2023-01-04
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | sapropterin | Unknown | 5 mg/kg | Oral |
| Subject | sapropterin | Unknown | 20 mg/kg | Oral |