drugset / Trial / NCT06302348
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
NaSingle-groupOpen-labelTreatment
Summary
The main purpose of this trial is to evaluate the long-term efficacy of sepiapterin on preserving neurocognitive functioning in children with PKU when treatment is initiated in early childhood.
Timeline
- Start
- 2024-03-04
- Primary completion
- 2031-02-28
- Completion
- 2031-02-28
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Sepiapterin | Small molecule | — | Oral |