drugset / Trial / NCT07255599
Evaluation of BH4 Responsiveness in Our PKU Patients
Non-randomizedSingle-groupOpen-labelDiagnostic
Summary
This study aims to evaluate BH4 responsiveness in our PKU patients and to correlate BH4 responsiveness with their genotype
Timeline
- Start
- 2025-09-01
- Primary completion
- 2026-01-01
- Completion
- 2026-09-01
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Comparator | sapropterin | Unknown | 20 mg/kg | — |