drugset / Trial / NCT07255599

Evaluation of BH4 Responsiveness in Our PKU Patients

NCT07255599

Phase 1 Active not recruiting 20 enrolled Sohag University
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

EvaluationDrugModalityDoseRoute
Comparator sapropterin Unknown 20 mg/kg

Indications