NCT04706013

Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency

Study of Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 15 (estimated)
• Sponsor: Medicure · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

The proposed clinical study is intended to evaluate oral P5P for the treatment of patients confirmed to have Pyridox(am)ine 5'-Phosphate Oxidase (PNPO) deficiency via genetic analysis. There is an unmet clinical need for pharmaceutical grade P5P, as to date none has been made commercially available. Patients will receive pharmaceutical grade P5P according to their normal oral P5P dosing regimen, as previously established by their physicians.

Conditions

• Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency

Interventions

Type	Name	Description
DRUG	Pyridoxal Phosphate	Oral tablets 50 mg

Timeline

• Start date: 2024-02-16
• Primary completion: 2026-11-01
• Completion: 2026-11-01
• First posted: 2021-01-12
• Last updated: 2025-10-16

Locations

7 sites across 2 countries: United States, Australia

Regulatory

• FDA-regulated drug study