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

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.

Timeline

Start

2024-02-16

Primary completion

2026-11

Completion

2026-11

Drugs

Indications