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Clinical Trial 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.


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms

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

NCT number NCT04706013
Study type Interventional
Source Medicure
Contact Pat Follows
Phone 204-594-3410
Email pfollows@medicure.com
Status Recruiting
Phase Phase 3
Start date February 16, 2024
Completion date May 2025

See also
  Status Clinical Trial Phase
Not yet recruiting NCT04550572 - Study of Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency Phase 3