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

NCT04706013 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2025-10-16

No results posted yet for this study

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

DRUG

Pyridoxal Phosphate

Oral tablets 50 mg

Sponsors & Collaborators

  • Medicure

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-02-16
Primary Completion
2026-11-30
Completion
2026-11-30
FDA Drug
Yes

Countries

  • United States
  • Australia

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04706013 on ClinicalTrials.gov