RecruitingPhase 3

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

United States15 participantsStarted 2024-02-16

Plain-language 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.

Who can participate

SexALL

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Inclusion criteria

✓. Patients with confirmed PNPO deficiency via genetic analysis, whose seizures are typically controlled on P5P (oral) therapy.
✓. Male and/or female patients.
✓. Aged ≥2 years

Exclusion criteria

✕. The patient has any condition or abnormality which may, in the opinion of the Investigator, compromise the safety of the patient, or influence their ability to comply with study procedures.
✕. Known or suspected allergy to the trial drug or the relevant drugs given in the trial.
✕. Involvement in a clinical research study within 4 weeks prior to screening and/or prior enrollment in the study. Participation in observational registry studies is permitted.

What they're measuring

Overall survival time (time to death), including incidence of death at 12 months

Timeframe: 12 months

Trial details

NCT IDNCT04706013

SponsorMedicure

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-11

Contact for this trial

Laura Cole, Ph.D.

204-487-7412 lcole@medicure.com

View on ClinicalTrials.gov More Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency trials