Status:
RECRUITING
Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency
Lead Sponsor:
Medicure
Conditions:
Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency
Eligibility:
All Genders
Phase:
PHASE3
Brief 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 unm...
Eligibility Criteria
Inclusion
- Patients with confirmed PNPO deficiency via genetic analysis, whose seizures are typically controlled on P5P (oral) therapy.
- a. Typically controlled is defined as receiving multiple doses of P5P daily to control seizures. Receiving P5P for a minimum of 30 days.
- Male and/or female patients.
- Aged ≥2 years
- (3) Patients with previous failed treatment on pyridoxine are eligible for the study (patient should be off pyridoxine for at least 24 hours).
- (4) Written informed consent (by parent or guardian if under the age of 18).
Exclusion
- 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.
Key Trial Info
Start Date :
February 16 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
November 1 2026
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT04706013
Start Date
February 16 2024
End Date
November 1 2026
Last Update
October 16 2025
Active Locations (7)
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1
Children's Hospital of Alabama
Birmingham, Alabama, United States, 35233
2
Lucile Packard Children's Hospital
Palo Alto, California, United States, 94304
3
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
4
Boston Children's Hospital
Boston, Massachusetts, United States, 02115