Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency
NCT ID: NCT04706013
Last Updated: 2025-10-16
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
15 participants
INTERVENTIONAL
2024-02-16
2026-11-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Single Arm Active
Pyridoxal 5'-Phosphate
Pyridoxal Phosphate
Oral tablets 50 mg
Interventions
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Pyridoxal Phosphate
Oral tablets 50 mg
Eligibility Criteria
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Inclusion Criteria
a. Typically controlled is defined as receiving multiple doses of P5P daily to control seizures. Receiving P5P for a minimum of 30 days.
2. Male and/or female patients.
3. 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 Criteria
2. Known or suspected allergy to the trial drug or the relevant drugs given in the trial.
3. 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.
ALL
No
Sponsors
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Medicure
INDUSTRY
Responsible Party
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Locations
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Children's Hospital of Alabama
Birmingham, Alabama, United States
Lucile Packard Children's Hospital
Palo Alto, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Duke Children's Hospital
Durham, North Carolina, United States
Akron's Children's Hospital
Akron, Ohio, United States
Queensland Children's Hospital
South Brisbane, Queensland, Australia
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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MEND-PNPO 16002
Identifier Type: -
Identifier Source: org_study_id
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