New hope for kids with rare seizure disorder: vitamin B6 therapy in final testing phase
NCT ID NCT04706013
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests an oral form of pyridoxal 5'-phosphate (P5P), a vitamin B6 derivative, for people with a rare genetic condition called PNPO deficiency. The condition causes hard-to-control seizures. The study will enroll 15 patients aged 2 years and older who already take P5P to manage seizures. Researchers will compare survival and seizure frequency to historical data from untreated patients. The goal is to provide a reliable pharmaceutical-grade P5P option.
Why investors are watching
Medicure is running a Phase 3 trial of pharmaceutical-grade pyridoxal 5'-phosphate (P5P) for patients with PNPO deficiency, a rare genetic condition. The trial enrolls only 15 participants and tests whether the drug works when patients take it as their doctors already prescribe. For a micro-cap company, this small trial is a major event because a positive result could lead to the first approved P5P product for a condition with no commercial option.
If it works: If the trial succeeds, Medicure could gain approval for the first pharmaceutical-grade P5P, giving it a market monopoly in a niche but desperate patient group. That approval would create a steady revenue stream from a drug with no current competitor.
If it fails: The trial could fail to show benefit or face delays, which would set the company back with no approved product and limited resources. Trials in rare diseases often fail, and with only 15 patients, a negative result would be hard to recover from.
AI-written from the trial record. Speculative, and not investment advice.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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About 15 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Feb 2024
- Expected to finish
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Nov 2026
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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Children (under 18), adults (18 to 64) and older adults (65 and over)
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. 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. 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: 1. 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. 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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
5 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Akron's Children's Hospital
RECRUITINGAkron, Ohio, 44308, United States
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Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
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Children's Hospital Colorado
RECRUITINGAurora, Colorado, 80045, United States
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Children's Hospital of Alabama
RECRUITINGBirmingham, Alabama, 35233, United States
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Duke Children's Hospital
ACTIVE_NOT_RECRUITINGDurham, North Carolina, 27705, United States
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Lucile Packard Children's Hospital
RECRUITINGPalo Alto, California, 94304, United States
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Queensland Children's Hospital
ACTIVE_NOT_RECRUITINGSouth Brisbane, Queensland, Australia