Oral P5P for Treating PNPO Deficiency
Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency
Recruiting
Phase 3
Interventional Study
Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Sex
- Any
- Trial phase
- Phase 3
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 15 (estimated)
- Sponsor
- Medicure · Industry
Who this trial is looking for
This trial is looking for patients with PNPO deficiency who are currently taking P5P for their seizures. Participants will receive a pharmaceutical-grade version of P5P based on their usual dosage.
Are You a Good Fit for This Trial?
You may be able to join if
- I have been diagnosed with PNPO deficiency through genetic testing.
- I am at least 2 years old.
- I take P5P to control my seizures and have done so for at least 30 days.
- I am male or female.
- I have tried pyridoxine before without success.
- If I am under 18, my parent or guardian can give consent.
You may not be able to join if
- I have a condition that may affect my safety or ability to follow study rules.
- I am allergic to the trial drug or related medications.
- I participated in another clinical trial in the last 4 weeks.
Summarized in plain language from this trial's official eligibility criteria. The full criteria are further down this page — only the research team can confirm whether you qualify.
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
Check Your Eligibility
About This Trial
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, a…
Trial Locations
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Eligibility Criteria
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 an…
Contacts