Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Ongoing, recruiting
Participants planned
3
Countries
1
Sites
3
Seizures associated with Pyridox(am)ine 5’-Phosphate Oxidase (PNPO) deficiency
To evaluate the efficacy of MC-1 for chronic treatment of patients with confirmed PNPO deficiency.
Key facts
- Sponsor
- Medicure International Inc.
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Trial duration
- 20 Jan 2026 → ongoing
- Decision date (initial)
- 2025-11-21
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Therapy, Prophylaxis, Efficacy, Safety
To evaluate the efficacy of MC-1 for chronic treatment of patients with confirmed PNPO deficiency.
Secondary objectives 1
- To evaluate the safety of MC-1. To prospectively document the natural history of PNPO disorder
Conditions and MedDRA coding
Seizures associated with Pyridox(am)ine 5’-Phosphate Oxidase (PNPO) deficiency
Regulatory references
- Scientific advice from competent authorities
- Food And Drug Administration
- Plan to share IPD
- No
- IPD plan description
- Individual participant data (IPD) will not be shared due to the nature of the study and data protection considerations. The study results will be published in aggregated form only, ensuring transparency while maintaining participant confidentiality. No requests for IPD sharing are anticipated, and no infrastructure for secure data sharing is in place.
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 5
- 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.
- Patients with previous failed treatment on pyridoxine are eligible for the study (patient should be off pyridoxine for at least 24 hours).
- Written informed consent (by parent or guardian if under the age of 18).
Exclusion criteria 4
- 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.
- Pregnant female, or at risk of becoming pregnant (a negative pregnancy test and documented confirmation of contraception is required for females of child- bearing potential).
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Incidence of death at 12 months. The incidence of death at 12 months will be compared between the study group and an external control group (without any P5P treatment).
Secondary endpoints 1
- Frequency of seizures (including but not limited to status epilepticus)
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD12674612 · Product
- Active substance
- Pyridoxal Phosphate
- Substance synonyms
- PYRIDOXAL 5-PHOSPHATE, Pyridoxal 5'-phosphate
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL
- Max daily dose
- 3 g gram(s)
- Max total dose
- 1095 g gram(s)
- Max treatment duration
- 52 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- MEDICURE
- Paediatric formulation
- No
- Orphan designation
- Yes
- Orphan designation number
- EU/3/18/1983
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Medicure International Inc.
- Sponsor organisation
- Medicure International Inc.
- Address
- Floor 1st, Limegrove Centre Limegrove Centre
- City
- Holetown
- Country
- Barbados
Scientific contact point
- Organisation
- Medicure International Inc.
- Contact name
- Marek Sawicki
Public contact point
- Organisation
- Medicure International Inc.
- Contact name
- Global Medical Services Polska Sp. z o.o.
Locations
1 EU/EEA country · 3 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Poland | Ongoing, recruiting | 3 | 3 |
| Rest of world | — | 0 | — |
Investigational sites
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Poland | 2026-01-20 | 2026-02-03 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 11 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol 2025-522346-46-00 | 3.1 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent and Adult Assent 13 PL | 2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF adults PL | 3 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child Assent 7-12 PL | 2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Pregnant Participant PL | 2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Pregnant Participant - Responsible Person PL | 2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF responsible person PL | 3 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis 2025-522346-46-00 | 3.1 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_PL 2025-522346-46-00 | 3.1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2025-07-31 | Poland | Acceptable 2025-11-17
|
2025-11-21 |