Study of Oral MC-1 for the Treatment of Patients with PNPO Deficiency
Key facts
- Where
- Gdansk, Lodz és Wroclaw, LengyelországSee the 3 sites
- Phase
- 3. fázis
Where in Lengyelország
- Uniwersyteckie Centrum KliniczneGdansk, LengyelországTaking people
- Instytut Centrum Zdrowia Matki PolkiLodz, LengyelországTaking people
- Wroclaw Medical UniversityWroclaw, LengyelországTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Medicure International Inc.
- Trial code
- EU 2025-522346-46-00
- Record updated
- 2025. november 21.
- Original description
To evaluate the efficacy of MC-1 for chronic treatment of patients with confirmed PNPO deficiency.Read full original descriptionShow less
To evaluate the efficacy of MC-1 for chronic treatment of patients with confirmed PNPO deficiency.
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; 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: 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; 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; 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).