Continuation Study With Oral MC-1 for Treatment of Patients With PNPO-Deficiency

What this study involves

The registry says this study is recruiting. Individual locations may have different availability.

Study type

Interventional

Phase

Phase 3

Age range

2 years - Not specified

Sex eligible

All sexes

Healthy volunteers

Not accepted

Participants

10 Estimated

These are starting points, not an eligibility decision. The research team must review the full requirements.

About the research

Continued access to MC-1 is required by subjects who have participated in the MEND-PNPO study, as no other pharmaceutical grade P5P is currently commercially available. The protocol duration is planned for until the finished product is commercially available in the respective countries where subjects are participating in the study.

Who may be able to participate?

The list below is the sponsor’s full registry wording. It can include conditions, prior treatment, test results and other requirements. A match in search does not mean you qualify.

Inclusion Criteria:

• The subject should have completed participation in the MEND-PNPO study (Protocol # 16002) and in the opinion of the Principal Investigator would benefit from long-term treatment with MC-1
• Written informed consent (by parent or guardian if under the age of 18)

Exclusion Criteria:

• 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.

Treatment, groups and participation

The registry may describe treatments and study duration without a complete visit calendar. Ask the study team for the exact schedule, procedures, risks, costs and alternatives. Keep your usual care plan unless your treating clinician tells you otherwise.

Assignment

Not applicable

Masking

No masking

What researchers are measuring

Outcome measures describe what is being studied. They are not proof that a treatment works.

Primary measures

Research locations (5)

The overall study status and a local site’s status can differ. Confirm that location’s availability before traveling.

5 locations

Study team and timeline

Lead sponsor

Medicure

Study start

Jul 15, 2025 Actual

Primary completion

Jul 15, 2029 Estimated

Study completion

Jul 15, 2029 Estimated

First posted

Nov 10, 2026 Actual

Last updated

Nov 10, 2026 Actual

Official study contacts