NCT07928388 Continuation Study With Oral MC-1 for Treatment of Patients With PNPO-Deficiency
| NCT ID | NCT07928388 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Medicure |
| Condition | Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency |
| Study Type | INTERVENTIONAL |
| Enrollment | 10 participants |
| Start Date | 2025-07-15 |
| Primary Completion | 2029-07-15 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 10 participants in total. It began in 2025-07-15 with a primary completion date of 2029-07-15.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
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.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT07928388 clinical trial?
This trial is open to participants of all sexes, aged 2 Years or older, studying Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07928388 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 10 participants.
Is NCT07928388 currently recruiting?
Yes, NCT07928388 is actively recruiting participants. Contact the research team at lcole@medicure.com for enrollment information.
Where is the NCT07928388 trial being conducted?
This trial is being conducted at Palo Alto, United States, Aurora, United States, Boston, United States, Brisbane, Australia and 1 additional location.
Who is sponsoring the NCT07928388 clinical trial?
NCT07928388 is sponsored by Medicure. The trial plans to enroll 10 participants.