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

November 4, 2026 updated by: Medicure

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

10

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Australia
    • Queensland
      • Brisbane, Queensland, Australia
        • Recruiting
        • Queensland Children's Hospital and Health Service
    • Victoria
      • Melbourne, Victoria, Australia
        • Recruiting
        • Royal Children's Hospital
  • United States
    • California
      • Palo Alto, California, United States, 94304
        • Active, not recruiting
        • Lucile Packard Children's Hospital
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Active, not recruiting
        • Children's Hospital Colorado
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Active, not recruiting
        • Boston Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Treatment Arm

Oral MC-1
Drug: Pyridoxal 5'-Phosphate

Oral tablets

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time Frame: Through study completion, up to 4 years

All adverse and serious adverse events will be collected, along with monitoring for symptoms and signs of B6 intoxication and changes to liver enzymes (evaluation for potential hepatotoxicity).
Through study completion, up to 4 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Medicure

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 15, 2025

Primary Completion (Estimated)

July 15, 2029

Study Completion (Estimated)

July 15, 2029

Study Registration Dates

First Submitted

October 28, 2026

First Submitted That Met QC Criteria

November 4, 2026

First Posted (Actual)

November 10, 2026

Study Record Updates

Last Update Posted (Actual)

November 10, 2026

Last Update Submitted That Met QC Criteria

November 4, 2026

Last Verified

November 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 16002B

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Subscribe