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Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency (MEND-PNPO)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT04706013
Recruitment Status : Not yet recruiting
First Posted : January 12, 2021
Last Update Posted : April 6, 2022
Information provided by (Responsible Party):

Brief Summary:
The proposed clinical study is intended to evaluate oral P5P for the treatment of patients confirmed to have Pyridox(am)ine 5'-Phosphate Oxidase (PNPO) deficiency via genetic analysis. There is an unmet clinical need for pharmaceutical grade P5P, as to date none has been made commercially available. Patients will receive pharmaceutical grade P5P according to their normal oral P5P dosing regimen, as previously established by their physicians.

Condition or disease Intervention/treatment Phase
Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency Drug: Pyridoxal Phosphate Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Study of Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency
Estimated Study Start Date : October 2022
Estimated Primary Completion Date : November 2023
Estimated Study Completion Date : November 2023

Arm Intervention/treatment
Experimental: Single Arm Active
Pyridoxal 5'-Phosphate
Drug: Pyridoxal Phosphate
Oral tablets 50 mg

Primary Outcome Measures :
  1. Overall survival time (time to death), including incidence of death at 12 months [ Time Frame: 12 months ]
    The study group will be compared to a historical control group (without active treatment) for overall survival by using the method of survival analysis

Secondary Outcome Measures :
  1. Frequency of seizures (including but not limited to status epilepticus) [ Time Frame: up to 12 months ]
    The frequency of seize will be compared between different treatment periods of the current single arm: Pharmaceutical grade oral P5P treatment period vs period prior to any P5P treatment (if data available)

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. 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.

  2. Male and/or female patients.
  3. Patients with previous failed treatment on pyridoxine are eligible for the study (patient should be off pyridoxine for at least 24 hours).
  4. Written informed consent (by parent or guardian if under the age of 18).

Exclusion Criteria:

  1. 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.
  2. Known or suspected allergy to the trial drug or the relevant drugs given in the trial.
  3. 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04706013

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Contact: Pat Follows 204-594-3410 pfollows@medicure.com

Sponsors and Collaborators
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Responsible Party: Medicure
ClinicalTrials.gov Identifier: NCT04706013    
Other Study ID Numbers: MEND-PNPO 16002
First Posted: January 12, 2021    Key Record Dates
Last Update Posted: April 6, 2022
Last Verified: April 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Pyridoxal Phosphate
Vitamin B Complex
Physiological Effects of Drugs