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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 : Recruiting
First Posted : January 12, 2021
Last Update Posted : February 26, 2024
Sponsor:
Information provided by (Responsible Party):
Medicure

Study Description
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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

Study Design
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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
Actual Study Start Date : February 16, 2024
Estimated Primary Completion Date : May 2025
Estimated Study Completion Date : May 2025

Resource links provided by the National Library of Medicine


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


Outcome Measures
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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)


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

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.
Contacts and Locations
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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


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

Locations
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United States, Alabama
Children's Hospital of Alabama Recruiting
Birmingham, Alabama, United States, 35233
Contact: Mackenzie Keith    205-934-8365    mkeith@uabmc.edu   
Principal Investigator: Monisha Goyal, MD         
United States, California
Lucile Packard Children's Hospital Recruiting
Palo Alto, California, United States, 94304
Contact: Rayann Solidum    415-424-1237    rsolidum@stanford.edu   
Principal Investigator: Courtney Wusthoff, MD         
United States, North Carolina
Duke Children's Hospital Recruiting
Durham, North Carolina, United States, 27705
Contact: Gloria Pinero    919-613-0767    gloria.pinero@duke.edu   
Principal Investigator: Muhammad Zafar, MD         
United States, Ohio
Akron's Children's Hospital Recruiting
Akron, Ohio, United States, 44308
Contact: Hilary Tonni    330-543-4734    HTonni@akronchildrens.org   
Principal Investigator: Matthew Ginsberg, MD         
Australia, Queensland
Queensland Children's Hospital Active, not recruiting
South Brisbane, Queensland, Australia
Sponsors and Collaborators
Medicure
More Information
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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: February 26, 2024
Last Verified: February 2024
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
Pyridoxal Phosphate
Vitamin B Complex
 Vitamins
Micronutrients
Physiological Effects of Drugs