- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04706013
Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency (MEND-PNPO)
February 22, 2024 updated by: Medicure
Study of Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency
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.
Study Overview
Status
Recruiting
Intervention / Treatment
Study Type
Interventional
Enrollment (Estimated)
15
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
- Name: Pat Follows
- Phone Number: 204-594-3410
- Email: pfollows@medicure.com
Study Locations
-
-
Queensland
-
South Brisbane, Queensland, Australia
- Active, not recruiting
- Queensland Children's Hospital
-
-
-
-
Alabama
-
Birmingham, Alabama, United States, 35233
- Recruiting
- Children's Hospital of Alabama
-
Contact:
- Mackenzie Keith
- Phone Number: 205-934-8365
- Email: mkeith@uabmc.edu
-
Principal Investigator:
- Monisha Goyal, MD
-
-
California
-
Palo Alto, California, United States, 94304
- Recruiting
- Lucile Packard Children's Hospital
-
Contact:
- Rayann Solidum
- Phone Number: 415-424-1237
- Email: rsolidum@stanford.edu
-
Principal Investigator:
- Courtney Wusthoff, MD
-
-
North Carolina
-
Durham, North Carolina, United States, 27705
- Recruiting
- Duke Children's Hospital
-
Principal Investigator:
- Muhammad Zafar, MD
-
Contact:
- Gloria Pinero
- Phone Number: 919-613-0767
- Email: gloria.pinero@duke.edu
-
-
Ohio
-
Akron, Ohio, United States, 44308
- Recruiting
- Akron's Children's Hospital
-
Contact:
- Hilary Tonni
- Phone Number: 330-543-4734
- Email: HTonni@akronchildrens.org
-
Principal Investigator:
- Matthew Ginsberg, MD
-
-
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
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
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.
- Male and/or female patients.
- Patients with previous failed treatment on pyridoxine are eligible for the study (patient should be off pyridoxine for at least 24 hours).
- 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.
- Known or suspected allergy to the trial drug or the relevant drugs given in the trial.
- 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.
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: Single Arm Active
Pyridoxal 5'-Phosphate
|
Oral tablets 50 mg
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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
|
12 months
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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)
|
up to 12 months
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
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)
February 16, 2024
Primary Completion (Estimated)
May 1, 2025
Study Completion (Estimated)
May 1, 2025
Study Registration Dates
First Submitted
January 8, 2021
First Submitted That Met QC Criteria
January 11, 2021
First Posted (Actual)
January 12, 2021
Study Record Updates
Last Update Posted (Actual)
February 26, 2024
Last Update Submitted That Met QC Criteria
February 22, 2024
Last Verified
February 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- MEND-PNPO 16002
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.
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