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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT04706013
Registration number
NCT04706013
Ethics application status
Date submitted
8/01/2021
Date registered
12/01/2021
Date last updated
26/02/2024
Titles & IDs
Public title
Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency
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Scientific title
Study of Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency
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Secondary ID [1]
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MEND-PNPO 16002
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Universal Trial Number (UTN)
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Trial acronym
MEND-PNPO
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency
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Condition category
Condition code
Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Pyridoxal Phosphate
Experimental: Single Arm Active - Pyridoxal 5'-Phosphate
Treatment: Drugs: Pyridoxal Phosphate
Oral tablets 50 mg
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Overall survival time (time to death), including incidence of death at 12 months
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Assessment method [1]
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The study group will be compared to a historical control group (without active treatment) for overall survival by using the method of survival analysis
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Timepoint [1]
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12 months
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Secondary outcome [1]
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Frequency of seizures (including but not limited to status epilepticus)
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Assessment method [1]
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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)
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Timepoint [1]
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up to 12 months
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Eligibility
Key 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).
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Minimum age
No limit
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
16/02/2024
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/05/2025
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Actual
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Sample size
Target
15
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
QLD
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Recruitment hospital [1]
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Queensland Children's Hospital - South Brisbane
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Recruitment postcode(s) [1]
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- South Brisbane
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Alabama
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Country [2]
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United States of America
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State/province [2]
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California
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Country [3]
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United States of America
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State/province [3]
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North Carolina
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Country [4]
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United States of America
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State/province [4]
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Ohio
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Medicure
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT04706013
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Pat Follows
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Address
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Country
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Phone
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204-594-3410
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT04706013
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