Trial Review

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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
Scientific title
Study of Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency
Secondary ID [1] 0 0
MEND-PNPO 16002
Universal Trial Number (UTN)
Trial acronym
MEND-PNPO
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Pyridoxal Phosphate

Experimental: Single Arm Active - Pyridoxal 5'-Phosphate


Treatment: Drugs: Pyridoxal Phosphate
Oral tablets 50 mg
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Overall survival time (time to death), including incidence of death at 12 months
Timepoint [1] 0 0
12 months
Secondary outcome [1] 0 0
Frequency of seizures (including but not limited to status epilepticus)
Timepoint [1] 0 0
up to 12 months

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).
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
16/02/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/05/2025
Actual
Sample size
Target
15
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment postcode(s) [1] 0 0
- South Brisbane
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
North Carolina
Country [4] 0 0
United States of America
State/province [4] 0 0
Ohio

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Medicure
Address
Country

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04706013
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Pat Follows
Address 0 0
Country 0 0
Phone 0 0
204-594-3410
Fax 0 0
Email 0 0
pfollows@medicure.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04706013