← Back to Search

Vitamin

Single Arm Active for Pyridoxine 5'-Phosphate Oxidase Deficiency (MEND-PNPO Trial)

Phase 3

Recruiting

Research Sponsored by Medicure

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months

Awards & highlights

MEND-PNPO Trial Summary

This trial aims to test the effectiveness of a new oral medication, P5P, for treating patients with a specific genetic disorder called PNPO deficiency. Currently, there is no commercially available pharmaceutical grade P

Who is the study for?

This trial is for patients with a confirmed genetic diagnosis of PNPO deficiency, who have their seizures typically controlled by taking oral P5P regularly. Both male and female patients can join if they've been on P5P for at least 30 days. Those previously treated unsuccessfully with pyridoxine may also participate but must not have taken it in the last 24 hours. Consent is required.Check my eligibility

What is being tested?

The study tests pharmaceutical grade Pyridoxal Phosphate (P5P) given orally to treat PNPO deficiency. Patients will continue their usual dose of P5P as prescribed by their doctors, but now using a pharmaceutical grade version that hasn't been commercially available before.See study design

What are the potential side effects?

Since this trial uses an established treatment regimen with a new pharmaceutical-grade product, side effects are expected to be similar to those experienced with current oral P5P therapy which could include gastrointestinal disturbances or sensory neuropathy.

MEND-PNPO Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Overall survival time (time to death), including incidence of death at 12 months

Secondary outcome measures

Frequency of seizures (including but not limited to status epilepticus)

MEND-PNPO Trial Design

1Treatment groups

Experimental Treatment

Group I: Single Arm ActiveExperimental Treatment1 Intervention

Pyridoxal 5'-Phosphate

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

MedicureLead Sponsor

7 Previous Clinical Trials

4,673 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are new patients currently being enrolled in this ongoing medical study?

"Indeed, the details on clinicaltrials.gov affirm that this research endeavor is actively pursuing candidates. Originally shared on February 16th, 2024 and most recently revised on February 20th, 2024, the investigation aims to enlist a total of 15 participants from two distinct locations."

Answered by AI

What is the upper limit of participants involved in this clinical study?

"Indeed, the details provided on clinicaltrials.gov imply that this study is actively enrolling participants. Originally shared on February 16th, 2024 and last revised on February 20th of the same year, the trial aims to recruit a total of 15 patients distributed across two distinct sites."

Answered by AI

Does the FDA endorse the use of Single Arm Active treatment protocol?

"According to our assessment at Power, the safety rating for Single Arm Active is graded as 3 due to its Phase 3 trial status, indicating a foundation of efficacy data and robust safety evidence."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2024. "Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04706013.