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

Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT04550572
• Other study ID #: 16002
• Status: Not yet recruiting
• Phase: Phase 3
• Start date: February 2021
• Est. completion date: June 2022

Study information

• Verified date: September 2020
• Source: Medicure
• Contact: Pat Follows
• Phone: 204-594-3410
• Email: pfollows[@]medicure.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

To evaluate the efficacy of oral P5P for chronic treatment of patients with confirmed PNPO deficiency.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 10
• Est. completion date: June 2022
• Est. primary completion date: April 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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 registry studies is permitted.

Related Conditions & MeSH terms

• Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency

Intervention

Drug:
• Pyridoxal 5'-Phosphate
Oral administration of Pyridoxal 5'-Phosphate given multiple times daily

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Medicure

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of death at 12 months		12 months
Secondary	Proportion of subjects remaining seizure free for 3 months from study drug initiation.		Baseline to 3 months
Secondary	Proportion of subjects remaining seizure free for 6 consecutive months from study drug initiation		Baseline to 6 months
Secondary	Proportion of subjects remaining seizure free for 9 consecutive months from study drug initiation		Baseline to 9 months
Secondary	Proportion of subjects remaining seizure free for 12 consecutive months from study drug initiation		Baseline to 12 months
Secondary	Frequency of seizures (per month) over treatment period		Baseline to 12 months
Secondary	Percentage of participants who completed or discontinued from the study		Baseline to 12 months