RECRUITING

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

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Conditions

Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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

Quick Facts

Study Start:2024-02-16
Study Completion:2025-05
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT04706013

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.

Ages Eligible for Study:Not specified
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. Patients with confirmed PNPO deficiency via genetic analysis, whose seizures are typically controlled on P5P (oral) therapy.
  2. 2. Male and/or female patients.
  3. 3. Patients with previous failed treatment on pyridoxine are eligible for the study (patient should be off pyridoxine for at least 24 hours).
  4. 4. Written informed consent (by parent or guardian if under the age of 18).
  1. 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. 2. Known or suspected allergy to the trial drug or the relevant drugs given in the trial.
  3. 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.

Contacts and Locations

Study Contact

Pat Follows
CONTACT
204-594-3410
pfollows@medicure.com

Study Locations (Sites)

Children's Hospital of Alabama
Birmingham, Alabama, 35233
United States
Lucile Packard Children's Hospital
Palo Alto, California, 94304
United States
Duke Children's Hospital
Durham, North Carolina, 27705
United States
Akron's Children's Hospital
Akron, Ohio, 44308
United States

Collaborators and Investigators

Sponsor: Medicure

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 Registration Dates

Study Start Date2024-02-16
Study Completion Date2025-05

Study Record Updates

Study Start Date2024-02-16
Study Completion Date2025-05

Terms related to this study

Additional Relevant MeSH Terms

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