Actively Recruiting
Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency
Led by Medicure · Updated on 2025-10-16
15
Participants Needed
7
Research Sites
141 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients with confirmed PNPO deficiency via genetic analysis whose seizures are typically controlled on oral P5P therapy
- Receiving multiple doses of P5P daily for seizure control for at least 30 days
- Male or female patients
- Aged 2 years or older
- Patients with previous failed pyridoxine treatment are eligible if off pyridoxine for at least 24 hours
- Written informed consent provided by patient or guardian if under 18 years old
You will not qualify if you...
- Any condition or abnormality that may compromise patient safety or ability to comply with study procedures as judged by the Investigator
- Known or suspected allergy to the trial drug or related drugs
- Participation in another clinical research study within 4 weeks prior to screening or prior enrollment in this study (participation in observational registry studies is allowed)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 7 locations
1
Children's Hospital of Alabama
Birmingham, Alabama, United States, 35233
Actively Recruiting
2
Lucile Packard Children's Hospital
Palo Alto, California, United States, 94304
Actively Recruiting
3
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
4
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
5
Duke Children's Hospital
Durham, North Carolina, United States, 27705
Active, Not Recruiting
6
Akron's Children's Hospital
Akron, Ohio, United States, 44308
Actively Recruiting
7
Queensland Children's Hospital
South Brisbane, Queensland, Australia
Active, Not Recruiting
Research Team
L
Laura Cole, Ph.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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