Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency
Start Date
2/16/2024
Completion Date
11/1/2026
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.
Eligibility Criteria
Age Range: No minimum to No maximum
Interventions
Pyridoxal Phosphate
Conditions
Locations
Children's Hospital of Alabama
Birmingham, Alabama 35233
United States
Lucile Packard Children's Hospital
Palo Alto, California 94304
United States
Children's Hospital Colorado
Aurora, Colorado 80045
United States
Boston Children's Hospital
Boston, Massachusetts 02115
United States
Duke Children's Hospital
Durham, North Carolina 27705
United States
Akron's Children's Hospital
Akron, Ohio 44308
United States
Queensland Children's Hospital
South Brisbane, Queensland
Australia