This clinical trial studies the safety, tolerability, and pharmacology of asfotase alfa when given to adults with HPP.
Asfotase alfa was formerly referred to as ENB-0040 Hypophosphatasia (HPP) is a life-threatening, genetic, and ultra-rare metabolic disease characterized by defective bone mineralization and impaired phosphate and calcium regulation that can lead to progressive damage to multiple vital organs, including destruction and deformity of bones, profound muscle weakness, seizures, impaired renal function, and respiratory failure. There are no approved disease-modifying treatments for patients with this disease. There is also limited data available on the natural course of this disease over time, particularly in patients with the juvenile-onset form.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
6
The initial IV dose to be administered to patients was set at one-tenth the no adverse effect level (NOAEL) as determined by one month toxicology studies in animals in which Asfotase Alfa was administered as a single weekly IV dose. The SC doses to be administered are lower than the IV doses and are thought to be near or at the anticipated daily efficacious dose. Dosing will be as follows: Cohort 1: In Week 1, patients will receive an IV infusion of Asfotase Alfa at a dose of 3 mg/kg. In Weeks 2, 3 and 4, patients will receive weekly SC injections of Asfotase Alfa at a dose of 1 mg/kg SC.
Cohort 2: In Week 1, patients will receive an IV infusion of Asfotase Alfa at a dose of 7 mg/kg. In Weeks 2, 3 and 4, patients will receive weekly SC injections of Asfotase Alfa at a dose of 1.5 mg/kg SC.
Barnes Jewish Hospital- Washington University School of Medicine
St Louis, Missouri, United States
Duke University Medical Center
Durham, North Carolina, United States
Department of Pediatrics & Child Health, Health Sciences Centre Winnipeg, University of Manitoba
Winnipeg, Manitoba, Canada
To determine the safety and tolerability of Asfotase Alfa given intravenously and given subcutaneously.
Time frame: Within the first 2 months (8 weeks).
To assess the pharmacokinetics (PK) of Asfotase Alfa given intravenously and subcutaneously
Time frame: Within the first 2 months (8 weeks)
To assess the bioavailability of the subcutaneous Asfotase Alfa
Time frame: Within the first 2 months (8 weeks)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.