A Multicenter, Open-Label, Dose Escalating Study of the Safety, Tolerability and Pharmacology of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Asfotase Alfa in Adults With Hypophosphatasia (HPP)
Sponsor study identifier
interventional
6
2 countries
3
Study Start
August 2008
Date type not reportedThis date has not been verified as actual or estimated.
Primary Completion
January 2009
Actual (reported)Study Completion
February 2009
Actual (reported)This clinical trial studies the safety, tolerability, and pharmacology of asfotase alfa when given to adults with HPP.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
February 2009
Actual (reported)Click on a node to explore related trials.
Some registry-reported names shown below are not yet linked in this graph.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
August 2008
Date type not reportedThis date has not been verified as actual or estimated.
August 19, 2008
Actual (reported)August 21, 2008
Actual (reported)January 2009
Actual (reported)February 2009
Actual (reported)To determine the safety and tolerability of Asfotase Alfa given intravenously and given subcutaneously.
Within the first 2 months (8 weeks).
To assess the pharmacokinetics (PK) of Asfotase Alfa given intravenously and subcutaneously
Within the first 2 months (8 weeks)
To assess the bioavailability of the subcutaneous Asfotase Alfa
Within the first 2 months (8 weeks)
3 HPP patients are to be enrolled in Cohort 1 and receive a single IV dose and three weekly SC doses of Asfotase Alfa . End of Study for patients in Cohort 1 is at 8 weeks.
Cohort 2 will begin when the safety and PK data for Cohort 1 weeks 1-4 has been reviewed by the DSMB. Cohort 2 will enroll 3 HPP patients and will receive a higher dose level than Cohort 1. Cohort 2 patients will have a single IV dose and three weekly SC doses of Asfotase Alfa . End of Study for patients in Cohort 2 is at 8 weeks.
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.
Barnes Jewish Hospital- Washington University School of Medicine
St Louis, Missouri, 63110, United States
Duke University Medical Center
Durham, North Carolina, 27710, United States
Department of Pediatrics & Child Health, Health Sciences Centre Winnipeg, University of Manitoba
Winnipeg, Manitoba, R3A 1S1, Canada
Millan JL, Narisawa S, Lemire I, Loisel TP, Boileau G, Leonard P, Gramatikova S, Terkeltaub R, Camacho NP, McKee MD, Crine P, Whyte MP. Enzyme replacement therapy for murine hypophosphatasia. J Bone Miner Res. 2008 Jun;23(6):777-87. doi: 10.1359/jbmr.071213.
Drake MT, Khosla S. Bone-targeted replacement therapy for hypophosphatasia. J Bone Miner Res. 2008 Jun;23(6):775-6. doi: 10.1359/jbmr.080305. No abstract available.
6 participants (actual enrollment reported by the registry).
First Submitted
August 19, 2008
First Posted
August 21, 2008
Study Start
August 2008
Date type not reportedThis date has not been verified as actual or estimated.
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.
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
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Primary Completion
January 2009
Actual (reported)Study Completion
February 2009
Actual (reported)Last Updated
March 29, 2019
Record last verified: 2019-03
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Source: ClinicalTrials.gov.
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