Sponsor study identifier
observational
48
7 countries
12
Study Start
August 2012
Date type not reportedThis date has not been verified as actual or estimated.
Primary Completion
June 2013
Actual (reported)Study Completion
February 2014
Actual (reported)This study aims to characterize the natural history of patients with severe perinatal or infantile onset HPP.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
February 2014
Actual (reported)Click on a node to explore related trials.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
August 16, 2011
Actual (reported)August 17, 2011
Actual (reported)August 2012
Date type not reportedThis date has not been verified as actual or estimated.
June 2013
Actual (reported)February 2014
Actual (reported)July 21, 2014
Actual (reported)Survival
Overall survival is defined as the time from birth to time of death.
Retrospective data collected on or before the data of abstraction.
Invasive Ventilator-free Survival Time
Retrospective data collected on or before the date of abstraction.
Patients with a confirmed diagnosis of perinatal or infantile onset hypophosphatasia (HPP)
Patients with perinatal and/or infantile onset HPP. Thirty-six patients were invasively ventilated or died, and 12 patients were censored.
Cedars-Sinai Medical Center
Los Angeles, California, United States
Indiana University school of medicine
Indianapolis, Indiana, 46202, United States
Shriners Hospital for Children
St Louis, Missouri, United States
Oregon Health & Science University
Portland, Oregon, United States
Cook Children's Health Care System
Fort Worth, Texas, United States
Royal Children's Hospital
Parkville, Australia
University of Manitoba Health Sciences Centre
Winnipeg, MB R3A 1R9, Canada
Whyte MP, Rockman-Greenberg C, Ozono K, Riese R, Moseley S, Melian A, Thompson DD, Bishop N, Hofmann C. Asfotase Alfa Treatment Improves Survival for Perinatal and Infantile Hypophosphatasia. J Clin Endocrinol Metab. 2016 Jan;101(1):334-42. doi: 10.1210/jc.2015-3462. Epub 2015 Nov 3.
This was an observational study and, as such, serious and/or other \[non-serious\] adverse events were not collected/assessed.
48 participants (actual enrollment reported by the registry).
First Submitted
August 16, 2011
First Posted
August 17, 2011
Study Start
August 2012
Date type not reportedThis date has not been verified as actual or estimated.
Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)
Contact the study team to confirm eligibility.
Universitatsmedizin Mainz, Villa
Mainz, 55131, Germany
Universitätsklinikum Würzburg Kinderklinik, Pädiatrische Infektiologie und Immunologie
Würzburg, 97080, Germany
Hospital Infantil Universitario Nino Jesus Universidad autonoma de Madrid
Madrid, Spain
National Taiwan University Hospital
Taipei, 10041, Taiwan
Birmingham Childrens Hospital
Birmingham, United Kingdom
Primary Completion
June 2013
Actual (reported)Study Completion
February 2014
Actual (reported)Last Updated
April 1, 2019
Results First Posted
July 21, 2014
Record last verified: 2019-03
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Source: ClinicalTrials.gov.
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