Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)
NCT ID: NCT01205152
Last Updated: 2019-03-13
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
10 participants
INTERVENTIONAL
2009-04-30
2016-08-31
Brief Summary
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Partial funding for this study was provided by the Office of Orphan Product Development (OOPD).
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Detailed Description
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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 limited data available on the natural course of this disease over time, particularly in patients with the juvenile-onset form.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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asfotase alfa
An initial single intravenous (IV) infusion of 2 mg/kg asfotase alfa, followed by subcutaneous (SC) injections of 1 mg/kg asfotase alfa 3 times per week
asfotase alfa
Interventions
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asfotase alfa
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Written informed consent by parent or other legal guardian prior to any study procedures being performed
* Parent or other legal guardian willing to comply with study requirements
Exclusion Criteria
* Clinically significant disease that precludes study participation
* Enrollment in any study (other than ENB-002-08) involving an investigational drug, device, or treatment for HPP (e.g., bone marrow transplantation)
24 Weeks
42 Months
ALL
No
Sponsors
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Alexion Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Alfred I. DuPont Hospital for Children
Wilmington, Delaware, United States
St. John's Medical Research Institute
Springfield, Missouri, United States
University of Nebraska Medical Center
Omaha, Nebraska, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
St. Vincent Hospital
Green Bay, Wisconsin, United States
Tawam Hospital
Al Ain City, Abu Dhabi Emirate, United Arab Emirates
Royal Maternity Hospital, Royal Belfast Hospital for Sick Children
Belfast, Northern Ireland, United Kingdom
Sheffield Children's Hospital
Sheffield, , United Kingdom
Countries
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References
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Padidela R, Yates R, Benscoter D, McPhail G, Chan E, Nichani J, Mughal MZ, Myer C 4th, Narayan O, Nissenbaum C, Wilkinson S, Zhou S, Saal HM. Characterization of tracheobronchomalacia in infants with hypophosphatasia. Orphanet J Rare Dis. 2020 Aug 6;15(1):204. doi: 10.1186/s13023-020-01483-9.
Whyte MP, Simmons JH, Moseley S, Fujita KP, Bishop N, Salman NJ, Taylor J, Phillips D, McGinn M, McAlister WH. Asfotase alfa for infants and young children with hypophosphatasia: 7 year outcomes of a single-arm, open-label, phase 2 extension trial. Lancet Diabetes Endocrinol. 2019 Feb;7(2):93-105. doi: 10.1016/S2213-8587(18)30307-3. Epub 2018 Dec 14.
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.
Related Links
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Hypophosphatasia Website
Hypophosphatasia Website for Healthcare Providers
HPP support group
US Hypophosphatasia Group (Soft Bones)
Other Identifiers
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FD-R-003745-03
Identifier Type: OTHER_GRANT
Identifier Source: secondary_id
ENB-003-08
Identifier Type: -
Identifier Source: org_study_id
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