Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)
NCT ID: NCT00744042
Last Updated: 2019-04-01
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
PHASE1/PHASE2
11 participants
INTERVENTIONAL
2008-09-30
2010-05-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)
NCT01205152
Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)
NCT02797821
Evaluate and Monitor Physical Performance of Adults Treated With Asfotase Alfa for Hypophosphatasia
NCT03418389
Retrospective, Non-interventional Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)
NCT02104219
Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
NCT02193867
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
asfotase alfa
asfotase alfa
asfotase alfa
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
asfotase alfa
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Documented diagnosis of severe HPP as indicated by:
* Total serum alkaline phosphatase at least 3 standard deviations (SD) below the mean for age
* Plasma pyridoxal 5'-phosphate (PLP) at least 4 times the upper limit of normal
* Radiographic evidence of HPP (hypophosphatasia), characterized by:
* Flared and frayed metaphyses
* Severe, generalized osteopenia
* Widened growth plates
* One or more HPP-related findings:
* History or presence of:
* Non-traumatic post-natal fracture
* Delayed fracture healing
* History of elevated serum calcium
* Functional craniosynostosis with decreased head circumference growth
* Nephrocalcinosis
* Respiratory compromise
* Rachitic chest deformity and/or vitamin B6 dependent seizures
* Failure to thrive
* Onset of symptoms prior to 6 months of age
* Age ≤ 36 months
* Otherwise medically stable (patient may be on ventilatory support)
* Legal guardian(s) must be willing to comply with the study
Exclusion Criteria
* Current or prior clinically significant cardiovascular, endocrinologic, hematologic, hepatic, immunologic, metabolic, infectious, urologic, pulmonary, neurologic, dermatologic, renal condition and/or other major disease which, in the opinion of the investigator, precludes study participation
* Treatment with an investigational drug within 1 month prior to the start of study drug administration
* Current enrollment in any other study involving an investigational new drug, device or treatment for HPP (e.g., bone marrow transplantation)
* Low serum calcium, phosphate or 25(OH) vitamin D
* Current evidence of a treatable form of rickets
* Prior treatment with bisphosphonate
36 Months
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Alexion Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Arkansas Children's Hospital
Little Rock, Arkansas, United States
Alfred I. duPont Hospital for Children
Wilmington, Delaware, United States
St. John's Hospital
Springfield, Missouri, United States
University of Nebraska Medical Center, Munroe-Meyer Institute
Omaha, Nebraska, United States
Vanderbilt Children's Hospital
Nashville, Tennessee, United States
St. Vincent Hospital
Green Bay, Wisconsin, United States
The University of Manitoba Health Sciences Centre
Winnipeg, Manitoba, Canada
Tawam-John Hopkins Hospital
Al Ain City, Abu Dhabi Emirate, United Arab Emirates
Sheffield Children's Hospital
Sheffield, England, United Kingdom
Royal Belfast Hospital for Sick Children
Belfast, Northern Ireland, United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
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.
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, 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.
Whyte MP, Greenberg CR, Salman NJ, Bober MB, McAlister WH, Wenkert D, Van Sickle BJ, Simmons JH, Edgar TS, Bauer ML, Hamdan MA, Bishop N, Lutz RE, McGinn M, Craig S, Moore JN, Taylor JW, Cleveland RH, Cranley WR, Lim R, Thacher TD, Mayhew JE, Downs M, Millan JL, Skrinar AM, Crine P, Landy H. Enzyme-replacement therapy in life-threatening hypophosphatasia. N Engl J Med. 2012 Mar 8;366(10):904-13. doi: 10.1056/NEJMoa1106173.
Related Links
Access external resources that provide additional context or updates about the study.
Hypophosphatasia Website
Hypophosphatasia Website for Healthcare Providers
HPP support group
US Hypophosphatasia Group (Soft Bones)
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
ENB-002-08
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.