Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)

Study Identifier:
ENB-002-08
ClinicalTrials.gov Identifier:
NCT00744042
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete

Study Details

Medical Condition
  • Unmapped
Study Drug
  • Biological: asfotase alfa
Date
Sep 2008 - May 2010
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: N/A - 36 Months
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Legal guardian(s) must provide informed consent prior to any study procedures
  • 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
  • History of sensitivity to any of the constituents of the study drug
  • 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
Sex
Female & Male
Age
N/A - 36 Months

Study Details

Medical Condition
  • Unmapped
Study Drug
  • Biological: asfotase alfa
Date
Sep 2008 - May 2010
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: N/A - 36 Months years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Legal guardian(s) must provide informed consent prior to any study procedures
  • 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
  • History of sensitivity to any of the constituents of the study drug
  • 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

Protocol Summary

This clinical trial studies the safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP.

Trial Locations

Location
Status
Location
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Status
N/A
Location
Alfred I. duPont Hospital for Children
Wilmington, Delaware, United States, 19803
Status
N/A
Location
St. John's Hospital
Springfield, Missouri, United States, 65804
Status
N/A
Location
University of Nebraska Medical Center, Munroe-Meyer Institute
Omaha, Nebraska, United States, 68114
Status
N/A
Location
Vanderbilt Children's Hospital
Nashville, Tennessee, United States, 37232
Status
N/A
Location
St. Vincent Hospital
Green Bay, Wisconsin, United States, 54301
Status
N/A
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