Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP)

Study Identifier:
ENB-006-09
ClinicalTrials.gov Identifier:
NCT00952484
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 2009 - Jul 2010
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 5 - 12 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Written informed consent from parent or legal guardian prior to participation
  • Patients \> 5 and \< 12 years of age with open growth plates at time of enrollment
  • Tanner stage of 2 or less indicating pre-pubescence
  • Documented history of HPP, as evidenced by:
  • Presence of HPP-related rickets on skeletal radiographs of the wrist and knee
  • Serum alkaline phosphatase (ALP) below age-adjusted normal range
  • Plasma PLP at least twice the upper limit of normal
  • 25(OH) vitamin D level \> 20 ng/mL
  • Ability of patient and parent/guardian to comply with study requirements
Exclusion Criteria
  • Serum calcium or phosphorus below age-adjusted normal range
  • History of sensitivity to any study drug constituent
  • Medical condition, serious intercurrent illness, or other extenuating circumstance that, in the opinion of the Investigator, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities
  • Treatment with an investigational drug within 1 month before start of study drug
  • Current enrollment in any other study involving an investigational new drug, device, or treatment for HPP (e.g., bone marrow transplantation)
  • Current evidence of a treatable form of rickets
  • Prior treatment with bisphosphonates
  • Bone fracture or orthopedic surgery within the past 12 months that, in the opinion of the Investigator would interfere with the ability of study patient to comply with study protocol
  • Major congenital abnormality other than those associated with HPP
Sex
Female & Male
Age
5 - 12 Years

Study Details

Medical Condition
  • Unmapped
Study Drug
  • Biological: asfotase alfa
Date
Sep 2009 - Jul 2010
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 5 - 12 Years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Written informed consent from parent or legal guardian prior to participation
  • Patients \> 5 and \< 12 years of age with open growth plates at time of enrollment
  • Tanner stage of 2 or less indicating pre-pubescence
  • Documented history of HPP, as evidenced by:
  • Presence of HPP-related rickets on skeletal radiographs of the wrist and knee
  • Serum alkaline phosphatase (ALP) below age-adjusted normal range
  • Plasma PLP at least twice the upper limit of normal
  • 25(OH) vitamin D level \> 20 ng/mL
  • Ability of patient and parent/guardian to comply with study requirements
Exclusion Criteria
  • Serum calcium or phosphorus below age-adjusted normal range
  • History of sensitivity to any study drug constituent
  • Medical condition, serious intercurrent illness, or other extenuating circumstance that, in the opinion of the Investigator, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities
  • Treatment with an investigational drug within 1 month before start of study drug
  • Current enrollment in any other study involving an investigational new drug, device, or treatment for HPP (e.g., bone marrow transplantation)
  • Current evidence of a treatable form of rickets
  • Prior treatment with bisphosphonates
  • Bone fracture or orthopedic surgery within the past 12 months that, in the opinion of the Investigator would interfere with the ability of study patient to comply with study protocol
  • Major congenital abnormality other than those associated with HPP

Protocol Summary

This clinical trial studied the safety and efficacy of asfotase alfa in children with HPP compared to a historical control group.

Trial Locations

Location
Status
Location
Shriners Hospital for Children
St Louis, Missouri, United States, 63131
Status
N/A
Location
The University of Manitoba Health Services Centre
Winnipeg, Manitoba, Canada, R3A 1S1
Status
N/A