Status:
COMPLETED
Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP)
Lead Sponsor:
Alexion Pharmaceuticals, Inc.
Conditions:
Hypophosphatasia (HPP)
Eligibility:
All Genders
5-12 years
Phase:
PHASE2
Brief Summary
This clinical trial studied the safety and efficacy of asfotase alfa in children with HPP compared to a historical control group.
Detailed Description
Asfotase Alfa was formerly referred to as ENB-0040 Hypophosphatasia (HPP) is a life-threatening, genetic, and ultra-rare metabolic disease characterized by defective bone mineralization and impaired ...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
September 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 1 2010
Estimated Enrollment :
13 Patients enrolled
Trial Details
Trial ID
NCT00952484
Start Date
September 1 2009
End Date
July 1 2010
Last Update
April 1 2019
Active Locations (2)
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1
Shriners Hospital for Children
St Louis, Missouri, United States, 63131
2
The University of Manitoba Health Services Centre
Winnipeg, Manitoba, Canada, R3A 1S1