Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP)
Completed · Phase 2
Conditions studied: Hypophosphatasia (HPP)
In brief
This clinical trial studied the safety and efficacy of asfotase alfa in children with HPP compared to a historical control group.
Key facts
- Study ID
- NCT00952484
- Run by
- Alexion Pharmaceuticals, Inc.
- People needed
- 13
- Starts
- 2009-09-01
- Expected to finish
- 2010-07-01
- Last updated by the study team
- 2019-04-01
Who can join
Age: 5 and older, up to 12. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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
Where it is running
- Shriners Hospital for Children — St Louis, Missouri, United States
- The University of Manitoba Health Services Centre — Winnipeg, Manitoba, Canada
Full record on ClinicalTrials.gov
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