Status:
WITHDRAWN
Safety and Efficacy Study of ENB-0040 in Juvenile Patients With Hypophosphatasia (HPP)
Lead Sponsor:
Alexion Pharmaceuticals, Inc.
Conditions:
Hypophosphatasia
Eligibility:
All Genders
5-12 years
Phase:
PHASE2
Brief Summary
This Clinical Trial is being conducted to study Hypophosphatasia (HPP), a bone disorder caused by gene mutations or changes. These gene mutations cause low levels of an enzyme needed to harden bone. T...
Detailed Description
Hypophosphatasia (HPP) is a rare inherited form of rickets and osteomalacia caused by inactivating mutations in the gene encoding the tissue-nonspecific isoenzyme of alkaline phosphatase (TNSALP). The...
Eligibility Criteria
Inclusion
- Written informed consent from parent or legal guardian prior to participation
- Boys \>/= 5 and \< 12 years of age and girls \>/= 5 and \< 10 years of age with open growth plates at time of enrollment
- Documented history of HPP, as evidenced by:
- Presence of HPP-related rickets on skeletal radiographs
- Serum alkaline phosphatase (ALP) below age-adjusted normal range
- Plasma PLP at least twice the upper limit of normal (\>/=220 nM)
- Ambulatory without the use of assistive devices
- 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 (eg, 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
- Major congenital abnormality other than those associated with HPP
Key Trial Info
Start Date :
July 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2014
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT00894075
Start Date
July 1 2009
End Date
December 1 2014
Last Update
August 4 2023
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