Safety and Efficacy Study of ENB-0040 in Juvenile Patients With Hypophosphatasia (HPP)
Study Identifier:
ENB-004-09
ClinicalTrials.gov Identifier:
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Other
Study Details
Medical Condition
- Unmapped
Study Drug
- Biological: ENB-0040
Date
Jul 2009 - Dec 2014
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
- 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 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 (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
Sex
Female & Male
Age
5 - 12 Years
Study Details
Medical Condition
- Unmapped
Study Drug
- Biological: ENB-0040
Date
Jul 2009 - Dec 2014
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
- 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 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 (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
Protocol 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. The purpose of this study is to test the safety of the study drug called ENB-0040 and see what effects is has on human juveniles and HPP.
Trial Locations
No locations found.