Safety and Efficacy Study of ENB-0040 in Juvenile Patients With Hypophosphatasia (HPP) (NCT00894075) | Clinical Trial Compass
WithdrawnPhase 2
Safety and Efficacy Study of ENB-0040 in Juvenile Patients With Hypophosphatasia (HPP)
Stopped: Withdrawn pending further review of clinical design.
0Started 2009-07
Plain-language 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.
Who can participate
Age range5 Years – 12 Years
SexALL
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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
✕
What they're measuring
1
Skeletal radiographs using a qualitative Clinical Global Impression of Change (CGI-C) scoring system