ALENDRONATE DISODIUM IN PEDIATRIC GAUCHER DISEASE

点击翻译按钮获取中文摘要

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The purpose of this study is to determine whether osteopenia and growth retardation observed in most children with Gaucher disease can be corrected by the addition of Fosamax¿ to the existing enzyme replacement therapy. To do so, a double blind, two-arm controlled trial of alendronate will be conducted on children with Gaucher disease who have received at least 18 months of regular enzyme therapy with Cerezyme. This study will be conducted with an initial three week single blind trial period to evaluate compliance, after which a 1:1 drug:placebo randomization will occur. At the end of the 18 month double blind placebo phase, there will be a 12 month open label extension to evaluate longer-term safety and efficacy. Ninety patients between 6 and 18 years of age who are receiving enzyme therapy will be randomized to receive alendronate or placebo for 18 months. Therapeutic outcome will primarily be monitored by measurement of bone density at the lumbar spine and total body. Changes in weight and height will be assessed with measurements being taken at the time of entry into the study and at 6 month intervals for 18 months and again at 30 months. Skeletal maturation, sexual maturity and responses to a pediatric quality of life questionnaire will also be evaluated. The successful outcome of these studies may lead to new therapeutic regimens for Gaucher disease that control or reverse osteopenia in children, and may lead to more effective preventive interventions for the pediatric and adult Gaucher bone disease