CLINICAL TRIAL: L2762G EFF OF NUTROPIN AQ FOR TRMT OF GROWTH RESTRICTION IN CHIL

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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. Nutropin AQ is a recombinant human growth hormone rhGH. The use of rhGH for the treatment of growth restriction has been demonstrated in other chronic diseases. Treatment with rhGH can result in significant improvement in growth and lean body mass. There are some small studies which have shown that rHGH may improve the forced vital capacity in pulmonary function. Children with Cystic Fibrosis characteristically have reduced linear growth, which is predictive of declines in pulmonary function. The restricted growth in Cystic Fibrosis is multi-factorial, but probably due to a combination of poor dietary intake and increased metabolic demands. Adequate nutritional intake has not improved growth in Cystic Fibrosis patients when compared to a normal population. The proposed study is a phase II, multicenter, randomized, controlled, open-label study of the safety and efficacy of using rhGH in growth restricted children with Cystic Fibrosis. The study is designed to determine the effect of Nutropin AQ on growth restriction, lean body mass and pulmonary function in children with Cystic Fibrosis. Safety parameters will be a measurement of blood glucose levels, pulmonary function testing, and any changes in serum chemistries, immunologic response to the rhGH, as well as reported or observed symptoms of subjects. Efficacy will be a measurement of height and weight, pulmonary function testing, and dual energy x-ray absorptiometry DEXA scans.