GROWTH HORMONE REPLACEMENT THERAPY IN CHILDHOOD ONSET HORMONE DEFICIENCY

生长激素替代疗法治疗儿童期激素缺乏症

• 批准号: 6306321
• 负责人: THOMAS MOSHANG
• 金额: $ 0.06万
• 依托单位: CHILDREN'S HOSP OF PHILADELPHIA
• 依托单位国家: 美国
• 财政年份: 1999
• 资助国家: 美国
• 起止时间: 1999-12-01 至 2001-02-28
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6306321
• 关键词: bone density; clinical research; disease /disorder onset; endocrine disorder; hormone therapy; human subject; human therapy evaluation; longitudinal human study; photon absorptiometry; somatotropin

This study is a randomized open-label multi-center (multi-national) two year study evaluating body mass composition and bone density in patients with childhood onset growth hormone deficient (GHD) after being treated with growth hormone for two years after attaining adult height. The hypothesis is that growth hormone is needed in adults for maintaining normal body composition. The intent of this study is to evaluate whether patients treated with growth hormone for two years after epiphyseal closure show any significant difference in body mass composition as compared to young adults with GHD in whom GH treatment is discontinued. Further, this study will evaluate differences in body composition in children continuing on the standard pediatric doses as compared to those children treated with standard adult doses. Children treated with GH until attainment of final height will be treated with both adult doses (0.3 mg rhGH per day) and pediatric doses (0.4mg/kg/day) for 2 years and body mass composition and bone density assessed by DEXA before and after treatment. At this time 2 patients are enrolled in the study. A third patient recruited for the study and begun on rhGH developed a recurrence of an astrocytoma. This was not drug related because an earlier MRI of the brain demonstrated probable recurrence.

本研究是一项为期两年的随机开放标签多中心（多国）研究，评估儿童期生长激素缺乏症（GHD）患者在达到成人身高后接受生长激素治疗两年后的体重组成和骨密度。假设是成年人需要生长激素来维持正常的身体成分。本研究的目的是评估在骨骺闭合后接受生长激素治疗两年的患者与停止生长激素治疗的年轻GHD患者相比，其体重组成是否有显著差异。此外，本研究将评估继续使用标准儿科剂量的儿童与使用标准成人剂量的儿童在身体成分上的差异。接受生长激素治疗直至达到最终身高的儿童将接受成人剂量（每天0.3 mg rhGH）和儿科剂量（每天0.4mg/kg）治疗2年，并在治疗前后通过DEXA评估体重组成和骨密度。目前有2名患者参加了这项研究。研究招募的第三名患者开始接受rhGH治疗后出现星形细胞瘤复发。这与药物无关，因为早期的脑部MRI显示可能复发。