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.

这项研究是一项随机开放标签的多中心（跨国）两年研究，评估儿童发作生长激素缺乏（GHD）患者的体重组成和骨密度，此后用生长激素治疗成人两年后。假设是成年人需要生长激素来维持正常的身体组成。这项研究的目的是评估与患有GHD治疗的年轻人相比，epiphyseal闭合后两年后接受生长激素治疗的患者是否有任何显着差异。 此外，与那些接受标准成人剂量治疗的儿童相比，这项研究将评估继续以标准小儿剂量的儿童的身体成分差异。 接受GH治疗的儿童直到达到最终高度的儿童将接受成人剂量（每天0.3 mg RHGH）和2年的儿科剂量（0.4mg/kg/day），并且在治疗前和治疗前通过DEXA评估了体重组成和骨密度。 目前，有2名患者参加了研究。 招募研究并开始RHGH的第三名患者出现了星形胶质细胞瘤的复发。 这与药物无关，因为较早的大脑MRI表现出可能复发。