GROWTH HORMONE REPLACEMENT THERAPY IN CHILDHOOD ONSET GROWTH HORMONE DEF

儿童期生长激素缺乏症的生长激素替代疗法

• 批准号: 6441958
• 负责人: CATHERINE M. GORDON
• 金额: $ 2.88万
• 依托单位: BOSTON CHILDREN'S HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2000
• 资助国家: 美国
• 起止时间: 2000-12-01 至 2001-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6441958
• 关键词: adolescence (12-20); body height; body weight; bone density; child (0-11); clinical research; clinical trial phase I; disease /disorder onset; dosage; epiphysis; hormone therapy; human subject; human therapy evaluation; longitudinal human study; normal ossification; osteogenesis; pituitary dwarfism; somatotropin; young adult human (21-34)

International multi-center randomized controlled trial to examine the effect of growth hormone replacement (hGH) in adolescent and young adult patients with childhood onset growth hormone deficient (GHD)previously treated to final height. Previous data obtained from adult GHD patients of childhood onset treated with hGH until epiphyseal closure indicate that in spite of satisfactory final height results, other developmental milestone may not have been achieved in these individuals. Evidence suggests that the attainment of adult body shape and of peak bone mass are usually not reached until after epiphyseal closure, i.e. after final height has been reached. Thus, patients only treated to final height often have a lower than normal lean body mass, and a lower than normal bone mineral content (BMC) and bone mineral density (BMD). This clinical study will test the hypothesis that continuing hGH replacement therapy for two years after epiphyseal closure will result in increased lean body mass, BMC, and BMD. The accepted daily replacement dosage for adults (0.017-0.034IU/kg/day) will also be evaluated to see if it is significant enough to cover the developmental phase following epiphyseal closure in young adults, or if a larger dosage closer to that used in children (0.070-0.100 IU/kg/day) is more appropriate. Three treatment arms will be included; pediatric dosage, adult dosage, and no treatment.

一项国际多中心随机对照试验，旨在检验生长激素替代疗法(HGH)对儿童发作性生长激素缺乏症(GHD)的青春期和青壮年患者的影响，这些患者曾接受过最终身高治疗。从儿童时期起病的成人GHD患者中获得的数据表明，尽管最终身高结果令人满意，但这些人可能没有达到其他发育里程碑。有证据表明，成人的体型和骨量峰值通常是在骨膜闭合后，即达到最终高度后才能达到的。因此，仅治疗到最终身高的患者通常瘦体重低于正常，骨矿含量(BMC)和骨密度(BMD)低于正常。这项临床研究将验证这一假设，即在骨膜关闭后继续进行hGH替代治疗两年将导致瘦体重、BMC和BMD增加。成人每日可接受的替代剂量(0.017-0.034IU/kg/天)也将进行评估，以确定该剂量是否足以覆盖年轻成人骨膜闭合后的发育阶段，或者更大的剂量是否更接近儿童使用的剂量(0.070-0.100 IU/kg/天)。将包括三个治疗臂：儿童剂量、成人剂量和不治疗。