Continuous Subcutaneous Hydrocortisone Infusion Treatment for CAH

连续皮下注射氢化可的松治疗CAH

• 批准号: 9348257
• 负责人: Deborah Merke
• 金额: $ 0.23万
• 依托单位: EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH & HUMAN DEVELOPMENT
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/9348257
• 关键词: Adrenal Cortex Diseases; Adrenal Glands; Adrenal gland hypofunction; Adult; Aldosterone; Androgens; Childhood; Circadian Rhythms; Comorbidity; Congenital adrenal hyperplasia; Data; Data Analyses; Dexamethasone; Disease; Drug Delivery Systems; Enrollment; Failure; Future; Glucocorticoids; Growth; Hormone replacement therapy; Hydrocortisone; Infertility; Infusion procedures; Life; Metabolic syndrome; Obesity; Osteoporosis; Patients; Physiological; Pilot Projects; Prednisone; Pump; Replacement Therapy; Safety; Steroid 21-Monooxygenase; Testing; Virilism; design; follow-up; improved; novel strategies; subcutaneous

This study is limited to patients with classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency, but has the potential to significantly improve the lives of patients with other forms of adrenal insufficiency. Each of these diseases requires life-long treatment and this novel approach to drug delivery has the potential to significantly improve patients lives. This pilot study is fully enrolled and is in follow-up only status and data analysis is underway. In addition, this study will provide information on the safety and tolerability of using a pump to deliver hydrocortisone, and will generate data that will be used in the design of future pediatric studies.

这项研究仅限于因21-羟化酶缺乏而患有经典先天性肾上腺皮质增生症的患者，但有可能显著改善其他形式肾上腺功能不全患者的生活。 这些疾病中的每一种都需要终身治疗，这种新的药物输送方法有可能显着改善患者的生活。该初步研究已全部入组，处于仅随访状态，数据分析正在进行中。此外，本研究将提供使用泵输送氢化可的松的安全性和耐受性信息，并将生成用于未来儿科研究设计的数据。