Continuous Subcutaneous Hydrocortisone Infusion Treatment for CAH

连续皮下注射氢化可的松治疗CAH

• 批准号: 8736955
• 负责人: Deborah Merke
• 金额: $ 0.85万
• 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/8736955
• 关键词: Adrenal Cortex Diseases; Adrenal Glands; Adrenal gland hypofunction; Adult; Aldosterone; Androgens; Childhood; Circadian Rhythms; Comorbidity; Congenital adrenal hyperplasia; Data; 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; Recruitment Activity; Replacement Therapy; Safety; Steroid 21-Monooxygenase; Testing; Virilism; design; 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 newly approved pilot study has enrolled one patient and is actively recruiting. 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。这项新批准的初步研究已入组1例患者，并正在积极招募。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。