Study of a corticotropin releasing factor-1 receptor antagonist for the treatment of congenital adrenal hyperplasia

促肾上腺皮质激素释放因子1受体拮抗剂治疗先天性肾上腺皮质增生症的研究

• 批准号: 10266561
• 负责人: Deborah Merke
• 金额: $ 0.2万
• 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/10266561
• 关键词: Adrenal Glands; Adrenal hormone preparation; Affinity; Androgens; CRF receptor type 1; Clinical Research; Congenital adrenal hyperplasia; Corticotropin; Corticotropin-Releasing Hormone; Dose; Glucocorticoids; Hormone Antagonists; Hypothalamic structure; Investigation; Multi-Institutional Clinical Trial; Phase; Pituitary Gland; healthy volunteer; novel therapeutics; receptor; safety study; small molecule

Excess glucocorticoid therapy is often needed to control the overproduction of adrenal androgens in congenital adrenal hyperplasia. A phase 2 multicenter clinical trial is underway which is exploring dose finding as well as efficacy of a high-affinity and selective small-molecule antagonist of CRH type 1 receptors in the pituitary gland. The mechanism of action has been validated in a previous proof-of-concept study and has been shown to be safe in Phase 1 clinical studies in healthy volunteers. This study represents an early stage of the investigation of a novel therapy in the treatment of congenital adrenal hyperplasia.

在先天性肾上腺皮质增生症中，通常需要过量糖皮质激素治疗来控制肾上腺雄激素的过度产生。一项2期多中心临床试验正在进行中，该试验正在探索垂体中CRH 1型受体的高亲和力和选择性小分子拮抗剂的剂量发现以及疗效。作用机制已在先前的概念验证研究中得到验证，并已在健康志愿者的I期临床研究中证明是安全的。这项研究代表了一种治疗先天性肾上腺皮质增生症的新疗法的早期研究阶段。