Modified-release Hydrocortisone Therapy as a Treatment for CAH

在16名患有典型CAH的成人（8名女性）中完成了一项II期、交叉药代动力学和药效学研究，比较了新开发的氢化可的松缓释制剂与传统氢化可的松。 这种新开发的氢化可的松制剂Chronocort旨在模拟正常的皮质醇昼夜节律。发现每日两次Chronocort接近生理皮质醇分泌。 与基线常规治疗相比，Chronocort在6个月时显示全天雄激素水平改善，并且这种雄激素降低是通过较低或相似的平均每日糖皮质激素剂量实现的。虽然体重指数没有显著变化，但对身体成分的研究表明，瘦体重（女性）增加，脂肪量（男性）减少。 这种新型氢化可的松制剂代表了CAH患者的潜在新治疗方法，目前正在国际多中心3期临床试验中进行研究。第三阶段研究现已完成，数据分析正在进行中。

A Phase 2, crossover pharmacokinetic and pharmacodynamic study in 16 adults (8 females) with classic CAH, comparing a newly-developed modifed-release formulation of hydrocortisone with conventional hydrocortisone, was completed. This newly developed formulation of hydrocortisone, Chronocort, was designed to mimic the normal cortisol circadian rhythm. Twice daily Chronocort was found to approximate physiologic cortisol secretion. Compared to conventional therapy at baseline, Chronocort at 6 months showed improved androgen levels throughout the day and this lowering of androgens was achieved with a lower or similar average daily glucocorticoid dose. Although there were no significant changes in body mass index, studies of body composition showed an increase in lean mass (females) and a decrease in fat mass (males). This novel hydrocortisone formulation represents a potential new treatment approach for patients with CAH and is currently being studied in an international multi-center phase 3 clinical trial. The phase 3 study is now completed and data analysis is underway.