Modified-release Hydrocortisone Therapy as a Treatment for CAH

第二阶段，16名成人(8名女性)典型CAH患者的交叉药代动力学和药效学研究已经完成，将新开发的氢化可的松改良释放制剂与传统氢化可的松进行比较。这种新开发的氢化可的松配方，Chronocort，旨在模拟正常的皮质醇昼夜节律。研究发现，每天两次的时间片与生理性皮质醇的分泌大致相同。与基线时的常规治疗相比，6个月后的Chronocort显示全天的雄激素水平都有所改善，这种雄激素水平的降低是在每日平均糖皮质激素剂量较低或类似的情况下实现的。尽管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，脂肪质量(男性)减少。这种新的氢化可的松制剂代表了一种潜在的治疗CAH患者的新方法，目前正在进行一项国际多中心3期临床试验。第三阶段研究现已完成，并正在进行数据分析。

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.