Phase 2 Multi-center Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia

这是一项含有安慰剂冲洗剂成分的奈瓦米贝(ATR-101)的多中心、单盲、多剂量研究。筛查期之后是安慰剂，基线导入期。然后，受试者接受14天的最低剂量奈瓦米贝治疗，然后是14天的单盲安慰剂洗脱期。安全性和有效性评估在治疗结束时和冲洗期之后进行。如果没有达到主要结果指标(17-OHP 2x ULN)，受试者将进入下一个更高的剂量水平。总共可能有5个ATR-101剂量水平。10名患者入选；9名患者完成了研究，1名患者因相关的严重不良事件而提前停药。两名受试者达到了初级Enpoin，另外5名受试者在治疗期间17-羟孕酮下降了27%至72%。最常见的副作用是胃肠道反应(30%)。不良事件没有剂量相关的趋势。 奈瓦米贝(ATR-101)在治疗2周内降低了17-羟孕酮水平。需要进行更大规模、持续时间更长的研究，以进一步评估其作为CAH附加疗法的疗效。

This was a multicenter, single-blind, multiple dose study of Nevanimibe (ATR- 101) with a placebo washout component. The Screening Period was followed by a placebo, baseline lead-in period. Subjects then received the lowest dose of Nevanimibe for 14 days followed by a single-blind placebo Washout Period of 14 days. Safety and efficacy assessments occurred at the end of treatment and following the washout period. If the primary outcome measure (17-OHP 2x ULN) was not achieved, the subject proceeded to the next higher dose level. A total of 5 ATR-101 dose levels were possible. Ten patients were enrolled; nine completed the study, and 1 discontinued early due to a related serious adverse event. Two subjects met the primary endpoin, and 5 others experienced 17-hydroxyprogesterone decreases ranging from 27% to 72% during treatment. the most common side effects were gastointestinal (30%). There were no dose-related trends in adverse events. Nevanimibe (ATR-101) decreased 17-hydroxyprogesterone levels within 2 weeks of treatment. Larger studies of longer duration are needed to further evaluate its efficacy as add-on therapy for CAH.