1] Department of Gastroenterology, University Hospital Heidelberg, Heidelberg, Germany [2] Department of Gastroenterology, Viernheim Hospital, Viernheim, Germany.
Internistische Facharztpraxis, Luedenscheid, Germany.
Am J Gastroenterol. 2014 Jul;109(7):1041-51. doi: 10.1038/ajg.2014.104. Epub 2014 May 6.
Phosphatidylcholine is a key component of the mucosal barrier. Treatment with modified release phosphatidylcholine aims to improve the impaired barrier function. The primary objective is to evaluate the efficacy of LT-02, a newly designed modified release phosphatidylcholine formula, in a multicenter setting.
This is a double-blinded, randomized, placebo-controlled, superiority study conducted in 24 ambulatory referral centers in Germany, Lithuania, and Romania. A total of 156 patients with an inadequate response to mesalazine, a disease activity score (Simple Clinical Colitis Activity Index (SCCAI)) of ≥ 5, and bloody diarrhea underwent treatment with 0, 0.8, 1.6, or 3.2 g LT-02. The primary end point was defined a priori as changes in SCCAI from baseline to the end of treatment. The primary statistical model was a general linear least-squares model. The study was funded by the sponsor Lipid Therapeutics, Heidelberg, Germany, and registered at http://clinicaltrials.gov/show/NCT01011322.
Baseline characteristics and dropouts were well balanced between all groups. The primary analyses revealed an SCCAI drop of 33.3% in the placebo group (from 9.0 to 6.0 points) compared with 44.3% in the 0.8 g LT-02 (from 8.8 to 4.9, P>0.05) and 40.7% in the 1.6 g groups (from 8.6 to 5.1, P>0.05). The 3.2 g group improved 51.7% from 8.5 to 4.1 (P=0.030 in comparison with placebo). The remission rate was 15% (6/40) in the placebo group compared with 31.4% (11/35) in the highest LT-02 dose group (P=0.089). Mucosal healing was achieved in 32.5% of placebo patients compared with 47.4% of LT-02 patients (P=0.098); the rates for histologic remission were 20% compared with 40.5%, respectively (P=0.016). There were 17 (48.6%) treatment-emergent adverse events in the highest dose group (and 0 serious adverse events (SAEs)) compared with 22 (55%) in the placebo group (4 SAEs).
The primary end point analysis showed a statistically significant improvement in disease activity during LT-02 treatment in comparison with placebo. The drug was found to be very safe.
磷脂酰胆碱是黏膜屏障的重要组成部分。采用缓释磷脂酰胆碱治疗旨在改善受损的屏障功能。主要目的是评估新型设计的缓释磷脂酰胆碱配方 LT-02 在多中心环境中的疗效。
这是一项在德国、立陶宛和罗马尼亚的 24 家门诊转诊中心进行的双盲、随机、安慰剂对照、优效性研究。共有 156 名对美沙拉嗪反应不足、疾病活动评分（简单临床结肠炎活动指数 (SCCAI)）≥5 和血性腹泻的患者接受 0、0.8、1.6 或 3.2 g LT-02 治疗。主要终点定义为从基线到治疗结束时 SCCAI 的变化。主要统计模型为一般线性最小二乘模型。该研究由德国海德堡的脂质治疗学赞助商资助，并在 http://clinicaltrials.gov 上注册，登记号为 NCT01011322。
所有组之间的基线特征和脱落情况均均衡。主要分析显示，安慰剂组 SCCAI 下降 33.3%（从 9.0 降至 6.0 分），而 0.8 g LT-02 组下降 44.3%（从 8.8 降至 4.9，P>0.05），1.6 g 组下降 40.7%（从 8.6 降至 5.1，P>0.05）。3.2 g 组从 8.5 改善了 51.7%至 4.1（与安慰剂相比，P=0.030）。安慰剂组的缓解率为 15%（6/40），而最高 LT-02 剂量组为 31.4%（11/35）（P=0.089）。安慰剂组黏膜愈合率为 32.5%，LT-02 组为 47.4%（P=0.098）；组织学缓解率分别为 20%和 40.5%（P=0.016）。最高剂量组有 17 例（48.6%）治疗出现不良事件（且无严重不良事件（SAE）），安慰剂组有 22 例（55%）（4 例 SAE）。
主要终点分析显示，与安慰剂相比，LT-02 治疗期间疾病活动有统计学意义的改善。该药物被发现非常安全。
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