一项基于鼻电位差的米格列醇治疗囊性纤维化的随机安慰剂对照试验。
A randomized placebo-controlled trial of miglustat in cystic fibrosis based on nasal potential difference.
机构信息
Pediatric Pulmonology & Cystic Fibrosis Unit, Cliniques St Luc, Université catholique de Louvain, 10 Avenue Hippocrate, 1200 Brussels, Belgium.
出版信息
J Cyst Fibros. 2012 May;11(3):231-6. doi: 10.1016/j.jcf.2011.12.004. Epub 2012 Jan 27.
BACKGROUND
Preclinical data suggest that miglustat could restore the function of the cystic fibrosis transmembrane conductance regulator gene in cystic fibrosis cells.
METHODS
Single-center, randomized, double-blind, placebo-controlled, crossover Phase II study in 11 patients (mean±SD age, 26.3±7.7 years) homozygous for the F508del mutation received oral miglustat 200 mgt.i.d. or placebo for two 8-day cycles separated by a 14-day washout period. The primary endpoint was the change in total chloride secretion (TCS) assessed by nasal potential difference.
RESULTS
No statistically significant changes in TCS, sweat chloride values or FEV(1) were detected. Pharmacokinetic and safety were similar to those observed in patients with other diseases exposed to miglustat.
CONCLUSIONS
There was no evidence of a treatment effect on any nasal potential difference variable. Further studies with miglustat need to adequately address criteria for assessment of nasal potential difference.
背景
临床前数据表明，米格列醇可能恢复囊性纤维化跨膜电导调节基因在囊性纤维化细胞中的功能。
方法
在 11 例纯合 F508del 突变的患者（平均年龄±标准差，26.3±7.7 岁）中进行了一项单中心、随机、双盲、安慰剂对照、交叉的 II 期研究，这些患者接受了米格列醇 200mg tid 或安慰剂治疗，每个 8 天的周期，间隔 14 天的洗脱期。主要终点是通过鼻电位差评估的总氯离子分泌（TCS）的变化。
结果
TCS、汗液氯化物值或 FEV1 均未检测到统计学上的显著变化。药代动力学和安全性与在接触米格列醇的其他疾病患者中观察到的相似。
结论
没有证据表明对任何鼻电位差变量有治疗作用。需要进一步研究米格列醇，以充分解决鼻电位差评估的标准。
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