VX-809能增加突变型CFTR(F508del-CFTR)的成熟,并纠正常见于囊性纤维化的CFTR突变,EC50为0.1 μM。
VX-809 (Lumacaftor) acts to correct CFTR mutations common in cystic fibrosis by increasing mutant CFTR (F508del-CFTR) maturation,EC50 of 0.1 μM in fisher rat thyroid cells. Phase 3.
~0.1 mM
动物 A (mg/kg) = 动物 B (mg/kg)×动物 B的Km系数/动物 A的Km系数 | |
例如,已知某工具药用于小鼠的剂量为88 mg/kg , 则用于大鼠的剂量换算方法:将88 mg/kg 乘以小鼠的Km系数(3),再除以大鼠的Km系数(6),得到该药物用于大鼠的等效剂量44 mg/kg。
[1] Van Goor F, et al. Proc Natl Acad Sci U S A, 2011, 108(46), 18843-18848.
分子式 C24H18F2N2O5 |
分子量 452.41 |
CAS号 936727-05-8 |
储存方式 ﹣20 ℃冷藏长期储存。冰袋运输 |
溶剂(常温) |
DMSO 90 mg/mL |
Water <1 mg/mL |
Ethanol 5 mg/mL |
体内溶解度
NCT Number | Conditions | Interventions | Sponsor/Collaborators | Phases | Start Date | Last Updated |
NCT00865904 | Cystic Fibrosis | Drug: VX-809|Drug: Placebo | Vertex Pharmaceuticals Incorporated | Phase 2 | 2009-03-01 | 2015-08-01 |
NCT01216046 | Cystic Fibrosis | Drug: VX-809|Drug: VX-770|Drug: VX-809 placebo|Drug: VX-770 placebo | Vertex Pharmaceuticals Incorporated | Phase 1 | 2010-10-01 | 2012-01-16 |
NCT00966602 | Cystic Fibrosis | Drug: VX-809|Drug: VX-770|Drug: VX-809 & VX-770|Drug: Placebo | Vertex Pharmaceuticals Incorporated | Phase 1 | 2009-09-01 | 2010-01-02 |
NCT01225211 | Cystic Fibrosis | Drug: Lumacaftor|Drug: Ivacaftor|Drug: Lumacaftor Placebo|Drug: Ivacaftor Placebo | Vertex Pharmaceuticals Incorporated | Phase 2 | 2010-10-01 | 2015-09-02 |
NCT01899105 | Cystic Fibrosis | Drug: lumacaftor|Drug: ivacaftor | Vertex Pharmaceuticals Incorporated | Phase 1 | 2013-07-01 | 2014-04-02 |
NCT01897233 | Cystic Fibrosis | Drug: Lumacaftor|Drug: Ivacaftor | Vertex Pharmaceuticals Incorporated | Phase 3 | 2013-07-01 | 2016-10-07 |
NCT02807415 | Cystic Fibrosis | Drug: Lumacaftor plus Ivacaftor | Hannover Medical School|Heidelberg University|University of Giessen | 2016-06-01 | 2016-06-22 | |
NCT01768663 | Cystic Fibrosis | Drug: Lumacaftor|Drug: Ivacaftor|Drug: Ciprofloxacin|Drug: Itraconazole|Drug: Rifampin | Vertex Pharmaceuticals Incorporated | Phase 1 | 2013-01-01 | 2013-11-14 |
NCT02390219 | Cystic Fibrosis|Advanced Lung Disease | Drug: Lumacaftor|Drug: Ivacaftor | Vertex Pharmaceuticals Incorporated | Phase 3 | 2015-03-01 | 2016-10-26 |
NCT01931839 | Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation | Drug: Lumacaftor|Drug: Ivacaftor | Vertex Pharmaceuticals Incorporated | Phase 3 | 2013-10-01 | 2016-04-27 |
NCT02514473 | Cystic Fibrosis | Drug: VX-809|Drug: Placebo|Drug: VX-770 | Vertex Pharmaceuticals Incorporated | Phase 3 | 2015-07-01 | 2016-10-24 |
NCT01807923 | Cystic Fibrosis, Homozygous for the F508del CFTR Mutation | Drug: Lumacaftor Plus Ivacaftor Combination|Drug: Ivacaftor|Drug: Placebo | Vertex Pharmaceuticals Incorporated | Phase 3 | 2013-05-01 | 2015-08-01 |
NCT01807949 | Cystic Fibrosis, Homozygous for the F508del CFTR Mutation | Drug: Placebo|Drug: Lumacaftor Plus Ivacaftor Combination|Drug: Ivacaftor | Vertex Pharmaceuticals Incorporated | Phase 3 | 2013-04-01 | 2016-08-15 |
NCT01910415 | Cystic Fibrosis | Drug: Lumacaftor|Drug: Lumacaftor Placebo|Drug: Ivacaftor|Drug: Ivacaftor Placebo|Drug: moxifloxacin hydrochloride | Vertex Pharmaceuticals Incorporated | Phase 1 | 2013-06-01 | 2014-08-08 |
NCT02653027 | Diabetes|Cystic Fibrosis | Drug: Lumacaftor-ivacaftor|Other: OGTT | Massachusetts General Hospital | 2016-01-01 | 2016-01-08 | |
NCT02858843 | Cystic Fibrosis|Diabetes | Drug: lumacaftor-ivacaftor | Massachusetts General Hospital | 2016-08-01 | 2016-08-15 | |
NCT02544451 | Cystic Fibrosis | Drug: Ivacaftor|Drug: Lumacaftor | Vertex Pharmaceuticals Incorporated | Phase 3 | 2015-08-01 | 2016-12-19 |
NCT02797132 | Cystic Fibrosis | Drug: lumacaftor/ ivacaftor | Vertex Pharmaceuticals Incorporated | Phase 3 | 2016-05-01 | 2016-07-22 |
NCT01888393 | Hepatic Impairment|Healthy | Drug: lumacaftor 200 mg q12h + ivacaftor 250 mg q12h | Vertex Pharmaceuticals Incorporated | Phase 1 | 2013-06-01 | 2013-11-18 |
NCT02823470 | Cystic Fibrosis | Drug: LUM/IVA|Device: activated smart device|Device: de-activated smart device | Vertex Pharmaceuticals Incorporated | Phase 4 | 2016-06-01 | 2017-03-22 |
注:以上所有数据均来自公开文献,并不保证对所有实验均有效,数据仅供参考。
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