JPRN-jRCT2080223018已完成1 期
Phase I/II Study of DS-5141b: Open-label study of DS-5141b in patients with Duchenne muscular dystrophy
DAIICHISANKYO Co.,Ltd.0 个研究点目标入组 8 人开始时间: 2015年11月18日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 8
研究概览
简要总结
The safety, tolerability, efficacy, and pharmacokinetics of DS-5141b in 8 subjects with DMD were evaluated. No safety concerns were found at either the DS-5141b 2.0 mg/kg or 6.0 mg/kg dose, indicating that the drug was well tolerated. Plasma DS-5141a concentrations generally increased dose-proportionally within the assessed DS-5141b dose range, with only minor cumulative Cmax and AUC168h at steady state. A certain level of efficacy was found in subjects with DMD.
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- >= 5age old 至 <= 10age old（—）
- 性别
- Male
入选标准
- •1)Confirmation of out-of-frame deletion(s) that could be corrected by dystrophin gene exon 45 skipping.
- •2)Intact muscles of adequate quality for biopsy to allow evaluation of the efficacy of the study drug.
- •3)Boys aged from 5 years to <11 years.
- •4)Patients able to walk at least 325 meters in the 6-minutes walk test.
- •5)Glucocorticoid-naive patients, or patients who have used glucocorticoids for at least 6 months prior to enrollment in this study with no dose changes for at least 3 months prior to enrollment.
排除标准
- •1)A genetic mutation that can not be expected the expression of dystrophin protein by dystrophin gene exon 45 skipping.
- •2)A concurrent illness other than DMD that can cause muscle weakness and/or impairment of motor function.
- •3)Current or history of severe disorder.
- •4)Left ventricular ejection fraction (LEVF) <55%.
- •5)Corrected QT interval (QTc) >0.45 sec.
研究者
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