PHASE II STUDY OF MULTIPLE DOSES OF DMP-777 IN ADULTS WITH CYSTIC FIBROSIS

本研究的目的是评价安全性， 8-环戊基-1，3-二丙基黄嘌呤在成人体内的药代动力学 轻度囊性纤维化患者。CPX在体外起作用以纠正 囊性纤维化最常见突变的细胞代谢， 即Df 508，以允许适当的易位和膜功能。 这是一项随机、双盲、安慰剂对照的剂量递增研究 CF成人口服CPX的研究。除了安全和 药代动力学数据，我们还将测量生物替代标记物 功效，即对汗液氯化物水平和鼻腔的影响 跨上皮电位差测量。

The objectives of the study are to evaluate the safety and pharmacokinetics of 8-cyclopentyl-1, 3-dipropylxanthine (CPX) in adult patients with mild cystic fibrosis. CPX acts in vitro to correct the cellular metabolism of the most common mutation of cystic fibrosis, namely Df508, to allow proper translocation and membrane function. This is a randomized, double-blind, placebo controlled ascending dose study of orally adminstered CPX in CF adults. Besides safety and pharmacokinetic data, we will also measure biological surrogate markers of efficacy, namely effect on sweat chloride levels and nasal transepithelial potential difference measurements.