CLINICAL TRIAL: ALTU- 135 TREATMENT IN PATIENTS WITH CF-RELATED EXOCRINE PANCREA

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 囊性纤维化(CF)是一种隐性遗传病，在美国约有30,000名儿童和成年人受到影响。尽管在治疗上取得了进展，但没有治愈的方法。预测的CF患者的存活年龄中值约为35岁左右。 本研究的研究对象为男性和女性，7岁或以上，有文献记载的外分泌胰腺功能不全(PI)。 该研究的主要目的是证明和证实ALTU-135治疗CF患者脂肪吸收不良的疗效。次要疗效目标包括对ALTU-135的蛋白酶和淀粉酶成分的疗效测量，以及在标记对标记的粪便收集期间测定大便重量和频率，作为ALTU-135治疗对吸收不良影响的临床测量。还将评估ALTU-135在研究期间的耐受性和安全性。 这是一项三期随机、双盲、安慰剂对照的临床研究。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Cystic fibrosis (CF) is a recessive genetic disease that affects approximately 30,000 children and adults in the United States. Despite progress in the treatment of CF, there is no cure. The predicted median approximate age of survival for a person with CF is the mid-30s. The study subject population of this study is Male and female with CF, 7 years of age or older, with documented exocrine Pancreatic Insufficieny (PI). The primary study objective is to demonstrate and confirm the efficacy of ALTU-135 for the treatment of fat malabsorption in patients with CF. The secondary efficacy objectives include efficacy measures of the protease and amylase components of ALTU-135, as well as determination of stool weight and frequency over the marker-to-marker stool collection periods as a clinical measure of the effect of ALTU-135 treatment on malabsorption. The tolerability and safety of ALTU-135 over the duration of the study will also be assessed. This is a Phase III, Randomized, Double-Blind, Placebo-Controlled Clinical Study.