ORAL THERACLEC-TOTAL IN CYSTIC FIBROSIS SUBJECTS

该子项目是利用NIH/NCRR资助的中心赠款提供的资源的许多研究子项目之一。子项目和研究者（PI）可能从另一个NIH来源获得主要资金，因此可以在其他CRISP条目中表示。所列机构为中心，不一定是研究者所在机构。这是一项在胰腺外分泌功能不全的囊性纤维化（CF）受试者中评价口服TheraCLEC-Total的多中心、II期、随机、双盲、平行剂量范围研究。本研究计划在三个剂量水平的TheraCLEC-Total中共入组约126例受试者（每组约42例受试者）。 这项研究将帮助我们评估CF患者中TheraCLEC-Total的剂量，并了解哪种剂量对治疗部分或完全外分泌胰腺功能不全引起的吸收不良效果最好。 研究分为四个不同的观察和评估阶段：筛选、基线、治疗和随访。缓解期包括门诊护理和住院护理。 主要终点是脂肪吸收系数（CFA）。次要终点是氮吸收系数（CNA）、粪便重量和数量、淀粉消化和碳水化合物吸收（测量血糖反应）以及生活质量（通过囊性纤维化问卷（CFQ）测量）。 在不良事件特征、生命体征、异常实验室检查（血液学、化学、凝血、尿分析和尿尿酸排泄）频率、粪便血红素和白色血细胞测定中记录安全性终点。 TheraCLEC-Total给药后血清中脂肪酶、蛋白酶和淀粉酶的浓度为药代动力学终点。

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. This is a multicenter, Phase II, randomized, double-blind, parallel-dose ranging study of oral TheraCLEC-Total in cystic fibrosis (CF) subjects with exocrine pancreatic insufficiency. The study plans to enroll a total of approximately 126 subjects at three dose levels of TheraCLEC-Total (approximately 42 subjects per arm). This study will help us to evaluate the doses of TheraCLEC-Total in CF patients and see which dose works the best for the treatment of malabsorption due to partial or complete exocrine pancreatic insufficiency. The study is separated into four distinct periods of observation and assessment: Screening, Baseline, Treatment, and Follow-Up. The Treatement Period includes a combination of both outpatient care and inpatient care. The primary endpoint is the coefficient of fat absorption (CFA). Secondary endpoints are the coefficient of nitrogen absorption (CNA), weight and number of stools, starch digestion, and carbohydrate absorption as measured blood glucose response and the quality of life as measured by a Cystic Fibrosis Questionnaire (CFQ). The safety endpoints are noted in adverse events profiles, vital signs, frequency of abnormal laboratory tests (hematology, chemistry, coagulation, urinalysis, and urinary uric acid excretion), stool heme and white blood cell assays. Concentrations of lipase, protease and amylase in serum after administration of TheraCLEC-Total are the pharmacokinetic endpoints.