ORAL THERACLEC-TOTAL IN CYSTIC FIBROSIS SUBJECTS

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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.