EUR-1009-M

EUR-1009-M

• 批准号: 7603247
• 负责人: John Paul Clancy
• 金额: $ 0.06万
• 依托单位: UNIVERSITY OF ALABAMA AT BIRMINGHAM
• 依托单位国家: 美国
• 财政年份: 2007
• 资助国家: 美国
• 起止时间: 2007-03-01 至 2008-02-29
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7603247
• 关键词: Aftercare; Age; Clinical; Clinical Research; Computer Retrieval of Information on Scientific Projects Database; Condition; Confidence Intervals; Dose; End Point; Enrollment; Exocrine pancreatic insufficiency; Fatty acid glycerol esters; Funding; Grant; Incidence; Institution; Judgment; Label; Lipase; Oral Administration; Pancreas; Parents; Patients; Physicians; Population Study; Range; Research; Research Design; Research Personnel; Resources; Safety; Screening procedure; Signs and Symptoms; Site; Source; Steatorrhea; Symptoms; Testing; United States; United States National Institutes of Health; base; capsule; children with cystic fibrosis; cystic fibrosis patients; day; design; treatment duration

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. Ewand S.p. A has designed an open-label, multiple-dose, single-treatment, multicenter, clinical study in younger patients with CF and Exocrine Pancreatic Insufficiency (EPI) (age younger than 7 years) to determine the efficacy and safety of Eurand PEP (Pancreatic Eszyme Product) microtabs in young CF patients with pancreatic insufficiency. The study population of subjects will be enrolled among approximately 10 sites in the United States. Patients will receive approximately 2,000 units/kg/meal (not to exceed 10,000 lipase units/kg/day). These doses are within the recommended range for CF patients with EPI. Although Eurand PEP Microtabs are available in microtablets containing 5,000 lipase units/capsule and minitablets containing 10,000, 15,000, or 20,000 lipase units/capsule, patients will receive Eurand PEP Microtabs only. This study design involves a 4-day screening period, a 7-day dose-stabilization period, and a 7-day treatment period. The primary efficacy endpoint is the percentage of "responders" that is, patients in whom sign and symptoms and steatorrhea are not present after administration of Eurand PEP Microtabs. The percentage and 95% confidence interval will be presented. The fecal fat content change from baseline at Days 11 and 18 will be tested using the Wilcoxon signed-rank test. The incidence of clinical symptoms of EPI following oral administration of Eurand PEP Microtabs will be summarized. The patient's condition after treatment with Eurand PEP Microtabs will be listed and summarized based on the physician's and parent's judgment. The results of this study will provide important and overdue information regarding both the safety and efficacy of this product in young children with CF and EPI.

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 Ewand S. p. A设计了一项开放标签、多次给药、单次治疗、多中心临床研究，在患有CF和外分泌胰腺功能不全（EPI）（年龄小于7岁）的年轻患者中进行，以确定Eurand PEP（胰腺酶产品）微片在患有胰腺功能不全的年轻CF患者中的疗效和安全性。受试者的研究人群将在美国约10家研究中心入组。患者将接受约2，000单位/kg/餐（不超过10，000脂肪酶单位/kg/天）。这些剂量在EPI CF患者的推荐范围内。虽然Eurand PEP Microtabs有含5，000脂肪酶单位/胶囊的微片和含10，000、15，000或20，000脂肪酶单位/胶囊的小片，但患者仅接受Eurand PEP Microtabs。本研究设计包括4天筛选期、7天 剂量稳定期和7天治疗期。 主要疗效终点是“应答者”的百分比，即在给予Eurand PEP Microtabs后不存在体征和症状以及脂肪代谢的患者。将列出百分比和95%置信区间。粪便脂肪含量较基线的变化， 第11天和第18天将使用Wilcoxon符号秩检验进行检验。将总结Eurand PEP Microtabs口服给药后EPI临床症状的发生率。根据医生和家长的判断，列出并总结患者接受Eurand PEP Microtabs治疗后的状况。本研究的结果将提供关于本产品在患有CF和EPI的幼儿中的安全性和有效性的重要和过期信息。