A Long-Term Prospective Observational Safety Study of the Incidence of and Risk Factors for Fibrosing Colonopathy in US Patients With Cystic Fibrosis Treated With Pancreatic Enzyme Replacement Therapy: A Harmonized Protocol Across Sponsors(The CF-FC Study)

AbbVie123 个研究点分布在 1 个国家目标入组 17 人开始时间: 2012年7月31日最近更新: 2021年3月22日

适应症

试验速览

阶段: 不适用
状态: 终止
发起方
入组人数: 17
试验地点: 123
主要终点: Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients receiving PERTZYE.

研究概览研究设计入排标准结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

This is a long-term study in cystic fibrosis patients who are participating in the Cystic Fibrosis Patient Registry to assess the occurrence and risk factors for a rare bowel disorder called fibrosing colonopathy (narrowing of the large intestine). Patients will be followed at their regular clinical care visits over a 10-year period and approached if they develop symptoms of fibrosing colonopathy for collection and use of further detailed information.

详细描述

This is a prospective, observational, population-based cohort study in US cystic fibrosis patients participating in the Cystic Fibrosis Patient Registry in order to assess the incidence of and risk factors for fibrosing colonopathy. Cystic fibrosis (CF) patients participating in the registry from participating sites, as well as new CF patients enrolled in the registry at these sites over a 2-year period, will serve as the base study population (estimated to include 24,500-25,000 cystic fibrosis patients between the first patient encounter documented in the registry from any participating site and the 31st of July 2014). Cystic fibrosis patients in the base study population will be followed at their regular clinical care visits and, any patients presenting over a 10-year period with signs and symptoms of suspected fibrosing colonopathy, based on a prospective definition, will be approached to obtain a study-specific informed consent for collection of additional data outside the standard registry data collection form in order to augment surveillance. Data routinely collected via the standard CF registry will be used to determine exposure to any specific pancreatic enzyme replacement therapy and to assess potential risk factors for the outcome of confirmed fibrosing colonopathy. An independent adjudication panel will be utilized to validate the diagnosis of fibrosing colonopathy based on a prospective case definition as well as decision rules.

研究设计

研究类型: Observational
观察模型: Cohort
时间视角: Prospective

入排标准

年龄范围: 0 Months 至 99 Years（Child, Adult, Older Adult）
性别: All
接受健康志愿者: 否

入选标准

•The inclusion criteria for enrollment in the Base Study Population
•Diagnosed with cystic fibrosis,
•Enrolled in the Cystic Fibrosis Patient Registry
•Receiving medical care at a Cystic Fibrosis Foundation-accredited care center providing data to the Cystic Fibrosis Patient Registry

排除标准

未提供

结局指标

主要结局

Incidence rate of fibrosing colonopathy confirmed by an independent adjudication panel in patients receiving PERTZYE.

时间窗: up to 10 years