EVALUATING THE SAFETY AND EFFICICACY OF INFLIXIMAB (REMICADE)

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。所列机构为中心机构，不一定为研究者机构。这是一项II期、52周、多中心、随机化、双盲、安慰剂对照临床研究，旨在比较英夫利西单抗与安慰剂的2种治疗方案（3 mg/kg或5 mg/kg）治疗尽管接受当前治疗但仍有症状的慢性酸中毒伴肺部受累受试者的安全性和疗效，目的是改善肺功能。将以探索性方式研究肺外受累。确定为特殊关注的两个受试者子集是基线时皮肤受累的受试者和基线时眼睛受累的受试者。将在第0、2、6、12、18和24周输注英夫利西单抗（3 mg/kg或5 mg/kg）或安慰剂。将在北美和欧洲的约25家临床试验机构入组约120例受试者。本研究的主要终点是第24周FVC预测值百分比较基线的变化。在第24周，将评估所有受试者的主要和次要终点。受试者将继续随访至第52周，以评估安全性和停药后任何临床效应的持久性。计划进行两次数据库锁定（第24周数据清理和定稿后1次，第52周数据清理和定稿后1次）。

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 Phase 2, 52wk, multicenter, randomized, double-blind, placebo-controlled clinical study to compare the safety and efficacy of 2 treatment regimens (3mg/kg or 5mg/kg) of infliximab with placebo in the treatment of subjects with chronic acidosis with pulmonary involvement who are symptomatic despite current treatment, with a goal of improving lung function. Extrapulmonary involvement will be investigated in a exploratory manner. Two subsets of subjects identified as special interests are subjects with skin involvement at baseline and subjects with eye involvement at baseline. Infliximab (3mg/kg or 5mg/kg) or placebo will be infused at weeks 0, 2, 6, 12, 18 and 24. Approximately 120 subjects will be enrolled in approximately 25 investigational centers located in North America and Europe. The primary end point of this study is the change from baseline in the percent of predicted FVC at wk 24. At wk 24, all subjects will be assessed for the primary and secondary endpoints. Subjects will continue to be followed to wk 52 for assessment of safety and the durability of any clinical effects after discontinuation of therapy. Two database locks are planned (1 after wk-24 data are cleaned and finalized and 1 after wk-52 data are cleaned and finalized).