SHEPHERD: SAFETY IN HEMOLYTIC PNH PATIENTS TREATED WITH ECULIZUMAB

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。 这是一项在约85例溶血性阵发性睡眠性血红蛋白尿症（PNH）患者中静脉（IV）给予依库珠单抗的开放标签多中心研究。符合入选标准的患者将接受600 mg依库珠单抗IV，每周一次，共4剂，然后在1周后接受900 mg依库珠单抗IV，共1剂，然后每2周一次接受900 mg依库珠单抗IV，共约24剂。完成研究的每例患者将接受约29次IV输注。考虑到12周入组期、2周筛选期、52周治疗期和8周治疗后期，估计研究持续时间约为74周。提前停用研究药物的患者仍然需要在研究药物末次给药后1、2、4和8周进行4次随访访视，并在研究药物末次给药后12周进行随访联系。在与假期、旅行或天气问题或患者的医疗需求相关的罕见情况下，患者可被允许将访视间隔改变2天。如果患者缩短了访视间隔，则可能需要额外访视以满足52周治疗期。将进行药代动力学（PK）和药效学（PD）（溶血%）采样，以研究剂量-效应关系。将探索关注的协变量。

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 an open-label multicenter study of Eculizumab administered intravenously (IV) to approximately 85 patients with hemolytic paroxysmal nocturnal hemoglobinuria (PNH). Patients who meet the entry criteria will receive 600 mg of Eculizumab IV once a week for 4 doses, followed by 900 mg Eculizumab IV 1 week later for 1 dose, then 900 mg Eculizumab IV every 2 weeks for approximately 24 doses. Each patient who completes the study will receive approximately 29 IV infusions. The estimated duration of the study, given a 12-week enrollment period, 2-week screening period, 52-week treatment period, and 8-week post-treatment period is approximately 74 weeks. Patients who prematurely discontinue study medication will nevertheless require 4 follow-up visits at 1, 2, 4, and 8 weeks post last dose of study medication and, a follow-up contact 12 weeks after their last dose of study medication. In rare circumstances related to holidays, travel or weather issues, or the patient's medical needs, a patient may be allowed to vary the visit interval by 2 days. If a patient has shortened visit intervals, additional visits may be required in order to meet the 52-week treatment period. Sampling for pharmacokinetics (PK) and pharmacodynamics (PD) (% hemolysis) will be done for the purpose of studying the dose-effect relationship. Covariates of interest will be explored.