SHEPHERD: SAFETY IN HEMOLYTIC PNH PATIENTS TREATED WITH ECULIZUMAB

这个子项目是利用由NIH/NCRR资助的中心拨款提供的资源的许多研究子项目之一。子项目和调查员(PI)可能从另一个NIH来源获得了主要资金，因此可能会出现在其他CRISE条目中。列出的机构是针对中心的，而不一定是针对调查员的机构。这是一项开放标签的多中心研究，Eculizumab静脉注射(IV)给大约85名溶血性阵发性睡眠性血红蛋白尿(PNH)患者。符合入选标准的患者将每周接受一次600毫克的Eculizumab IV，共4剂，然后在1周后接受900 mg Eculizumab IV，共1剂，然后每2周接受900 mg Eculizumab IV，共约24剂。每个完成研究的患者将接受大约29次静脉注射。考虑到12周的注册期、2周的筛查期、52周的治疗期和8周的治疗后期，这项研究的估计持续时间约为74周。然而，过早停止研究用药的患者仍需要在最后一剂研究用药后1、2、4和8周进行4次随访，并在最后一剂研究用药后12周进行随访接触。在与假期、旅行或天气问题或患者的医疗需要有关的极少数情况下，患者可能被允许将就诊间隔更改为2天。如果患者缩短了就诊间隔，可能需要额外就诊才能达到52周的治疗期。将进行药代动力学(PK)和药效学(PD)(溶血百分比)采样，以研究剂量-效应关系。将探索感兴趣的协变量。

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.