PATIENTS WITH TRANSFUSION DEPENDANT (PNH)

输血依赖 (PNH) 患者

• 批准号: 7375310
• 负责人: STEVEN E COUTRE
• 金额: $ 0.89万
• 依托单位: STANFORD UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2005
• 资助国家: 美国
• 起止时间: 2005-12-01 至 2006-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7375310
• 关键词: PATIENTS; TRANSFUSION; DEPENDANT; PNH

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. Hypothesis: The long-term safety of Eculizumab, a humanized monoclonal antibody that specifically binds the terminal complement protein C5, will be evaluated in patients with transfusion-dependent hemolytic paroxysmal nocturnal hemoglobinuria (PNH). This is an open-label study of patients who have completed the TRIUMPH (GCRC Protocol #853), SHEPHERD (GCRC Protocol #899) or X03-001 trials. Goals: PNH is an acquired clonal disorder of the hematopoietic stem cell, characterized by intravascular hemolysis, hemoglobinuria, anemia, and thrombosis (1). It is a rare condition with an incidence of 2 to 6 per million and a prevalence between 10,000 and 30,000 in the United States. The estimated survival of PNH patients after diagnosis is 50% at 10 yrs and 28% at 25 yrs, with a majority of the deaths resulting from thrombosis or bone marrow hypoplasia. Experimental Design: This is an open-label study of approximately 2 yrs (104 weeks). Patients who have completed the SHEPHERD or X03-001 trials will directly enter the open-label phase of this extension. To preserve the blinded nature of the TRIUMPH study, patients who have completed the TRIUMPH study will undergo a blind induction period prior to entering the open-label phase of this trial. Open-Label Treatment Phase: Patients will receive 900 mg eculizumab via IV infusion every 2 weeks.

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。假设：Eculizumab是一种特异性结合终末补体蛋白C5的人源化单克隆抗体，将在输注依赖性溶血性突发性夜间血红蛋白尿（PNH）患者中进行长期安全性评估。这是一项开放标签研究，患者已完成TRIUMPH （GCRC协议#853）、SHEPHERD （GCRC协议#899）或X03-001试验。目的：PNH是一种获得性造血干细胞克隆性疾病，以血管内溶血、血红蛋白尿、贫血和血栓形成为特征(1)。这是一种罕见的疾病，发病率为百万分之2至6，在美国的患病率为1万至3万。确诊后PNH患者10年的估计生存率为50%，25年的估计生存率为28%，其中大多数死亡是由于血栓形成或骨髓发育不全。实验设计：这是一项为期约2年（104周）的开放标签研究。完成SHEPHERD或X03-001试验的患者将直接进入该扩展的开放标签阶段。为了保持TRIUMPH研究的盲性，已完成TRIUMPH研究的患者在进入该试验的开放标签阶段之前将经历一段盲诱导期。开放标签治疗阶段：患者将每2周接受900mg eculizumab静脉输注。