DarTTP: an Observational, International, Multicentric Study on Daratumumab in Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Proportion of responders to daratumumab
研究概览
简要总结
Data about efficacy and safety of daratumumab in iTTP refractory or intolerant to standard immunosuppressive treatments are scarce. Therefore, the investigators aim at collecting evidence on a larger number of patients through a collaborative, international study.
详细描述
iTTP in an autoimmune disease caused by autoantibodies directed against the metalloproteinase ADAMTS13. Rituximab is the standard immune suppressive treatment suggested from international guidelines. However, 10-15% of patients do not achieve a sustained ADAMTS13 remission with rituximab, and a significant portion of responders eventually need re-treatment after 12 months or less. Other therapeutic options are scarce and based on old immunosuppressive agents or splenectomy, all burdened by relevant toxicity and lack of solid efficacy data. Recently, targeting CD20-negative long-lived plasma cells appears to be a promising strategy in refractory iTTP. The anti-CD38 monoclonal antibody daratumumab has been employed in selected iTTP patients with good results. However, evidence stems only from isolated case reports.
The DarTTP study aims to collect evidence on a larger number of patients about the efficacy and safety of daratumumab in iTTP subjects who are refractory or intolerant to previous immunosuppressive treatments. The primary endpoint is the proportion of patients with ADAMTS13 activity levels above 20% of normal at 6 months from the first daratumumab administration.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 99 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •patients with a confirmed diagnosis of iTTP (i.e., ADAMTS13 activity <10% with anti-ADAMTS13 antibodies detected);
- •aged ≥ 18 years;
- •male and female patients;
- •treated with daratumumab for iTTP.
排除标准
- •patients unwilling or unable to provide their informed consent;
- •follow-up < 6 months after daratumumab administration.
研究组 & 干预措施
Adult patients with iTTP
Patients with a confirmed diagnosis of iTTP, aged ≥ 18 years, treated with daratumumab for iTTP
结局指标
主要结局
Proportion of responders to daratumumab
时间窗: From the first daratumumab administration to 12 weeks after the last dose
Number of patients responding to daratumumab / total number of patients treated with daratumumab (response defined as the achievement of ADAMTS13 activity levels above 20% of normal at any timepoint from the first daratumumab administration to 12 weeks after the last dose, without new additional immunosuppressants)
次要结局
- Safety of daratumumab(From the first daratumumab administration to 24 weeks after)
- Duration of response(From the date of the first documented ADAMTS13 activity >20% after the first dose of daratumumab until the date of ADAMTS13 relapse (i.e., ADAMTS13 activity <20%), assessed up to 3 years.)