Clinical and Immunological Long-term Follow-up of Patients With Pemphigus Included in the "RITUXIMAB 3" Trial With Medico-economic Evaluation of the Treatment.
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 80
- 试验地点
- 25
- 主要终点
- Evaluate the occurrence of long-term serious AEs and AEs in patients initially randomized in the rituximab arm and in those who have secondarily received the product during the course of their pemphigus
研究概览
简要总结
Pemphigus is an autoimmune disease specific to the skin and mucous membranes characterized by the production of IgG4 isotype autoantibodies (AC) directed mainly against two proteins involved in interkeratinocyte adhesion: desmoglein 1 (Dsg1 ) and desmoglein 3 (Dsg3) (1-3). The binding of auto-AC on these proteins disrupts their adhesion function, resulting in inter-keratinocyte dysjunction called "acantholysis" responsible for the formation of intraepidermal bubbles.
Treatment of pemphigus is typically based on systemic corticosteroids. High doses are usually necessary because of the frequent cortico-resistance of the disease. In recent years, several studies have focused on the treatment of pemphigus with anti-CD20: rituximab. The "Ritux 3" study (NCT00784589), a randomized, multicentre, randomized, non-blind clinical trial involving 90 patients, found that the use of rituximab as first-line therapy in combination with short corticosteroid therapy was extremely effective and that cortisone sparing was thus obtained limited the occurrence of side effects of treatment. On the other hand, this study showed that the 2 rituximab maintenance infusions of 500 mg to M12 and M18 allowed the maintenance of a high rate of complete remission up to the 3rd year of follow-up.
Questions remain to explain the long-term action of rituximab, in particular that of the evolution of these auto-reactive B cells (specific DSG) away from lymphocyte reconstitution B, as well as the evolution of auto-AC. anti-DSG and total IgG CAs, so as to ensure that the disappearance of the auto-reactive compartment is not accompanied by a long-term overall immunosuppression (and therefore a possible risk of infection).
The immunological changes induced in the long term as well as the precise mechanism of action of these treatments and particularly rituximab which allows a complete remission 5 years after treatment in many patients remain little known.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient included in the RITUXIMAB 3 study
- •Major patient who has read and understood the newsletter and signed the consent form
排除标准
- •Person deprived of liberty by an administrative or judicial decision
- •Person placed under the safeguard of justice
- •Person under tutorship or curators
- •Pregnant or lactating woman
研究组 & 干预措施
Rituximab
Rituximab in combination with reduced corticosteroids is administrated
干预措施: corticosteroids'therapy (Drug)
Rituximab
Rituximab in combination with reduced corticosteroids is administrated
干预措施: Rituximab (Drug)
Standard corticosteroid
Standard corticosteroid is administrated
干预措施: corticosteroids'therapy (Drug)
结局指标
主要结局
Evaluate the occurrence of long-term serious AEs and AEs in patients initially randomized in the rituximab arm and in those who have secondarily received the product during the course of their pemphigus
时间窗: 1 day: 5 to 7 years after the inclusion into RITUXIMAB 3 study
Collect of SAE and AE
Evaluate the long-term relapse rate in patients initially randomized in the rituximab arm and in those who have secondarily received the product during the course of their pemphigus
时间窗: 1 day: 5 to 7 years after the inclusion into RITUXIMAB 3 study
Collect of relapses
次要结局
未报告次要终点