Treatment of Anti-Interferon-Gamma Autoantibody Associated Acquired Immunodeficiency Syndrome With Bortezomib: Pilot Study
试验速览
- 阶段
- 2 期
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- Adverse event
研究概览
简要总结
This is pilot study aimed to investigate the application of proteosome inhibitor, Bortezomib in treatment of patients with neutralizing autoantibody to IFN-γ. The investigators hypothesis is that bortezomib will reduce the antibody level and restore interferon-gamma function, resulting in clinical improvement and should be well-tolerated and safe for use in patients with autoantibody to IFN-γ.
详细描述
The patients with autoantibody to IFN-γ and past or current history of proven opportunistic infection will receive 2 treatment cycles of bortezomib subcutaneously (4 injections of 1.3 mg Bortezomib /m2 body surface per cycle), followed by low dose oral cyclophosphamide for 4 month after completion treatment with bortezomib.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 21 Years 至 60 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 21 - 60 years
- •Positive anti-interferon-gamma autoantibody
- •Anti-HIV negative
- •Past or current infection with opportunistic infection (OI) such as nontuberculous mycobacteria, proven by culture
- •Ability to give written consent, informed written consent
- •Negative pregnancy test in premenopausal woman
- •Receving antimicrobial for treatment of OI for at least one month.
排除标准
- •Pregnancy or lactation
- •Absolute neutrophil count <1.5 × 109/ L or platelet count <100× 109/ L or hemoglobin level < 8 g/dL
- •Past history of myocardial infarction or heart failure within 6 months before enrollment or prolonged QT interval > 450 msec at screening
- •Renal insufficiency (GFR < 30 ml/min)
- •Abnormal liver function test (AST> 3 times of UNL)
- •Known cancer or receiving other immunosuppressive agent
- •Known intolerability to Bortezomib
研究组 & 干预措施
Bortezomib and cyclophosphamide
Two cycles of bortezomib administered subcutaneously, followed by 4 months of low dose oral cyclophosphamide.
干预措施: Bortezomib (Drug)
Bortezomib and cyclophosphamide
Two cycles of bortezomib administered subcutaneously, followed by 4 months of low dose oral cyclophosphamide.
干预措施: Cyclophosphamide (Drug)
结局指标
主要结局
Adverse event
时间窗: 6 months after first dose of bortezomib (after completion 4 months of cyclophosphamide)
Grade 4 adverse event (probably related)
Titers of anti-interferon-gamma antibody after treatment with bortezomib
时间窗: 8 weeks after first dose of bortezomib (after completion 2 cycles of bortezomib)
Change in titers of anti-interferon-gamma antibody after treatment with
Titers of anti-interferon-gamma antibody after treatment with bortezomib and cyclophosphamide
时间窗: 1 year after first dose of bortezomib
Change in titers of anti-interferon-gamma antibody after treatment with bortezomib and cyclophosphamide
次要结局
- Disease relapse(6 month and 1 year after first dose of bortezomib)