A Double-Blind, Randomized, Placebo-Controlled Study to Assess the Safety and Efficacy of Intravenous Immunoglobulin Treatment in Patients With Systemic Sclerosis
试验速览
- 阶段
- 不适用
- 入组人数
- 14
- 试验地点
- 4
- 主要终点
- To study the effects of IVIG on the skin in patients with scleroderma
研究概览
简要总结
The purpose of this research is to study the effects of Privigen (intravenous immunoglobulin) on the skin in patients with scleroderma. Approximately 24 subjects will take part in this investigator-initiated study at Georgetown University Hospital and Johns Hopkins Hospital. This study will last for one year (12 months). This research is being done because systemic sclerosis can cause severe, progressive organ involvement. The investigators hope this study treatment will improve the outcomes in this disease, including skin, muscle, joint, gastrointestinal, and lung involvement.
详细描述
The investigators propose a double-blind controlled trial of 24 patients with Privigen® (Immune Globulin Intravenous (Human), 10% liquid treatment) with 3:1 randomization. Subjects with scleroderma will be given 2 g/kg/mo of Privigen® or placebo (Albuminar®-5) for 6 months. Scleroderma is generally a progressive disease, and while the skin does improve with time in some patients, others have progressive disease in spite of aggressive treatment. Also, spontaneous improvement in other organ systems is even less likely. Patients entered into the trial will have failed to respond to standard of care treatment over the past 4 months. Thus, the investigators feel that any actual improvement observed can be attributed to the IVIG treatment. Since this is a pilot study, future larger controlled trials will be necessary to clearly demonstrate the effectiveness, but the investigators are hoping that this study will give us signals that will guide the future clinical trial.
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
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diffuse systemic sclerosis with active skin involvement as defined as a mRSS >12, with no improvement or worsening in the previous 4 months in spite of treatment with methotrexate, cellcept, imuran, or anti-TNF agent;
- •18 years of age or older;
- •Disease duration of less than 5 years from the first non-Raynaud's symptom of scleroderma.
排除标准
- •Use of more than 10 mg of prednisone, or any dose of cytoxan, d-penicillamine, or rituximab in the last 3 months;
- •History of deep vein thrombosis (DVT), stroke, or other thromboembolic phenomenon;
- •History of anaphylaxis or other serious reaction to human blood or blood products.
- •Absolute IgA deficiency
- •A prior receipt of IVIg treatment
结局指标
主要结局
To study the effects of IVIG on the skin in patients with scleroderma
时间窗: At the 4 month visit
The modified Rodnan Skin Score (mRSS) will be used to determine the changes in skin
次要结局
- To evaluate the effects of IVIG on pulmonary function(At the 4 month visit)
- To determine any toxicity of IVIG in scleroderma(At the 4 month visit)
- Muscle, joint, and inflammatory parameters(At the 4 month visit)