Phase I/II Clinical Trial Evaluating Rituximab in Ocular Cicatricial Pemphigoid
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 3
- 试验地点
- 2
- 主要终点
- 2. The Proportion of Patients That Experience a Grade 3, Grade 4, or Grade 5 Toxicity Based Reaction on the NCI-CTC System at the Time of Their Infusions and During Follow-up Visits.
研究概览
简要总结
Cicatricial pemphigoid is an autoimmune blistering disease which affects the skin, mucous membranes, and, in a small subset of patients, the eye. Progressive ocular disease can lead to irreversible damage and blindness. Conventional treatments include systemic steroids, dapsone, and immunosuppressive agents. These treatments, however, are not successful with all patients. Rituximab has been very effective in the treatment of other autoimmune disorders, and has recently been shown to be effective for autoimmune blistering pemphigus. We propose that it will also be effective in the treatment of cicatricial pemphigoid.
详细描述
Cicatricial pemphigoid is an autoimmune blistering disease which can affect the skin, mucous membranes, and, in a small subset of patients, the eyes. Progressive ocular disease can lead to irreversible damage and blindness. Conventional treatments have included high dose systemic steroids, dapsone, and immunosuppressive agents such as azathioprine, methotrexate, cyclophosphamide, and mycophenolate mofetil. However, there are a subgroup of patients who fail to respond to these treatments, develop intolerable side effects, or have contraindications to their use. Patients may also develop resistance to these conventional treatment modalities. For these reasons, alternative treatment modalities are needed. Rituximab has been very effective in the treatment of other autoimmune disorders. It has recently been shown to be effective in the treatment of another autoimmune blistering disorder known as pemphigus. We thus propose that Rituximab will be effective in the treatment of cicatricial pemphigoid.
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
- 19 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of ocular cicatricial pemphigoid (symptoms of conjunctivitis, irritation, burning, increased lacrimation, photophobia, dryness of the eyes along with conjunctival inflammation, trichiasis, and scarring
- •One of the following:
- •Failed response to the use of one or more conventional treatments for a minimum of 10 weeks; or
- •Minimal conventional medication doses, with a significant adverse effects, contradiction to use, or progressive disease despite treatment
- •Adults age 19 and older
- •Adequate renal function as indicated by serum creatinine levels less than 1.5
排除标准
- •known hypersensitivity to rituximab or its components
- •Age less than 19 years
- •Any other condition deemed by the investigator to be a significant hazard to the subject if the investigational therapy were initiated.
研究组 & 干预措施
Rituximab
The Rituximab dose is 1000 mg (1gm) given as an IV infusion every two weeks for 2 doses (Days 1 and 15).
干预措施: Rituximab (Drug)
结局指标
主要结局
2. The Proportion of Patients That Experience a Grade 3, Grade 4, or Grade 5 Toxicity Based Reaction on the NCI-CTC System at the Time of Their Infusions and During Follow-up Visits.
时间窗: 16 weeks
Number of Participants With no Evidence of Further Scarring (Fosters Staging) at 16 Weeks
时间窗: 16 weeks
Stages Characteristics I Subconjunctival scarring and fibrosis II Fornix foreshortening (a-d describes % loss of inferior fornix depth) 1. 0-25% 2. 25-50% 3. 50-75% 4. 75-100% III Presence of symblepharon and number (n) (a-d describes % of horizontal involvement by symblepharons and n is the number of symblepharons countable) a. 0-25% b. 25-50% c. 50-75% d. 75-100% IV Ankyloblepharon, frozen globe
次要结局
- 1. Stability of Visual Acuity (Snellen's Test) at 16 Weeks(16 weeks)
- 2. Stability of Visual Acuity (Snellen's Test) at 24 Weeks(24 weeks)
研究者
Craig Elmets
Principal Investigator
University of Alabama at Birmingham