Proof-of-Concept Study of IVIg Efficacy in Patients With Cutaneous Lupus Erythematosus
试验速览
- 阶段
- 早期 1 期
- 状态
- 已完成
- 入组人数
- 16
- 试验地点
- 2
- 主要终点
- Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Activity Score (CLASI - TAS)
研究概览
简要总结
The purpose of this research study is to show that non-steroidal treatment with intravenous immunoglobulin (IVIg) can replace current systemic immunosuppressive therapy in cutaneous lupus erythematosis (CLE) patients.
详细描述
The ultimate goal of this pilot project is to generate proof-of-concept data showing that treatment with intravenous immunoglobulin (IVIg) can replace current systemic immunosuppressive therapy in cutaneous lupus erythematosis (CLE) patients. This project has relevant clinical implications due to the severe side effects of and lack of response to current therapies.
From the review of literature, it can be postulated that:
- the beneficial effects of IVIg for patients with CLE should be prompt, with marked improvement within a few weeks;
- clinical improvement should last several weeks after the last infusion; and
- remission may be prolonged by maintenance IVIg therapy.
Although this is only a non-controlled study, the investigator expects that IVIg will improve CLE, including those resistant to standard treatments. It is anticipated that treatment with IVIg will facilitate healing of extensive cutaneous lesions and achieve rapid remission. Maintenance therapy with repeated monthly pulses of IVIg is expected to keep the disease in remission during the treatment-free follow up observational period. The results will provide the basis a multicenter randomized controlled study to identify which CLE subsets will benefit the most and which protocol will provide the optimal clinical outcome.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the trial.
- •Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
- •Be at least 18 years of age at time of informed consent.
- •Have had a diagnosis of CLE
- •Currently has active CLE (any subtype) established by standard clinical and histo- and immunopathologic criteria
- •Falls into one of the two following cohorts:
- •Cohort 1 - Has received a standard systemic therapy without a therapeutic response for a minimum of one month
- •Cohort 2 - Has not received any systemic treatment
排除标准
- •Subject is not over 18 years of age.
- •Subject cannot understand or follow directions.
- •Subject is a female of child-bearing potential and unwilling to use a form of highly effective birth control.
- •Subject is pregnant, planning to get pregnant, or breast feeding.
- •Subject has a known history of immunoglobulin A (IgA) deficiency.
研究组 & 干预措施
IVIg as a monotherapy
IVIg will be used as a first line treatment. Topical treatment will be stopped at the beginning of IVIg therapy.
干预措施: IVIg (Drug)
结局指标
主要结局
Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Activity Score (CLASI - TAS)
时间窗: Initial, 1st Visit - 9th Visit
Disease activity will be measured using the CLASI activity score that describes the activity of the disease. This score ranges from 0-70, with higher scores indicating more severe skin disease. This clinical assessment tool enables standardized assessments of response to therapy. Results expressed relative to the mean initial value of all patients, taken as 100%. Each proceeding visit will be relative to the initial visit. A decrease in percentage represents improvement while an increase in percentage indicates worsening of CLE. Visits occur on a month-to-month basis.
Skindex 29
时间窗: Initial, 1st Visit - 9th Visit
The subjects also evaluated their skin-specific quality of life with the Skindex-29 - the questionnaire consisting of 29 items used to calculate three subscales: symptoms (pain, itch, burning, sensitivity), emotions (depression, anxiety, embarrassment, anger) and functioning (sleep, relationships with others). All assessments were repeated at all study visits. Results expressed relative to the mean initial value of all patients, taken as 100%. Each proceeding visit will be relative to the initial visit. A decrease in percentage represents improvement while an increase in percentage indicates worsening of CLE. Visits occur on a month-to-month basis.
次要结局
- Mean Percent Change in Physician's Subjective Assessment of Improvement (PSAI)(Initial, 1st Visit - 9th Visit)
- Mean Percent Change in Physician's Subjective Assessment of Severity (PSAS)(Initial, 1st Visit - 9th Visit)
- Cutaneous Lupus Erythematosus Disease Area and Severity Index - Total Damage Score (CLASI - TDS)(Initial, 1st Visit - 9th Visit)
研究者
Sergei Grando
Professor
University of California, Irvine