Mepolizumab for the Treatment of Eosinophilic Fasciitis; An Open-Label, Single-Arm, Exploration Study
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 发起方
- Mayo Clinic
- 主要终点
- Modified Rodnan Skin Score (mRSS)
研究概览
简要总结
This is a proof of concept pilot study to investigate the efficacy of mepolizumab in the treatment of eosinophilic fasciitis.
详细描述
This is an exploratory study designed to generate preliminary data in evaluating the efficacy of mepolizumab (Nucala) in the treatment of EF using mean change in the Rodnan Skin score (mRSS) before and after treatment. The study is powered at 90% to detect a 4 point change in the mRSS at a 5% significant level with a minimum target accrual of 6 patients.
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
- •Male and female patients 18 years or older.
- •History and physical examination consistent with EF
- •Full thickness skin biopsy characteristic of eosinophilic fascitis and/or MRI radiographic findings characteristic of EF
- •Documented peripheral eosinophilia (≥500 microliter)
- •Patients who are willing and capable of cooperating to the extent and degree required by the protocol; and
- •Patients who read and sign an approved informed consent for this study
排除标准
- •Eosinophilic fascitis disease duration > 5 years
- •Known history of adverse reaction to mepolizumab (Nucala)
- •Pregnant females
- •Females actively trying to conceive
- •Vulnerable study population
- •Asthma requiring inhaled cortiosteroids
研究组 & 干预措施
Single Arm
Patients with a biopsy proven diagnosis of eosinophilic fasciitis
干预措施: Mepolizumab (Drug)
结局指标
主要结局
Modified Rodnan Skin Score (mRSS)
时间窗: 24 weeks
Mean change before and after treatment. The mRSS is a standardized scored assessment of tissue sclerosis as assessed by palpitation.
次要结局
- Localized Scleroderma Skin Severity Index (mLoSSI) score(24 weeks)
- Localized Scleroderma Cutaneous Assessment Tool (LoSCAT)(24 weeks)
- Dermatology Quality of Life Index (DLQI)(24 weeks.)
- Physician's Global Assessment of Disease Activity (PhysGA-A)(24 weeks)
- Physician's Global Assessment of Disease Damage (PhysGA-D)(24 weeks.)
- Localized Scleroderma Damage Index (LoSDI) score.(24 weeks)
- Patient Global Assessment of Disease Severity (PtGA-S)(24 weeks.)
研究者
Jason Sluzevich MD
Assistant Professor of Dermatology, College of Medicine
Mayo Clinic