An Open Label Evaluation of Intralesional Injection of Sodium Thiosulfate in the Treatment of Cutaneous or Tendon Calcinosis in Connective Tissue Disease
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 20
- 试验地点
- 2
- 主要终点
- Primary endpoint: Change in size of calcinosis lesion from baseline to 12 weeks
研究概览
简要总结
The specific objective of this study is to perform a small, open-label study to assess the safety and efficacy of intralesional, subcutaneous injection of STS on calcinosis symptoms and lesion size in systemic sclerosis (SSc), mixed connective tissue disease (MCTD) and dermatomyositis (DM) patients. Injection will be guided by ultrasound, lesion size assessed by ultrasound, and symptom burden by patient-reported outcome measures.
详细描述
Calcinosis is a condition in which calcium builds up in the skin and tissue under the skin. It develops in ∼30% of adult dermatomyositis,18%-49% of systemic sclerosis patients, 25-40% of patients with limited systemic sclerosis. There is no standard treatment recommended by physicians currently. Many treatments have been tested, all with limited benefits and without good evidence that any are effective. They are, based only on single cases or small numbers of patients in studies called "case series." But in some cases, these agents may be effective.
There are several case series that have shown the effectiveness of injecting a chemical compound called sodium thiosulfate (or "STS") into calcinosis lesions. There was some improvement in terms of pain control, and a decrease in size and resolution of the calcinosis. The aim of our study is to test intralesional sodium thiosulfate (STS) injection for symptoms relief of calcinosis. STS has been approved by the U.S. Food and Drug Administration for certain conditions related to childhood cancers. It has not been approved by the FDA to treat calcinosis. The specific aims of this study are to assess change in calcinosis size and quality-of-life measures.
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
- •Clinical diagnosis of systemic sclerosis, mixed connective tissue disease or inflammatory myopathy
- •Must be over 18 years of age
- •Participants must be competent to give informed consent
- •Participants must have radiographic evidence (xray or ultrasound) of calcinosis.
- •Participants must need symptomatic relief
排除标准
- •Pregnant women will be excluded
研究组 & 干预措施
Sodium thiosulfate (STS)
Open label, single-arm, receiving 1-5ml of STS (250mg/ml) sodium thiosulfate (STS) injections to calcinosis lesions.
干预措施: Sodium Thiosulfate (STS) (Drug)
结局指标
主要结局
Primary endpoint: Change in size of calcinosis lesion from baseline to 12 weeks
时间窗: 12 weeks
Change in size of calcinosis lesions will be measured by x-ray at baseline and then again at 12 weeks.
次要结局
- Change in pain (interference)(up to 12 weeks)
- Change in Pain Intensity(up to 12 weeks)
- Health-related Quality of Life using the EQ-5D-5L(up to 12 weeks)
- Scleroderma Health Assessment Questionnaire (SHAQ)(up to 12 weeks)
- Change in calcinosis related symptom severity(up to 12 weeks)
研究者
Robyn T. Domsic, MD, MPH
Associate Professor
University of Pittsburgh