Tofacitinib for calcinosis cutis associated with juvenile dermatomyositis: an open label single arm study
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 22
- 试验地点
- 1
- 主要终点
- Effect of Tofacitinib on calcinosis cutis in children with JDM as assesed by reduction in Agatston score using ultra low-dose whole body CT scan.
研究概览
简要总结
**Aim -**To study the role of tofacitinib in reducing the burden of calcinosis cutis associated with juvenile dermatomyositis over a 6 month follow-up period
**Primary Objectives -**To study the effect of tofacitinib as an adjunct to standard therapy in children 2 to 18 years with JDM/JDM SSc overlap (Annexure 1 and 2) and calcinosis cutis, on the burden of calcinosis cutis as assessed by reduction in Agatston score ( Annexure 5 ) using ultra-low-dose whole-body CT scan at 24 ±2 weeks follow-up compared to baseline. Standard therapy includesStable doses of steroids (<1mg/kg/day during previous 2 weeks) (if on steroids) and either ofMethotrexate (10-15 mg/m2/week) ORMMF (800 to 1000/m2/day) ORHCQ (5mg/kg/day)
Secondary Objective- To study the effect of tofacitinib as an adjunct to standard therapy in children (<18 yr) with JDM and calcinosis cutis on disease activity as assessed by proportion of children demonstrating improvement in clinical scores (CMAS and CDASI) at 24±2 weeks follow-up compared to baseline, Assess the number of children experiencing adverse events including infections, anemia, lymphopenia, transaminitis, and renal dysfunction,To assess the ability to taper steroids by analyzing the change in doses (mean) of steroids, To study the association of baseline laboratory parameters with outcomes including Myositis-associated and specific autoantibodies and INF levels (subject to availability of funds)
Methodology -
Study design - Open label single arm study
Study settings -Pediatric Rheumatology Clinic, Department of Pediatrics, All India Institute of Medical Sciences, New Delhi.
Study duration -Feb 2024 – Feb 2025 (enrolment shall start after ethical approval.)
**Study population-**Children ( 2-18years age ) diagnosed as JDM / JDM SSc overlap and calcinosis cutis coming to Pediatric Rheumatology services in the Department of Pediatrics AIIMS, New Delhi shall be screened for eligibility in the trial.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 盲法
- None
入排标准
- 年龄范围
- 2.00 Year(s) 至 18.00 Year(s)（—）
- 性别
- All
入选标准
- •1Children age 2 to 18 years with -Juvenile Dermatomyositis (JDM) OR Juvenile Dermatomyositis Systemic Scleroderma overlap (JDM-SSc) AND -calcinosis cutis on physical examination or previous imaging.
排除标准
- •1Patient cyclophosphamide during the preceding 1 month or rituximab in the last 6 months OR -Topical tacrolimus OR.
- •Active Tuberculosis OR.
- •Hemoglobin (Hb) levels less than 9 g per dL OR.
- •Absolute lymphocyte count less than 500 cells per mm3 -Absolute neutrophil count less than 1000 cells per mm3.
结局指标
主要结局
Effect of Tofacitinib on calcinosis cutis in children with JDM as assesed by reduction in Agatston score using ultra low-dose whole body CT scan.
时间窗: At baseline and 24 ±2 week follow-up
次要结局
- 1)Proportion of children demonstrating improvement in clinical scores ( CDASI and CMAS)(2)Number of subjects with adverse events.)
研究者
Dr Narendra Bagri
AIIMS New Delhi