Efficacy of Topical Ruxolitinib in Acute Skin GVHD: A Pilot Study
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Number of patients with a clinical response at Day 7 (± 3 days)
研究概览
简要总结
The purpose of this study is to see if ruxolitinib, a Janus Kinase (JAK) 1/2 inhibitor, is safe for people who have acute skin graft versus host disease (GVHD) following allogenic hematopoietic stem cell transplantation (HSCT).
详细描述
The purpose of this study is to see if ruxolitinib, a Janus Kinase (JAK) 1/2 inhibitor, is safe for people who have acute skin graft versus host disease (GVHD) following allogenic hematopoietic stem cell transplantation (HSCT). This is a single arm, open-label pilot study. There is no randomization. This study will enroll approximately 10 patients with underlying malignancies with acute skin GVHD. Participants will be treated with topical ruxolitinib for 28 days or until the end of treatment if earlier resolution of GVHD is achieved.
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
- 2 Years 至 35 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥2 through age 35 years
- •Acute skin GVHD (any grade).
- •Underlying diagnosis of malignancy.
- •No concurrent systemic immune suppression to treat acute GVHD. This excludes agents like calcineurin inhibitors, mycophenolate mofetil, and abatacept which prevent acute GVHD and are not used for treatment as per clinical practice may continue.
- •Use of prior topical steroids or topical tacrolimus will be allowed but these will need to be discontinued 24 hours prior to study enrollment.
排除标准
- •Active uncontrolled skin infections.
- •History of allergic reactions to topical ruxolitinib.
- •Concomitant steroids or have received investigational therapies within the previous 30 days.
研究组 & 干预措施
Ruxolitinib
Ruxolitinib 1.5% cream will be applied topically twice a day to areas of erythroderma and pruritus.
干预措施: Ruxolitinib 1.5% cream (Drug)
结局指标
主要结局
Number of patients with a clinical response at Day 7 (± 3 days)
时间窗: From enrollment to Day 7 (± 3 days)
Participants will be evaluated by monitoring the clinical response, tolerability and safety of topical ruxolitinib at Day 7 (± 3 days). Patients who respond by Day 7 (± 3 days) will continue treatment until resolution of symptoms or till Day 28 (± 3 days).
Number of patients with a clinical response at Day 28 (± 3 days)
时间窗: From enrollment to Day 28 (± 3 days)
Participants will be evaluated by monitoring the clinical response, tolerability and safety of topical ruxolitinib at Day 28 (± 3 days).
次要结局
- Number of patients needing systemic immune suppression due to lack of response from progressive symptoms or adverse events secondary to topical ruxolitinib(From enrollment to Day 28 (± 3 days))
- Number of patients with a relapse of primary malignancy by 1 year post HSCT(From enrollment to 1 year post HSCT)
- Blood ruxolitinib concentration levels at documented body surface area (BSA) applications and their association with adverse effects and relapse rates.(Day 7 (± 3 days))