Topical Ruxolitinib Cream for Refractory Cutaneous Dermatomyositis
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- CDASI
研究概览
简要总结
This study will assess the safety and efficacy of topical ruxolitinib for treating the refractory cutaneous manifestations in patients with dermatomyositis. The investigators' hypothesis is that topical ruxolitinib will be both safe and effective for such patients.
详细描述
Dermatomyositis (DM) is an immune-mediated disease that most commonly affects the skin and the muscles. Cutaneous involvement of DM can lead to ulceration with secondary infection, in addition to permanent skin damage in the form of atrophy, scarring, calcinosis, decreased range of motion or lipoatrophy. Furthermore, persistent cutaneous DM is associated with adverse psychological effects and physical symptoms such as pain, burning, and pruritus. The combination of these contribute to a significant negative impact on DM patients' quality of life. Thus, effective treatment of cutaneous DM represents an important therapeutic goal.
Cutaneous DM symptoms and inflammation are often refractory to currently available topical medications . Additionally, continued topical corticosteroid use is associated with well-known adverse effects, including potential worsening of DM-related skin atrophy and adrenal insufficiency.
Recently, topical ruxolitinib has been developed and studied in several dermatologic conditions. In phase 2 and 3 trials in atopic dermatitis, topical ruxolitinib 1.5% cream resulted in significant efficacy in terms of improving both disease activity and itching, without significant side effects. The investigators propose to study use of topical ruxolitinib 1.5% cream as an adjunct for treatment of refractory cutaneous DM lesions in patients who have previously failed other topical medication options.
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 89 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients 18 years and older with refractory cutaneous symptoms related to either classic dermatomyositis (CD), juvenile dermatomyositis (JD), or amyopathic dermatomyositis (AD). Diagnosis will be based on either Bohan and Peter criteria (CD and JD) or Sontheimer's criteria (AD) (19-22).
- •Patients must have had a skin biopsy with histologic features consistent with dermatomyositis and current cutaneous manifestations consistent with dermatomyositis.
- •Patients will be considered to have refractory disease if cutaneous manifestations exist despite treatment with systemic corticosteroids and at least one steroid-sparing systemic treatment commonly found to be useful in patients with dermatomyositis. These may include azathioprine, cyclosporine, mycophenolate mofetil, IVIG, methotrexate, hydroxychloroquine, cyclophosphamide, chlorambucil, sirolimus, tacrolimus, and rituximab.
- •Patients must have sufficiently active cutaneous involvement of dermatomyositis (BSA>1% to <20%, CDASI activity score > 6, and Physician Global Assessment (PGA) activity score >2).
- •Patients must have tried and failed at least one commonly prescribed topical medication in the past, with the last application of a topical medication to active skin lesions occurring greater than 2 weeks prior to enrollment.
- •o Commonly prescribed topical medications for dermatomyositis include corticosteroids or calcineurin inhibitors (tacrolimus or pimecrolimus).
- •Patients must have been on a stable systemic medication regimen for at least 2 months (60 days) and must agree to keep the regimen stable throughout the study period. As patients with dermatomyositis are commonly treated with combination regimens that include both topical and systemic immunosuppressive medications, any added risk of adverse effects related to ruxolitinib 1.5% cream is considered negligible.
- •Patients must be agreeable to use appropriate contraceptive measures while enrolled in the study.
- •Women of childbearing potential must be willing to practice abstinence or use either an oral contraceptive medication or IUD if sexually active.
- •Women of childbearing potential must be willing to have monthly urine pregnancy tests while enrolled in the study
- •Men of childbearing potential must be willing to practice abstinence or use condoms if sexually active.
排除标准
- •Patients with dermatomyositis who have minimal-to-no active cutaneous disease (mild involvement with < 1% total body surface area involved and/or CDASI activity score of < 6).
- •Patients who have > 20% total BSA involvement of cutaneous dermatomyositis.
- •Patients who have used a common prescription topical medication within the previous 2 weeks.
- •Patients whose cutaneous findings are not consistent with dermatomyositis and/or have previous biopsy results suggestive of an alternative diagnosis
- •Patients not on stable systemic medication regimens for at least 2 months and/or who will not agree to keep the regimen stable throughout the study period.
- •Patients who have previously taken a systemic Janus kinase inhibitor but had a poor response, patients who are currently taking systemic Janus kinase inhibitors, or patients who have used a topical Janus kinase inhibitor for their dermatomyositis or any other condition and had poor responses.
- •Patients with inflammatory myositis other than dermatomyositis, such as polymyositis or inclusion body myositis.
- •Patients with clear features of an overlap autoimmune myositis or with an inflammatory myositis not consistent with dermatomyositis, such as polymyositis or inclusion body myositis.
- •Patients with an active malignancy other than non-melanoma skin cancer, or with malignancy-associated dermatomyositis.
- •Patients younger than 18 years old
研究组 & 干预措施
Treatment
1.5 % Ruxolitinib cream
干预措施: Ruxolitinib Topical Cream (Drug)
结局指标
主要结局
CDASI
时间窗: 8 weeks
Change in Cutaneous Dermatomyositis Activity and Severity Index (CDASI) activity score (range 0 - 100; higher scores indicate greater disease activity) at 8 weeks
次要结局
- CDASI(week 12)
研究者
Anthony Fernandez, MD, PhD
Director of Medical Dermatology; W.D. Steck Chair of Clinical Dermatology
The Cleveland Clinic