Topical Ruxolitinib Cream for Refractory Cutaneous Dermatomyositis

Inclusion Criteria:

• Patients 18 years and older with refractory cutaneous symptoms related to eitherclassic dermatomyositis (CD), juvenile dermatomyositis (JD), or amyopathicdermatomyositis (AD). Diagnosis will be based on either Bohan and Peter criteria (CDand JD) or Sontheimer's criteria (AD) (19-22).

• Patients must have had a skin biopsy with histologic features consistent withdermatomyositis and current cutaneous manifestations consistent withdermatomyositis.

• Patients will be considered to have refractory disease if cutaneous manifestationsexist despite treatment with systemic corticosteroids and at least onesteroid-sparing systemic treatment commonly found to be useful in patients withdermatomyositis. These may include azathioprine, cyclosporine, mycophenolatemofetil, 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 topicalmedication in the past, with the last application of a topical medication to activeskin lesions occurring greater than 2 weeks prior to enrollment. o Commonly prescribed topical medications for dermatomyositis includecorticosteroids or calcineurin inhibitors (tacrolimus or pimecrolimus).

• Patients must have been on a stable systemic medication regimen for at least 2months (60 days) and must agree to keep the regimen stable throughout the studyperiod. As patients with dermatomyositis are commonly treated with combinationregimens that include both topical and systemic immunosuppressive medications, anyadded risk of adverse effects related to ruxolitinib 1.5% cream is considerednegligible.

• Patients must be agreeable to use appropriate contraceptive measures while enrolledin the study.

• Women of childbearing potential must be willing to practice abstinence or useeither an oral contraceptive medication or IUD if sexually active.

• Women of childbearing potential must be willing to have monthly urine pregnancytests while enrolled in the study

• Men of childbearing potential must be willing to practice abstinence or usecondoms if sexually active.

Exclusion Criteria:

• Patients with dermatomyositis who have minimal-to-no active cutaneous disease (mildinvolvement with < 1% total body surface area involved and/or CDASI activity scoreof < 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/orhave previous biopsy results suggestive of an alternative diagnosis

• Patients not on stable systemic medication regimens for at least 2 months and/or whowill not agree to keep the regimen stable throughout the study period.

• Patients who have previously taken a systemic Janus kinase inhibitor but had a poorresponse, patients who are currently taking systemic Janus kinase inhibitors, orpatients who have used a topical Janus kinase inhibitor for their dermatomyositis orany other condition and had poor responses.

• Patients with inflammatory myositis other than dermatomyositis, such as polymyositisor inclusion body myositis.

• Patients with clear features of an overlap autoimmune myositis or with aninflammatory myositis not consistent with dermatomyositis, such as polymyositis orinclusion body myositis.

• Patients with an active malignancy other than non-melanoma skin cancer, or withmalignancy-associated dermatomyositis.

• Patients younger than 18 years old