Topical Ruxolitinib in Acute Skin GVHD (Graft Versus Host Disease)

Efficacy of Topical Ruxolitinib in Acute Skin GVHD: A Pilot Study

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).

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.

Inclusion Criteria:

• 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.

Exclusion Criteria:

• Active uncontrolled skin infections.
• History of allergic reactions to topical ruxolitinib.
• Concomitant steroids or have received investigational therapies within the previous 30 days.