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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
Ruxolitinib is a Janus Kinase (JAK) 1/2 inhibitor. JAKs are intracellular tyrosine kinases that play an important role in the development and function of immune cells that are involved in the pathogenesis of acute GVHD. For this study, ruxolitinib cream will be manufactured by Incyte and stored and distributed by the investigational pharmacy at Cincinnati Children's Hospital Medical Center (CCHMC) using standard operating procedures. Ruxolitinib 1.5% cream will be applied as a thin film topically twice a day to areas of erythroderma and pruritus. Participants and guardians will receive an informational instruction sheet describing the amount, frequency, and technique of applying the cream. Patients may continue to use standard of care GVHD prophylaxis and moisturizers.
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Number of patients with a clinical response at 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).
Time frame: From enrollment to Day 7 (± 3 days)
Number of patients with a clinical response at Day 28 (± 3 days)
Participants will be evaluated by monitoring the clinical response, tolerability and safety of topical ruxolitinib at Day 28 (± 3 days).
Time frame: From enrollment to Day 28 (± 3 days)
Number of patients needing systemic immune suppression due to lack of response from progressive symptoms or adverse events secondary to topical ruxolitinib
Time frame: From enrollment to Day 28 (± 3 days)
Number of patients with a relapse of primary malignancy by 1 year post HSCT
Time frame: 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.
Time frame: Day 7 (± 3 days)
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