Acute Skin Graft Versus Host Disease (GVHD)
Conditions
Brief summary
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).
Detailed description
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.
Interventions
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.
Sponsors
Children's Hospital Medical Center, Cincinnati
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Single arm, open-label pilot study. No randomization
Eligibility
Sex/Gender
ALL
Age
2 Years to 35 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of patients with a clinical response at Day 7 (± 3 days) | 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) | 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). |
Secondary
| Measure | Time frame |
|---|---|
| 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) |
Countries
United States
Contacts
CONTACTMonica Trapp, BSN, RN, TCTCN
PRINCIPAL_INVESTIGATORZahra Hudda, MD
Children's Hospital Medical Center, Cincinnati
Outcome results
None listed