About this trial
This clinical trial will study ruxolitinib-based treatment of acute graft-versus-host-disease (GVHD) that developed following allogeneic hematopoietic cell transplant. Acute GVHD occurs when donor cells attack the healthy tissue of the body. The most common symptoms are skin rash, jaundice, nausea, vomiting, and/or diarrhea. The standard treatment for GVHD is high dose steroids such as prednisone or methylprednisolone, which suppresses the donor cells, but sometimes there can be either no response or the response does not last. In these cases, the GVHD can become dangerous or even life threatening. High dose steroid treatment can also cause serious complications. Researchers have developed a system, called the Minnesota risk system, to help predict how well the GVHD will respond to steroids based on the symptoms present at the time of diagnosis. The Minnesota risk system classifies patients with newly diagnosed acute GVHD into two groups with highly different responses to standard steroid treatment and long-term outcomes. This protocol maximizes efficiency because all patients with grade II-IV GVHD are eligible for screening and treatment is assigned according to patient risk. Patients with lower risk GVHD, Minnesota standard risk, have high response rates to steroid treatment. In this trial the researchers will test whether ruxolitinib alone is as effective (non-inferior) as steroid-free therapy and safe. Patients will be randomized to two different doses of ruxolitinib to identify the dose which maximizes efficacy while minimizing toxicities such as hematologic and infectious toxicities. Patients with higher risk GVHD, Minnesota high risk, have unacceptable outcomes with systemic corticosteroid treatment alone and the researchers will test whether adding ruxolitinib, a proven effective second line GVHD treatment, can improve outcomes when added to systemic corticosteroids as first line treatment.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Standard risk cohort: Minnesota standard risk GVHD (except patients with grade I [<50% BSA rash])
High risk cohort: Minnesota high risk GVHD 3 GVHD that developed after DLI for mixed chimerism or poor graft function is allowed
No prior systemic acute GVHD treatment. Topical or non-absorbed steroids are permitted.
All donor types, HLA-matches, conditioning regimens, or GVHD prophylaxis strategies are acceptable
Disqualifiers
Systemic treatment with ruxolitinib or any other JAK inhibitor within 7 days of study entry
Prior use of ruxolitinib to treat GVHD at any time
Relapsed, progressing or persistent malignancy requiring withdrawal of systemic immunosuppression
Relapse prior to development of GVHD unless subsequently in remission for at least 3 months
Trial design
Parallel
Treatments tested in this trial
Ruxolitinib
DrugRuxolitinib twice daily for 56 days followed by a short taper Given orally
Methylprednisolone
DrugStarting dose 2 mg/kg/d for at least three days, then taper Given IV or orally
Treatment groups
Trial outcomes
Primary outcomes
Day 28 Treatment Response
Day 28 response to treatment defined as complete (CR), very good partial (VGPR), or partial (PR) response without intervening therapy or death to ruxolitinib monotherapy. CR - All evaluable organs (skin, liver, GI tract) stage 0. For a response to be scored as CR, the patient must be in CR on the date of assessment and have had no intervening additional GVHD therapy. VGPR - Stage 0 liver and GI and residual stage 1 skin GVHD. For a response to be scored as VGPR, the patient must be in VGPR on the date of assessment and have had no intervening additional GVHD therapy. PR - An improvement by one or more stages in one or more organ involved with GVHD symptoms without worsening in others. For a response to be scored as PR, the patient must be in PR on the date of assessment and have had no intervening additional GVHD therapy.
Secondary outcomes
Steroid-refractory GVHD
Steroid Refractory (SR) GVHD is defined as GVHD that either does not achieve a clinical response or an additional line of therapy is initiated by day 28 from the initiation of systemic steroid therapy for non-responsive GVHD.
Durable response at day 56
Durable response at day 56 is defined as CR, VGPR, or PR by day 28 and no GVHD flare, intervening treatment or death by day 56
GVHD flares
Flare is defined as an increase in acute GVHD severity in at least one target organ by at least one stage that is treated with an additional line of treatment or an increase in steroid dose in methylprednisolone equivalents (MPE) by at least 25%
Cumulative systemic corticosteroid dose
Sponsors and contacts
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John Levine
Lead sponsor
Icahn School of Medicine at Mount Sinai
Sponsor institution
Incyte Corporation
Collaborator
National Cancer Institute (NCI)
Collaborator