Phase 2 Study of Ruxolitinib-Based Primary Treatment for Acute GVHD

Status: Recruiting
Location: See all (11) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY

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
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• 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

• ≥18 years of age

• Standard risk cohort: Hematopoietic engraftment with absolute neutrophil count (ANC) ≥ 1000/μL and platelet count ≥20,000. Use of growth factor supplementation and transfusions to maintain adequate hematologic parameters are allowed.

• High risk cohort: Hematopoietic engraftment with ANC ≥ 500/uL and platelet count ≥20,000. Use of growth factor supplementation and transfusions to maintain adequate hematologic parameters are allowed.

Locations
United States
California
City of Hope Comprehensive Cancer Center
NOT_YET_RECRUITING
Duarte
Florida
Moffitt Cancer Center
NOT_YET_RECRUITING
Tampa
Georgia
Winship Cancer Institute, Emory University
NOT_YET_RECRUITING
Atlanta
Massachusetts
Dana Farber Cancer Institute
NOT_YET_RECRUITING
Boston
Massachusetts General Hospital
NOT_YET_RECRUITING
Boston
Minnesota
Mayo Clinic
NOT_YET_RECRUITING
Rochester
Missouri
Washington University
NOT_YET_RECRUITING
St Louis
New York
Icahn School of Medicine at Mount Sinai
RECRUITING
New York
Ohio
Ohio State University
NOT_YET_RECRUITING
Columbus
Texas
MD Anderson Cancer Center
NOT_YET_RECRUITING
Houston
Washington
Fred Hutchinson Cancer Research Center
NOT_YET_RECRUITING
Seattle
Contact Information
Primary
Rachel Young, BA
rachel.young@mssm.edu
646-937-1246
Backup
Janna Baez, MA
janna.baez@mssm.edu
646-937-1552
Time Frame
Start Date: 2025-05-14
Estimated Completion Date: 2028-04-14
Participants
Target number of participants: 98
Treatments
Experimental: Minnesota Standard Risk lower dose
Patients receive lower dose ruxolitinib orally (PO) twice daily (BID) for 56 days then begin taper PO once daily (QD) for 1 week in the absence of disease progression or unacceptable toxicity
Experimental: Minnesota Standard Risk higher dose
Patients receive higher dose ruxolitinib PO BID for 56 days then begin taper PO BID for 1 week, followed by PO QD for 1 week in the absence of disease progression or unacceptable toxicity.
Experimental: Minnesota High Risk
Patients receive higher dose ruxolitinib PO BID for 56 days then begin taper PO BID for 1 week, followed by PO QD for 1 week in the absence of disease progression or unacceptable toxicity. Patients also receive systemic corticosteroid (methylprednisolone or similar) for a minimum of 3 days, then taper dose every 3-5 days in the absence of disease progression or unacceptable toxicity.
Sponsors
Leads: John Levine
Collaborators: Incyte Corporation, National Cancer Institute (NCI)

This content was sourced from clinicaltrials.gov