Biomarker-Guided Feasibility/Efficacy Trial of Ruxolitinib in Patients With High-Risk of Chronic Graft-Versus-Host Disease Development After Allogeneic Hematopoietic Cell Transplantation
City of Hope Medical Center
Summary
This phase I trial studies how well biomarker-guided ruxolitinib works for the prevention of chronic graft versus host disease (GVHD) in patients that have undergone allogeneic hematopoietic cell transplant (HCT). Allogeneic HCT is the most effective therapy for patients with high-risk blood and bone marrow malignancies. GVHD is a disease caused when cells from a donated stem cell graft attack the normal tissue of the transplant patient. Symptoms include jaundice, skin rash or blisters, a dry mouth, or dry eyes. In chronic GVHD (cGVHD), symptoms occur more than three months after transplantation. Despite significant advances in how allogeneic HCTs are conducted, cGHVD remains a major limitation to the long-term success of the transplant and can impact patients' quality of life post-transplant. Checking GVHD biomarkers in patients' blood after allogeneic HCT may help doctors predict how likely the patient is to develop cGVHD. This information can be used to help guide patients with high levels to receive cGVHD preventative therapy with ruxolitinib. Ruxolitinib works by blocking some of the enzymes that are needed for the development of cGVHD, which may be an effective way to prevent cGVHD in patients with high levels of GVHD biomarkers.
Description
PRIMARY OBJECTIVES: I. To assess the safety and feasibility of ruxolitinib administration in patients with elevated levels of chronic GVHD biomarkers at day +100 after allogeneic hematopoietic cell transplantation (HCT). (Safety lead-in) II. Evaluate the efficacy of ruxolitinib in preventing chronic GVHD by assessing 1-year moderate-to-severe cGVHD-free survival (CGFS) defined as first occurrence of moderate to severe chronic GVHD or death, whichever occurs first, after the first dose of ruxolitinib. (Expansion cohort) SECONDARY OBJECTIVES: I. Assess the overall survival (OS) at 1 and 2 yea…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * PRE-SCREENING: Documented informed consent of the participant and/or legally authorized representative * Assent, when appropriate, will be obtained per institutional guidelines * PRE-SCREENING: Agreement to allow the use of archival tissue from diagnostic tumor biopsies * If unavailable, exceptions may be granted with study principal investigator (PI) approval * PRE-SCREENING: Age: ≥ 18 years * PRE-SCREENING: Karnofsky performance status ≥ 80 * PRE-SCREENING: Patients must have undergone allogeneic hematopoietic cell transplantation with peripheral blood stem cells…
Interventions
- OtherBest Practice
Receive SOC treatment
- OtherBiomarker Analysis
Undergo GHVD biomarker analysis
- ProcedureBiospecimen Collection
Undergo blood sample collection
- OtherQuestionnaire Administration
Ancillary studies
- DrugRuxolitinib
Given PO
Location
- City of Hope Medical CenterDuarte, California