Biomarker-Guided Ruxolitinib for Preventing Chronic GVHD
This study is testing if a drug called ruxolitinib can help prevent chronic graft versus host disease (cGVHD) in people who have had an allogeneic hematopoietic cell transplant (HCT). HCT is a treatment for certain blood cancers, but it can lead to cGVHD, where donor cells attack your body. Symptoms of cGVHD can include skin rash, dry mouth, or dry eyes. This study will use biomarker analysis to guide treatment. Researchers want to see how safe ruxolitinib is and how well it prevents moderate-to-severe cGVHD up to one year after starting the drug. The study plans to enroll 42 participants aged 18 and older.
- Study design
- This is an interventional study with a planned enrollment of 42 participants. It includes a safety lead-in segment and an expansion cohort to evaluate the drug's effectiveness.
- What's involved
- You would receive standard of care treatment, undergo GHVD biomarker analysis, provide blood samples, and complete questionnaires. The safety lead-in segment measures toxicity for one 28-day cycle, and the expansion cohort assesses cGVHD up to one year.
- Compensation
- Not stated in the trial record.
- Follow-up
- The study measures chronic GVHD up to 1 year after the first dose of ruxolitinib and moderate-to-severe cGVHD-free survival up to 12 months.
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NCT07025538
Biomarker-Guided Ruxolitinib for the Prevention of Chronic Graft Versus Host Disease After Allogeneic Hematopoietic Cell Transplantation
Recruiting
PHASE1Ages 18+InterventionalPreventionCity of Hope Medical CenterInvestigator-initiated
~42 participants
Updated 2026-07-13 on ClinicalTrials.gov
What's tested:Best PracticeBiomarker AnalysisBiospecimen CollectionQuestionnaire AdministrationRuxolitinib
At a glance
Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence of unacceptable toxicity (UT) (Safety lead-in segment)
Measured over From first dose of ruxolitinib to end of first cycle (Cycle length = 28 days)
+4 more outcomes measured
Conditions
Where it's being run
1 sites across 1 statesCalifornia1
Study leadership
- Amandeep Salhotra · PRINCIPAL_INVESTIGATOR · City of Hope Medical Center
Who to contact
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Do you actually qualify for this trial?
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Eligibility criteria
Inclusion
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 as graft source. Note: Patients receiving manipulated graft are not included
PRE-SCREENING: Morphologic remission per day +30 bone marrow
PRE-SCREENING: Any conditioning regimen (myeloablative, reduce intensity/non-myeloablative conditioning) is allowed
PRE-SCREENING: Any GVHD prophylaxis (tacrolimus-sirolimus, tacrolimus-methotrexate, or post-transplant cyclophosphamide) is allowed
PRE-SCREENING: Life expectancy of more than 6 months
PRE-SCREENING: Absolute neutrophil count (ANC) \> 1000/mm\^3 (to be performed between day +70 and +100 after HCT unless otherwise stated)
PRE-SCREENING: Hemoglobin ≥ 8.0 gm/dL (to be performed between day +70 and +100 after HCT unless otherwise stated)
PRE-SCREENING: Platelets ≥ 50,000/mm\^3 (to be performed between day +70 and +100 after HCT unless otherwise stated)
Note: Patients with lower counts can enroll if infection cytomegalovirus (CMV)/human herpesvirus 6 (HHV6), etc. is being treated actively
PRE-SCREENING: Total bilirubin ≤ 1.5 X upper limit of normal (ULN) (unless has Gilbert's disease) (to be performed between day +70 and +100 after HCT unless otherwise stated)
PRE-SCREENING: Aspartate aminotransferase (AST) =\< 3.0 x ULN (to be performed between day +70 and +100 after HCT unless otherwise stated)
PRE-SCREENING: Alanine aminotransferase (ALT) =\< 3.0 x ULN (to be performed between day +70 and +100 after HCT unless otherwise stated)
PRE-SCREENING: Glomerular filtration rate (GFR) ≥ 50 ml/min (to be performed between day +70 and +100 after HCT unless otherwise stated)
PRE-SCREENING: Women of childbearing potential (WOCBP): negative urine or serum pregnancy test (to be performed between day +70 and +100 after HCT unless otherwise stated)
If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required
PRE-SCREENING: Agreement by females and males of childbearing potential to use an effective method of birth control or abstain from heterosexual activity for the course of the study through at least 3 months after the last dose of protocol therapy
Childbearing potential defined as not being surgically sterilized (men and women) or have not been free from menses for \> 1 year (women only)
AFTER DAY +100 TEST RESULTS PATIENTS WITH MODERATE TO HIGH-RISK cGVHD (TREATMENT ARM): Documented informed consent of the participant and/or legally authorized representative
Assent, when appropriate, will be obtained per institutional guidelines
AFTER DAY +100 TEST RESULTS PATIENTS WITH MODERATE TO HIGH-RISK cGVHD (TREATMENT ARM): Elevated serum/plasma levels of ST2, CXCL9, MMP-3, and OPN as indicated by moderate or severe risk of chronic GVHD in the test results
AFTER DAY +100 TEST RESULTS PATIENTS WITH MODERATE TO HIGH-RISK cGVHD (TREATMENT ARM): No use of ruxolitinib or other Jak inhibitors in the past 14 days
AFTER DAY +100 TEST RESULTS PATIENTS WITH MODERATE TO HIGH-RISK cGVHD (TREATMENT ARM): Morphologic remission per day +100 bone marrow
AFTER DAY +100 TEST RESULTS PATIENTS WITH MODERATE TO HIGH-RISK cGVHD (TREATMENT ARM): Adequate hematopoietic recovery (hemoglobin \[Hgb\] ≥ 8 g/dL, platelets \[PLT\] ≥ 50K/ mm\^3)
AFTER DAY +100 TEST RESULTS PATIENTS WITH MODERATE TO HIGH-RISK cGVHD (TREATMENT ARM): Negative serum or urine pregnancy test (female participants with childbearing potential only)
AFTER DAY +100 TEST RESULTS PATIENTS WITH MODERATE TO HIGH-RISK cGVHD (TREATMENT ARM): Absence of active infection not responding to antibiotics
AFTER DAY +100 TEST RESULTS PATIENTS WITH MODERATE TO HIGH-RISK cGVHD (TREATMENT ARM): Absence of progressive acute GVHD. Note: prednisone administration (flat dose of \< 0.25 mg/kg) is allowed. Patients receiving any other medication to control active/progressive GVHD will be excluded
AFTER DAY +100 TEST RESULTS PATIENTS WITH MODERATE TO HIGH-RISK cGVHD (TREATMENT ARM): Absence of any clinically significant uncontrolled sickness
AFTER DAY +100 TEST RESULTS PATIENTS WITH LOW-RISK cGVHD (CONTROL ARM): Documented informed consent of the participant and/or legally authorized representative
Assent, when appropriate, will be obtained per institutional guidelines
AFTER DAY +100 TEST RESULTS PATIENTS WITH LOW-RISK cGVHD (CONTROL ARM): Low serum/plasma levels of ST2, CXCL9, MMP-3, and OPN as indicated by low risk of chronic GVHD in the test results
AFTER DAY +100 TEST RESULTS PATIENTS WITH LOW-RISK cGVHD (CONTROL ARM): No use of ruxolitinib or other Jak inhibitors in the past 14 days
AFTER DAY +100 TEST RESULTS PATIENTS WITH LOW-RISK cGVHD (CONTROL ARM): Morphologic remission per day +100 bone marrow
AFTER DAY +100 TEST RESULTS PATIENTS WITH LOW-RISK cGVHD (CONTROL ARM): Adequate hematopoietic recovery (Hgb ≥ 8 g/dL, PLT ≥ 50K/ mm\^3)
AFTER DAY +100 TEST RESULTS PATIENTS WITH LOW-RISK cGVHD (CONTROL ARM): Negative serum or urine pregnancy test (female participants with childbearing potential only)
AFTER DAY +100 TEST RESULTS PATIENTS WITH LOW-RISK cGVHD (CONTROL ARM): Absence of active infection not responding to antibiotics
AFTER DAY +100 TEST RESULTS PATIENTS WITH LOW-RISK cGVHD (CONTROL ARM): Absence of progressive acute GVHD. Note: prednisone administration (flat dose of \< 0.25 mg/kg) is allowed. Patients receiving any other medication to control active/progressive GVHD will be excluded
AFTER DAY +100 TEST RESULTS PATIENTS WITH LOW-RISK cGVHD (CONTROL ARM): Absence of any clinically significant uncontrolled sickness
Exclusion
PRE-SCREENING: Prior chemotherapy \< 14 days prior to study biospecimen collection on day +100 post-HCT
PRE-SCREENING: Previous use of ruxolitinib or other JAK-inhibitors is allowed but administration should be stopped for at least 14 days prior to enrollment. Note: Previous use of Jak inhibitors before enrollment (including the pre-HCT period) should be recorded
PRE-SCREENING: History of allergic reactions attributed to compounds of similar chemical or biologic composition to study agent
PRE-SCREENING: Active/progressive acute GVHD at the time of screening. Prednisone administration (flat dose of \< 0.25 mg/kg) is allowed. Patients receiving any other medication to control active/progressive GVHD will be excluded
PRE-SCREENING: Patients with history of major adverse cardiovascular event (MACE)/other thrombosis (myocardial infarction \[MI\]/stroke and pulmonary embolism \[PE\]/deep vein thrombosis \[DVT\]) in the past 6 months
PRE-SCREENING: Patients with a history of tuberculosis
PRE-SCREENING: Clinically significant uncontrolled illness
PRE-SCREENING: Active infection not responding to antibiotics
PRE-SCREENING: Other active malignancy. Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial
PRE-SCREENING: Females only: Pregnant or breastfeeding
PRE-SCREENING: Any other condition that would, in the investigator's judgment, contraindicate the patient's participation in the clinical study due to safety concerns with clinical study procedures
PRE-SCREENING: Prospective participants who, in the opinion of the investigator, may not be able to comply with all study procedures (including compliance issues related to feasibility/logistics)
What this trial measures
- Incidence of unacceptable toxicity (UT) (Safety lead-in segment)From first dose of ruxolitinib to end of first cycle (Cycle length = 28 days)
UT in a given patient will be defined as any of the following that are assigned an attribution level of at least possibly related to ruxolitinib administration. 1) Any grade 3 or higher non-hematological adverse events, per National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version (v) 5.0 toxicity criteria that last more than 5 days. 2) Prolonged myelosuppression, defined as ≥ grade 4 neutropenia or thrombocytopenia that persists for more than 7 days. 3) Hy's law cases. 4) Any other regimen-related cause of death. 5) Permanent discontinuation or dose reduction of ruxolitinib due to any drug-related toxicity (regardless of grade). In addition, septic UT is defined as: any grade 5 sepsis-related toxicity that is assigned an attribution level of at least possibly related to the addition of ruxolitinib to the conditioning regimen.
- Chronic graft versus host disease (cGVHD) (Expansion cohort)Up to 1 year post first dose of ruxolitinib
Will be evaluated and scored according to National Institutes of Health (NIH) Consensus Staging.
- Moderate-to-severe cGVHD free survival (GFS)From date of starting first dose of ruxolitinib to first occurrence of moderate-to-severe cGVHD or death, whichever occurs first, assessed up to 12 months post first dose of ruxolitinib
GFS will be censored at the last follow-up if patients are alive and remain free of moderate-to-severe cGVHD. GFS will be assessed in both the safety lead-in segment and expansion cohort. Will be analyzed using the Kaplan-Meier curves.
- Patients completing at least 80% of planned ruxolitinib (Feasibility) (Expansion cohort)Up to 1 cycle of ruxolitinib (Cycle length = 28 days)
Patients who take at least one dose of ruxolitinib will be evaluable for feasibility. The point estimate and exact 95% confidence intervals (CIs) will be provided.
- Tolerability of ruxolitinib (Expansion cohort)Up to 12 cycles (Cycle length = 28 days)
Patients who have ruxolitinib dose interruption, reduction, or early stopping due to adverse events attributable to ruxolitinib are deemed to be intolerable to ruxolitinib.