Recruiting
Phase 1

Ruxolitinib

Sponsor:

City of Hope Medical Center

Code:

NCT07025538

Conditions

Hematopoietic and Lymphatic System Neoplasm

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Best Practice

Biomarker Analysis

Biospecimen Collection

Questionnaire Administration

Ruxolitinib

Study Details

Brief 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.

Conditions

Hematopoietic and Lymphatic System Neoplasm

Study ID

NCT07025538

Start date

Apr 29, 2026

Status verified date

Jul, 2026

Completion date

Jun 22, 2028

Anticipated

Primary completion date

Jun 22, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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 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 Criteria:

  • 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)

Study Design

Enrollment

42 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Prevention

Interventions and Outcome Measures

Arms

experimental: Arm I (ruxolitinib)

Starting between days +105 and +130 post-HCT, patients receive ruxolitinib PO BID on days 1-28 of each cycle. Cycles repeat every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Patients also undergo additional blood sample collection throughout the trial.

active comparator: Arm II (SOC treatment)

Starting between days +105 and +130 post-HCT, patients receive SOC treatment for up to 1 year in the absence of disease progression or unacceptably toxicity. Patients also undergo additional blood sample collection throughout the trial.

Interventions

Best Practice

Receive SOC treatment

Biomarker Analysis

Undergo GHVD biomarker analysis

Biospecimen Collection

Undergo blood sample collection

Questionnaire Administration

Ancillary studies

Ruxolitinib

Given PO

Primary outcome measure

  • Incidence of unacceptable toxicity (UT) (Safety lead-in segment) [ Time Frame: From first dose of ruxolitinib to end of first cycle (Cycle length = 28 days) ]
  • Chronic graft versus host disease (cGVHD) (Expansion cohort) [ Time Frame: Up to 1 year post first dose of ruxolitinib ]
  • Moderate-to-severe cGVHD free survival (GFS) [ Time Frame: 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 ]
  • Patients completing at least 80% of planned ruxolitinib (Feasibility) (Expansion cohort) [ Time Frame: Up to 1 cycle of ruxolitinib (Cycle length = 28 days) ]
  • Tolerability of ruxolitinib (Expansion cohort) [ Time Frame: Up to 12 cycles (Cycle length = 28 days) ]

Central Contacts and Locations

Locations

City of Hope Medical Center

Recruiting

Duarte, California, United States, 91010

Contacts

Amandeep Salhotra

626-218-2405asalhotra@coh.org

Principal Investigator:

Amandeep Salhotra

More Information

Sponsor

City of Hope Medical Center

Last update posted

Jul 13, 2026

Last verified

Jul, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by City of Hope Medical Center on 2026-07-13.