A Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease

Running, not enrolling · Phase 2

Conditions studied: Chronic Graft-versus-host-disease

In brief

This study will be conducted to determine the preliminary efficacy of axatilimab in combination with ruxolitinib and to assess the contribution of axatilimab to the combination treatment effect in participants with cGVHD.

Key facts

Study ID

NCT06388564

Run by

Incyte Corporation

People needed

130

Starts

2024-10-11

Expected to finish

2029-12-01

Last updated by the study team

2026-05-22

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• ≥ 12 years of age at the time of informed consent.
• New-onset moderate or severe cGVHD, as defined by the 2014 NIH Consensus Development Project Criteria for Clinical Trials in cGVHD, requiring systemic therapy.
• History of 1 allo-SCT (any type of stem cell donor, any conditioning regimen, and source of hematopoietic stem cells).
• Adequate hematologic function independent of platelet transfusion and growth factors for at least 7 days prior to study entry: ANC ≥ 0.75 × 109/L and platelet count ≥ 20 × 109/L.
• Willingness to avoid pregnancy or fathering children.

You may not qualify if…

• Received more than 1 prior allo-SCT. Prior autologous HCT is allowed.
• Has overlap cGVHD, defined as simultaneous presence of features or characteristics of aGVHD in a patient with cGVHD.
• Received previous systemic treatment for cGVHD, including systemic corticosteroids and extracorporeal photopheresis.
• Received systemic corticosteroids within 2 weeks prior to C1D1, regardless of indication.
• Initiated systemic treatment with CNIs or mTOR inhibitors within 2 weeks prior to C1D1.
• Prior treatment with a JAK inhibitor within 8 weeks before randomization. Participants who received a JAK inhibitor for the treatment of aGVHD are eligible only if they achieved a response (CR or PR) to JAK inhibitor treatment and did not discontinue due to toxicity.
• Evidence of relapse of the primary hematologic disease or treatment for relapse after the allo-SCT was performed, including DLIs for the treatment of molecular relapse.
• History of acute or chronic pancreatitis.
• History of thromboembolic events (such as deep vein thrombosis, pulmonary embolism, stroke, myocardial infarction) in the 6 months prior to study entry.
• Active symptomatic myositis.
• Severe renal impairment, that is, estimated CrCl < 30 mL/min measured or calculated by Cockcroft-Gault equation in adults and Schwartz formula in pediatric participants, or end-stage renal disease on dialysis. Participants with CrCl of 30 to 59 mL/min on treatment with fluconazole are not eligible.
• Impaired liver function, defined as total bilirubin > 1.5 × ULN and/or ALT and AST > 3 × ULN in participants with no evidence of liver cGVHD.
• Currently active significant cardiac disease, such as uncontrolled arrhythmias, uncontrolled hypertension, or Class 3 or 4 congestive heart failure as defined by New York Heart Association, or a history of myocardial infarction or unstable angina within 6 months prior to randomization.
• Pregnant or breastfeeding.
• Other protocol-defined Inclusion/Exclusion Criteria may apply.

Where it is running

• City of Hope Medical Center — Duarte, California, United States
• University of California-Los Angeles Medl Cntr-Oncology Center Bowyer Clinic — Los Angeles, California, United States
• Stanford Cancer Center — Stanford, California, United States
• University of Colorado Cancer Center — Aurora, Colorado, United States
• Colorado Blood Cancer Institute — Denver, Colorado, United States
• Smilow Cancer Center-Yale — New Haven, Connecticut, United States
• Mayo Clinic Jacksonville — Jacksonville, Florida, United States
• Northwestern University — Chicago, Illinois, United States
• The University of Chicago Medicine — Chicago, Illinois, United States
• University of Maryland-Greenebaum Cancer Center — Baltimore, Maryland, United States
• Washington University — St Louis, Missouri, United States
• Fred and Pamela Buffett Cancer Center — Omaha, Nebraska, United States
• Memorial Sloan Kettering Cancer Center — New York, New York, United States
• Weill Cornell Medicine — New York, New York, United States
• Mount Sinai Hospital — New York, New York, United States
• Cleveland Clinic — Cleveland, Ohio, United States
• The Ohio State University — Columbus, Ohio, United States
• University of Pennsylvania Abramson Cancer Center — Philadelphia, Pennsylvania, United States
• Hillman Cancer Center — Pittsburgh, Pennsylvania, United States
• Vanderbilt University Medical Center — Nashville, Tennessee, United States
• Md Anderson Cancer Center — Houston, Texas, United States
• Fred Hutchinson Cancer Center — Seattle, Washington, United States
• University of Washington — Seattle, Washington, United States
• Froedtert & the Medical College of Wisconsin — Milwaukee, Wisconsin, United States
• Mayo Clinic Hospital — Phoenix, Arizona, United States

Full record on ClinicalTrials.gov

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