A Study to Evaluate Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease

Recruiting now · Phase 3 · Has a placebo group

Conditions studied: Chronic Graft-versus-host-disease

In brief

This study will be conducted to compare the efficacy of axatilimab versus placebo in combination with corticosteroids as initial treatment for moderate or severe chronic graft-versus-host disease (cGVHD).

Key facts

Study ID

NCT06585774

Run by

Incyte Corporation

People needed

240

Starts

2025-01-21

Expected to finish

2030-03-31

Last updated by the study team

2026-08-06

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 allo-HCT from any donor HLA type (related or unrelated donor with any degree of HLA matching) using any graft source (bone marrow, peripheral blood stem cells, or cord blood). Recipients of myeloablative, nonmyeloablative, or reduced-intensity conditioning are eligible.
• Adequate hematologic function with ANC ≥ 0.5 × 109/L independent of growth factors for at least 7 days prior to study entry.
• Willingness to avoid pregnancy or fathering children.

You may not qualify if…

• Received more than 1 prior allo-HCT. Prior autologous HCT is allowed.
• Has overlap cGVHD, defined as simultaneous presence of features or characteristics of aGVHD in a patient with cGVHD.
• Received more than 7 days of systemic corticosteroid treatment for cGVHD or unable to begin a prednisone dose ≥ 1.0 mg/kg per day (or methylprednisolone equivalent) for cGVHD.
• Received previous systemic treatment for cGVHD, including extracorporeal photopheresis.
• Systemic treatment with CNIs or mTOR inhibitors started within 2 weeks prior to C1D1.
• Prior treatment with CSF-1R targeted therapies.
• Active, uncontrolled bacterial, fungal, parasitic, or viral infection.
• Evidence of relapse of the primary hematologic disease or treatment for relapse after the allo-HCT was performed, including DLIs for the treatment of molecular relapse.
• History of acute or chronic pancreatitis.
• Active symptomatic myositis.
• History or current diagnosis of cardiac disease indicating significant risk of safety for participation in the study, such as uncontrolled or significant cardiac disease.
• 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 endstage renal disease on dialysis.
• 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.
• Pregnant or breastfeeding.
• Other protocol-defined Inclusion/Exclusion Criteria may apply.

Where it is running

• University of Arizona Cancer Center-Out Pt. — Tucson, Arizona, United States (enrolling)
• City of Hope Medical Center — Duarte, California, United States (enrolling)
• University of California San Diego Medical Center, Moores Cancer Center — La Jolla, California, United States (enrolling)
• University of Southern California — Los Angeles, California, United States (enrolling)
• UCSF — San Francisco, California, United States (enrolling)
• Colorado Blood Cancer Institute — Denver, Colorado, United States (enrolling)
• Childrens National Hospital — Washington D.C., District of Columbia, United States (enrolling)
• Miami Cancer Institute — Miami, Florida, United States (enrolling)
• Orlando Health Cancer Institute Downtown Orlando — Orlando, Florida, United States (enrolling)
• Memorial Cancer Institute — Pembroke Pines, Florida, United States (enrolling)
• Emory University-Winship Cancer Institute — Atlanta, Georgia, United States (enrolling)
• Northwestern Memorial Hospital — Chicago, Illinois, United States (enrolling)
• University of Illinois — Chicago, Illinois, United States (enrolling)
• The University of Kansas Cancer Center — Kansas City, Kansas, United States (enrolling)
• John Hopkins Hospital — Baltimore, Maryland, United States (enrolling)
• Massachusetts General Hospital — Boston, Massachusetts, United States (enrolling)
• Dana Farber Cancer Institute — Boston, Massachusetts, United States (enrolling)
• University of Michigan — Ann Arbor, Michigan, United States (enrolling)
• Henry Ford Hospital — Detroit, Michigan, United States (enrolling)
• Corewell Health Hematology Oncology — Grand Rapids, Michigan, United States (enrolling)
• Hackensack University Medical Center — Hackensack, New Jersey, United States (enrolling)
• Rutgers Cancer Institute of Nj — New Brunswick, New Jersey, United States (enrolling)
• University of Rochester Medical Center — Rochester, New York, United States (enrolling)
• Stony Brook University Medical Center — Stony Brook, New York, United States (enrolling)
• University of Alabama Birmingham — Birmingham, Alabama, United States (enrolling)

Full record on ClinicalTrials.gov

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