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RECRUITINGPhase 3INTERVENTIONAL

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

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease (AXemplify-357)

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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).

Who May Be Eligible (Plain English)

Who May Qualify: - ≥ 12 years of age at the time of willing to sign a consent form. - 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 white blood cell count (ANC) at least 0.5 × 109/L independent of growth factors for at least 7 days prior to study entry. - Willingness to avoid pregnancy or fathering children. Who Should NOT Join This Trial: - 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. ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
Inclusion Criteria: * ≥ 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. Exclusion Criteria: * 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.

Treatments Being Tested

DRUG

INCA034176

IV infusion

DRUG

Placebo

IV infusion

DRUG

Corticosteroids

Oral/IV Infusion

Locations (20)

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