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).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
240
Event Free Survival (EFS)
Defined from the date of randomization to the date of any predefined event, whichever occurs first.
Time frame: Up to 3 years
Objective Response (OR)
Defined for each treatment group as CR or PR at 6 months Cycle 7 (28-day cycles), Day 1, in the absence of new systemic therapy for cGVHD. Responses defined by the 2014 NIH consensus criteria.
Time frame: 6 months
Event Free Survival 2
Defined from the date of randomization to the date of any predefined event, whichever occurs first.
Time frame: Up to 3 years
Proportion of participants with a ≥ 7-point improvement in mLSS total score
Time frame: Up to 3 years
Overall Response
Defined as CR or PR at 12 months in the absence of new systemic therapy for cGVHD.
Time frame: 12 Months
DOR (in responders only)
Defined as the time from the date of first response (PR or CR) to the date of progression of cGVHD from nadir in any organ, start of new systemic treatment for cGVHD, or death from any cause, whichever comes first.
Time frame: Up to 3 years
Best Overall Response (BOR)
Defined as the best response of CR or PR at any timepoint up to the initiation of new therapy for cGVHD.
Time frame: Up to 3 years
Overall Survival (OS)
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University of Alabama Birmingham
Birmingham, Alabama, United States
RECRUITINGUniversity of Arizona Cancer Center-Out Pt.
Tucson, Arizona, United States
RECRUITINGCity of Hope Medical Center
Duarte, California, United States
RECRUITINGUniversity of California San Diego Medical Center, Moores Cancer Center
La Jolla, California, United States
RECRUITINGUniversity of Southern California
Los Angeles, California, United States
RECRUITINGUCSF
San Francisco, California, United States
RECRUITINGColorado Blood Cancer Institute
Denver, Colorado, United States
RECRUITINGChildrens National Hospital
Washington D.C., District of Columbia, United States
RECRUITINGMiami Cancer Institute
Miami, Florida, United States
RECRUITINGOrlando Health Cancer Institute Downtown Orlando
Orlando, Florida, United States
RECRUITING...and 118 more locations
Defined as the time from the date of randomization to the date of death due to any cause.
Time frame: Up to 3 years
Nonrelapse mortality (NRM)
Defined as the time from the date of randomization to the date of death in the absence of primary hematologic disease relapse.
Time frame: Up to 3 years
Failure-free survival (FFS)
Defined as the time from the date of randomization to the date of addition or initiation of another systemic therapy for cGVHD, relapse of underlying disease, or death due to any cause.
Time frame: Up to 3 years
Relapse of hematologic diseases
Defined as the reappearance of symptoms of underlying disease.
Time frame: Up to 3 years
Time to primary hematologic disease relapse
Defined as the time from the date of randomization to the date of relapse.
Time frame: Up to 3 years
Percent reduction in daily corticosteroid dose
Time frame: 6 months
Proportion of participants who tapered off all corticosteroids
Time frame: 6 months
Number of participants with Treatment-emergent Adverse Events (TEAEs)
Defined as adverse events reported for the first time or worsening of a pre-existing event after the first dose of study treatment.
Time frame: Up to 3 years and 30 days
Change from baseline in circulating monocyte number and phenotype (CD14/16)
Time frame: Up to 3 years and 30 days
Change from baseline in soluble markers for bone resorption and formation, including bone-specific alkaline phosphatase (BAP) and C-terminal telopeptide (CTX)
Time frame: Up to 3 years and 30 days