This study will be conducted to determine the clinical efficacy of axatilimab in Japanese participants with chronic graft-versus-host disease (cGVHD).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
21
IV infusion
Fujita Health University Hospital
Aichi, Japan
University of Fukui Hospital
Fukui, Japan
Overall Response Rate in the First 6 Cycles
The overall response rate will be assessed by the number of participants with objective response by Cycle 7 (28-day cycles), Day 1, with responses defined by the 2014 NIH consensus criteria.
Time frame: Up to Cycle 7 (Day 169)
Proportion of participants with a ≥ 7-point improvement in modified Lee symptom scale (mLSS) score
Time frame: Up to 2 years
Overall Response Rate
Defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD.
Time frame: Up to 2 years
Duration of Response
Defined as the time from initial partial response or complete response until documented progression of cGVHD, start of new therapy, or death for any reason.
Time frame: Up to 2 years
Organ-specific Response Rate
Organ-specific response is defined as the number of participants with objective response for the nine individual organs based on 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD (skin, eyes, mouth, esophagus, upper gastrointestinal \[GI\], lower GI, liver, lungs and joints and fascia).
Time frame: Up to 2 years
Percent reduction in average daily dose (or equivalent) of corticosteroids
Time frame: Up to 2 years
Proportion of participants who discontinue corticosteroid use
Time frame: Up to 2 years
Number of participants with Treatment-emergent Adverse Events (TEAEs)
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Kyushu University Hospital
Fukuoka, Japan
Hiroshima University Hospital
Hiroshima, Japan
Hokkaido University Hospital
Hokkaido, Japan
Kobe City Medical Center General Hospital
Hyōgo, Japan
Tokai University Hospital
Kanagawa, Japan
National Hospital Organization Kumamoto Medical Center
Kumamoto, Japan
Tohoku University Hospital
Miyagi, Japan
Japanese Red Cross Aichi Medical Center Nagoya Daiichi Hospital
Nagoya, Japan
...and 6 more locations
Defined as adverse events reported for the first time or worsening of a pre-existing event from the time the participant signs the informed consent form (ICF) until at least 30 days after the end of trial (EOT) or until start of new cGVHD therapy.
Time frame: Up to 2 years and 30 days
Change from baseline in Karnofsky/Lansky performance status
Time frame: Up to 2 years and 30 days
Axatilimab pharmacokinetic (PK) in Plasma
Axatilimab concentration in plasma.
Time frame: Up to 2 years and 30 days
Number of Participants with Anti-Drug Antibody (ADA)
Time frame: Up to 2 years and 30 days