A Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease
Axatilimab: Axatilimab will be administered at protocol defined dose.
Ruxolitinib: Ruxolitinib will be administered at protocol defined dose.
Corticosteroids: Corticosteroids will be administered at protocol defined dose.
Study summary
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.
Eligibility
Sex
ALL
Min age
12 Years
Max age
—
Healthy volunteers
No
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 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.
Exclusion Criteria:
* 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.
Primary outcome measure(s)
Objective Response Rate — 6 months Defined as Complete Response (CR) or Partial Response (PR) at 6 months in the absence of new systemic therapy for cGVHD. Response assessment will be based on the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD.
Trial sites (68)
Facility
City
Region
Status
Mayo Clinic Hospital
Phoenix
Arizona
City of Hope Medical Center
Duarte
California
University of California-Los Angeles Medl Cntr-Oncology Center Bowyer Clinic
Los Angeles
California
Stanford Cancer Center
Stanford
California
University of Colorado Cancer Center
Aurora
Colorado
Colorado Blood Cancer Institute
Denver
Colorado
Smilow Cancer Center-Yale
New Haven
Connecticut
Mayo Clinic Jacksonville
Jacksonville
Florida
Northwestern University
Chicago
Illinois
The University of Chicago Medicine
Chicago
Illinois
University of Maryland-Greenebaum Cancer Center
Baltimore
Maryland
Washington University
St Louis
Missouri
Fred and Pamela Buffett Cancer Center
Omaha
Nebraska
Memorial Sloan Kettering Cancer Center
New York
New York
Weill Cornell Medicine
New York
New York
Mount Sinai Hospital
New York
New York
Cleveland Clinic
Cleveland
Ohio
The Ohio State University
Columbus
Ohio
University of Pennsylvania Abramson Cancer Center
Philadelphia
Pennsylvania
Hillman Cancer Center
Pittsburgh
Pennsylvania
Vanderbilt University Medical Center
Nashville
Tennessee
Md Anderson Cancer Center
Houston
Texas
Fred Hutchinson Cancer Center
Seattle
Washington
University of Washington
Seattle
Washington
Froedtert & the Medical College of Wisconsin
Milwaukee
Wisconsin
Az Sint-Jan Brugge - Oostende Av - Campus Sint-Jan
Bruges
Belgium
Universitair Ziekenhuis Antwerpen (Uza)
Edegem
Belgium
Jessa Ziekenhuis
Hasselt
Belgium
Universitair Ziekenhuis (Uz) Leuven
Leuven
Belgium
Universitaire Ziekenhuis Leuven - Gasthuisberg
Leuven
Belgium
Centre Hospitalier Universitaire (Chu) de Liege
Liège
Belgium
AZ DELTA
Roeselare
Belgium
Arthur J E Child Comprehensive Cancer Centre
Calgary
Alberta
Princess Margaret Cancer Centre - University Health Network
Toronto
Ontario
Chu Sainte-Justine
Montreal
Quebec
Vancouver General Hospital
Vancouver
Canada
Klinikum Der Johann Wolfgang Goethe University
Frankfurt am Main
Germany
Universitatklinikum Freiburg
Freiburg I. Breisgau
Germany
Universitatsklinikum Hamburg Eppendorf
Hamburg
Germany
Universitaetsklinikum Jena
Jena
Germany
+ 28 more sites — see the full list on the official registry below.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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