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Active, not recruiting Phase 2

A Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease

NCT06388564 · tracked via the Priya Life Science UK tracker
Phase
Phase 2
Started
2024-10-11
Last updated
2026-10-15

Condition(s) studied

Chronic Graft-versus-host-disease

Investigational drug(s) / intervention(s)

Axatilimab →Ruxolitinib →Corticosteroids →

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)

FacilityCityRegionStatus
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.

Official registry record

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.

View NCT06388564 on ClinicalTrials.gov ↗ ← All trials in the UK