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

May 20, 2026 updated by: Incyte Corporation

A Phase 2, Open-Label, Randomized, Multicenter Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
130

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Expanded Access

Expanded Access

A way for patients with serious diseases or conditions who cannot participate in a clinical trial to gain access to a medical product that has not been approved by the U.S. Food and Drug Administration (FDA). Also called compassionate use. There are different expanded access types.
Available

Available

  • Available: Expanded access is currently available for this investigational treatment, and patients who are not participants in the clinical study may be able to gain access to the drug, biologic, or medical device being studied.
  • No longer available: Expanded access was available for this intervention previously but is not currently available and will not be available in the future.
  • Temporarily not available: Expanded access is not currently available for this intervention but is expected to be available in the future.
  • Approved for marketing: The intervention has been approved by the U.S. Food and Drug Administration for use by the public.
 outside the clinical trial. See expanded access record.

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.
  • Name: Incyte Corporation Call Center (US)
  • Phone Number: 1.855.463.3463
  • Email: medinfo@incyte.com

Study Contact Backup

  • Name: Incyte Corporation Call Center (ex-US)
  • Phone Number: +800 00027423
  • Email: eumedinfo@incyte.com

Study Locations

  • Belgium
      • Bruges, Belgium, 08000
        • Az Sint-Jan Brugge - Oostende Av - Campus Sint-Jan
      • Edegem, Belgium, 02650
        • Universitair Ziekenhuis Antwerpen (UZA)
      • Hasselt, Belgium, 03500
        • Jessa ziekenhuis
      • Leuven, Belgium, 03000
        • Universitair Ziekenhuis (Uz) Leuven
      • Leuven, Belgium, 03000
        • Universitaire Ziekenhuis Leuven - Gasthuisberg
      • Liège, Belgium, 04000
        • Centre Hospitalier Universitaire (CHU) de Liege
      • Roeselare, Belgium, 08800
        • AZ Delta
  • Canada
      • Vancouver, Canada, V5Z1M9
        • Vancouver General Hospital
    • Alberta
      • Calgary, Alberta, Canada, T3N 4N1
        • Arthur J E Child Comprehensive Cancer Centre
    • Ontario
      • Toronto, Ontario, Canada, M5G 2M9
        • Princess Margaret Cancer Centre - University Health Network
    • Quebec
      • Montreal, Quebec, Canada, H3T 1C5
        • CHU Sainte-Justine
  • Germany
      • Frankfurt am Main, Germany, 60590
        • Klinikum Der Johann Wolfgang Goethe University
      • Freiburg I. Breisgau, Germany, 79106
        • Universitatklinikum Freiburg
      • Hamburg, Germany, 20251
        • Universitatsklinikum Hamburg Eppendorf
      • Jena, Germany, 07747
        • Universitaetsklinikum Jena
      • Mannheim, Germany, 68167
        • University Hospital Mannheim
      • Regensburg, Germany, 93053
        • Universitaetsklinikum Regensburg
  • Italy
      • Bergamo, Italy, 24127
        • Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII
      • Bologna, Italy, 40138
        • Aou Policlinico S. Orsola-Malpighi
      • Milan, Italy, 20122
        • Fondazione Irccs Ca Granda Ospedale Maggiore Policlinico Di Milano Uo Dermatologia
      • Rome, Italy, 00168
        • Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro Cuore
      • Rome, Italy, 00165
        • Ospedale Pediatrico Bambino Gesù IRCCS
      • Rozzano, Italy, 20089
        • IRCCS Istituto Clinico Humanitas
      • Torino, Italy, 10126
        • A.O.U. Città della Salute e Della Scienza di Torino
  • Spain
      • Barcelona, Spain, 08036
        • Hospital Clinic Barcelona Main
      • El Palmar, Spain, 30120
        • Hospital Universitario Virgen de la Arrixaca
      • Granada, Spain, 18014
        • Hospital Universitario Virgen de Las Nieves
      • Granvia de L'hospitalet 199-203, Spain, 08908
        • Ico Institut Catala D Oncologia
      • Madrid, Spain, 28034
        • Hospital Universitario Ramon y Cajal
      • Madrid, Spain, 28041
        • Hospital Universitario 12 de Octubre
      • Madrid, Spain, 28007
        • Hospital General Universitario Gregorio Marañon
      • Málaga, Spain, 29011
        • Hospital Regional Universitario de Málaga
      • Salamanca, Spain, 37007
        • Hospital Clínico Universitario de Salamanca
      • Santander, Spain, 39008
        • Hospital Universitario Marques de Valdecilla
      • Seville, Spain, 41013
        • Hospital Universitario Virgen del Rocio
      • Valencia, Spain, 46026
        • Hospital Universitari i Politecnic La Fe
      • Valencia, Spain, 46010
        • Hospital Clinico Universitario de Valencia
  • United Kingdom
      • Birmingham, United Kingdom, B15 2TH
        • Queen Elizabeth Hospital
      • Cambridge, United Kingdom, CB2 0QQ
        • Cambridge University Hospitals NHS Foundation Trust
      • Cardiff, United Kingdom, CF14 4XW
        • University Hospital of wales
      • Glasgow, United Kingdom, G51 4TF
        • Queen Elizabeth University Hospital
      • Leeds, United Kingdom, LS9 7TF
        • St James University Hospital
      • Liverpool, United Kingdom, L7 8YA
        • Clatterbridge Cancer Center
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85054
        • Mayo Clinic Hospital
    • California
      • Duarte, California, United States, 91010
        • City of Hope Medical Center
      • Los Angeles, California, United States, 90095
        • University of California-Los Angeles Medl Cntr-Oncology Center Bowyer Clinic
      • Stanford, California, United States, 94305
        • Stanford Cancer Center
    • Colorado
      • Aurora, Colorado, United States, 80045
        • University of Colorado Cancer Center
      • Denver, Colorado, United States, 80218
        • Colorado Blood Cancer Institute
    • Connecticut
      • New Haven, Connecticut, United States, 06510
        • Smilow Cancer Center-Yale
    • Florida
      • Jacksonville, Florida, United States, 32224
        • Mayo Clinic Jacksonville
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Northwestern University
      • Chicago, Illinois, United States, 60637
        • The University of Chicago Medicine
    • Maryland
      • Baltimore, Maryland, United States, 21201
        • University of Maryland-Greenebaum Cancer Center
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Washington University
    • Nebraska
      • Omaha, Nebraska, United States, 68198
        • Fred and Pamela Buffett Cancer Center
    • New York
      • New York, New York, United States, 10021
        • Memorial Sloan Kettering Cancer Center
      • New York, New York, United States, 10021
        • Weill Cornell Medicine
      • New York, New York, United States, 10029
        • Mount Sinai Hospital
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic
      • Columbus, Ohio, United States, 43210
        • The Ohio State University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • University of Pennsylvania Abramson Cancer Center
      • Pittsburgh, Pennsylvania, United States, 15232
        • Hillman Cancer Center
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt University Medical Center
    • Texas
      • Houston, Texas, United States, 77030
        • Md Anderson Cancer Center
    • Washington
      • Seattle, Washington, United States, 98195
        • University of Washington
      • Seattle, Washington, United States, 98109
        • Fred Hutchinson Cancer Center
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Froedtert & the Medical College of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Treatment Group A
Axatilimab will be administered at a protocol defined starting dose plus ruxolitinib at a protocol defined starting dose.
Drug: Axatilimab
Axatilimab will be administered at protocol defined dose.
Drug: Ruxolitinib
Ruxolitinib will be administered at protocol defined dose.
Experimental: Treatment Group B
Ruxolitinib will be administered at a protocol defined starting dose.
Drug: Ruxolitinib
Ruxolitinib will be administered at protocol defined dose.
Experimental: Treatment Group C
Corticosteroids alone will be administered at a protocol defined starting dose.
Drug: Corticosteroids
Corticosteroids will be administered at protocol defined dose.
Other Names:
  • prednisone, prednisolone, methylprednisolone

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Objective Response Rate
Time Frame: 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.
6 months

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Proportion of participants with a ≥ 7-point improvement in modified Lee symptom scale (mLSS) score
Time Frame: Up to 2 years
Up to 2 years
Axatilimab pharmacokinetic (PK) in Plasma
Time Frame: Up to 2 years and 30 days
Axatilimab concentration in plasma.
Up to 2 years and 30 days
Number of participants with Treatment-emergent Adverse Events (TEAEs)
Time Frame: Up to 2 years and 30 days
Defined as adverse events reported for the first time or worsening of a pre-existing event after the first dose of study treatment.
Up to 2 years and 30 days
Duration of Response
Time Frame: Up to 2 years
Defined as the time from the first response (PR or CR) to the date of progression of cGVHD, start of new systemic therapy or death from any cause.
Up to 2 years
Best overall response in the first 6 months
Time Frame: Up to 6 months
Define as PR or CR in the first 6 months.
Up to 6 months
OR at 12 months, defined as CR or PR at 12 months (C14D1) in the absence of new systemic therapy for cGVHD.
Time Frame: 12 months
Defined as CR or PR at 12 months in the absence of new systemic therapy for cGVHD
12 months
Proportion of participants who remain corticosteroid-free
Time Frame: 4 weeks, 8 weeks and 6 months
4 weeks, 8 weeks and 6 months
Organ-specific response in the first 6 cycles and on study, based on the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD.
Time Frame: Up to 2 years
Based on the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD.
Up to 2 years
Failure-free Survival (FFS)
Time Frame: Up to 2 years and 30 days
Defined as the time from date of randomization to date of initiation of a new cGVHD treatment, malignancy relapse, or death due to any cause.
Up to 2 years and 30 days
Ruxolitinib PK in Plasma
Time Frame: Up to 2 years and 30 days
Ruxolitinib concentration in plasma.
Up to 2 years and 30 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Incyte Corporation

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Incyte Medical Monitor, Incyte Corporation

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 11, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 1, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2029

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 25, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 25, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 29, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 22, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 20, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • INCA34176-254
  • 2022-502168-19-00 (Registry Identifier: EU CT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Incyte shares data with qualified external researchers after a research proposal is submitted. These requests are reviewed and approved by a review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. The trial data availability is according to the criteria and process described on https://www.incyte.com/our-company/compliance-and-transparency

IPD Sharing Time Frame

Data will be shared after the primary publication or 2 years after the study has ended for market authorized products and indications.

IPD Sharing Access Criteria

Data from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.incyteclinicaltrials.com website. For approved requests, the researchers will be granted access to anonymized data under the terms of a data sharing agreement.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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