A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Axatilimab Monotherapy in Japanese Participants With Recurrent or Refractory Active Chronic Graft-Versus-Host Disease

October 1, 2026 updated by: Incyte Biosciences Japan GK

A Phase 3, Open-Label, Multicenter Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Axatilimab Monotherapy in Japanese Participants With Recurrent or Refractory Active Chronic Graft-Versus-Host Disease After at Least 2 Lines of Systemic Therapy

This study will be conducted to determine the clinical efficacy of axatilimab in Japanese participants with chronic graft-versus-host disease (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.
21

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 3

Contacts and Locations

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

Study Locations

  • Japan
      • Aichi, Japan, 470-1192
        • Fujita Health University Hospital
      • Fukui, Japan, 910-1193
        • University of Fukui Hospital
      • Fukuoka, Japan
        • Kyushu University Hospital
      • Hiroshima, Japan, 734-8551
        • Hiroshima University Hospital
      • Hokkaido, Japan, 060-8648
        • Hokkaido University Hospital
      • Hyōgo, Japan, 650-0047
        • Kobe City Medical Center General Hospital
      • Kanagawa, Japan, 259-1193
        • Tokai University Hospital
      • Kumamoto, Japan, 860-0008
        • National Hospital Organization Kumamoto Medical Center
      • Miyagi, Japan, 980-8574
        • Tohoku University Hospital
      • Nagoya, Japan, 453-8511
        • Japanese Red Cross Aichi Medical Center Nagoya Daiichi Hospital
      • Okayama, Japan, 700-8558
        • Okayama University Hospital
      • Osaka, Japan, 541-8567
        • Osaka Prefectural Hospital Organization Osaka International Cancer Institute
      • Osaka, Japan, 545-8586
        • Osaka Metropolitan University Hospital
      • Saitama-shi, Saitama,, Japan, 330-8777
        • Saitama Prefectural Children'S Medical Center
      • Tokyo, Japan, 113-8677
        • Tokyo Metropolitan Cancer and Infectious Diseases Center Komagome Hospital
      • Tokyo, Japan, 157-8535
        • National Center For Child Health and Development

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

6 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • At least 6 years of age at the time of signing the ICF.

  • Ability to comprehend and willingness to sign a written ICF for the study.

    • For participants 6 to 17 years old, a parent/guardian must provide consent for pediatric participants; when applicable, pediatric participants should also sign an assent form.

  • Japanese participants who are allo-HSCT recipients with active, refractory, or recurrent cGVHD requiring systemic immune suppression despite at least 2 lines of prior systemic therapy.

    • Active cGVHD is defined as the presence of signs and symptoms of cGVHD per the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD.

    • Refractory disease is defined as meeting any of the following criteria:

      • The development of 1 or more new sites of disease while being treated for cGVHD.
      • Progression of existing sites of disease despite at least 1 month of standard or investigational therapy for cGVHD.
      • Participants who did not achieve a response within 3 months on prior therapy for cGVHD and for whom the treating physician believes a new systemic therapy is required.

    • Recurrent cGVHD is defined as active, symptomatic disease (after an initial response to prior therapy) based on the NIH 2014 consensus criteria by organ-specific or global assessment or for which the physician believes a new line of systemic therapy is required.

  • Participants may have persistent, active aGVHD and cGVHD manifestations (overlap syndrome), as defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD.

  • Karnofsky performance score of ≥ 60 (if aged 16 years or older); Lansky performance score of ≥ 60 (if aged younger than 16 years).

  • Adequate organ and bone marrow functions evaluated during the 14 days prior to the start of study treatment.

  • Creatinine clearance ≥ 30 mL/min based on the Cockcroft-Gault formula in adult participants and Schwartz formula in pediatric participants.

  • Concomitant use of a systemic corticosteroid is allowed but not required. Topical and inhaled corticosteroid agents are allowed. If a participant is taking a corticosteroid, it must be a stable dose for at least 2 weeks prior to the start of study treatment.

  • Concomitant use of protocol-defined immunosuppressant is allowed but not required.

  • Willingness to avoid pregnancy or fathering children based on protocol-defined criteria.

Exclusion Criteria:

  • Has aGVHD without manifestations of cGVHD.
  • Any evidence (histologic, cytogenetic, molecular, hematologic, or mixed) of relapse of the underlying cancer or post-transplant lymphoproliferative disease at the time of screening.
  • History of acute or chronic pancreatitis.
  • History of myositis.
  • History or other evidence of severe illness, uncontrolled infection, allergy to excipients, or any other conditions that would make the participant, in the opinion of the investigator, unsuitable for the study.
  • Has acquired immunodeficiency syndrome.
  • History of latent or active TB based on protocol-defined criteria.
  • Active HBV or HCV infection that requires treatment, or at risk for HBV reactivation (ie, positive HBsAg).
  • Pregnant or breastfeeding.
  • Previous exposure to CSF-1R targeted therapies.
  • Use of any agent other than corticosteroids, or the immunosuppressant for the treatment of cGVHD within 2 weeks or 5 half-lives, whichever is shorter, prior to the start of study treatment.
  • Has received an investigational treatment within 28 days prior to the start of study treatment.
  • Currently participating in any other interventional study.

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: N/A
  • Interventional Model: Single Group 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: Axatilimab Dose

Axatilimab at the protocol-defined dose.
Drug: INCA034176

IV infusion

Other Names:
  • Axatilimab

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
Overall Response Rate (ORR) in the First 6 Treatment Cycles, as Defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD
Time Frame: up to the end of Cycle 6 (up to 168 days)

ORR was defined as the percentage of participants with a physician-assessed complete response (CR) or a physician-assessed partial response (PR), evaluated as defined by the 2014 National Institutes of Health (NIH) Consensus Development Project on Criteria for Clinical Trials in chronic graft-versus-host disease (cGVHD). A participant was considered an objective responder if they had an overall response of CR or PR at any timepoint up to the beginning of Cycle 7. CR was defined as the resolution of all manifestations in each organ or site, and PR was defined as improvement in at least 1 organ or site without progression in any other organ or site.
up to the end of Cycle 6 (up to 168 days)

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
Percentage of Participants With a ≥7-point Improvement in Modified Lee Symptom Scale (mLSS) Score
Time Frame: before Cycle 7 Day 1 (up to 168 days); during study (up to 12.8 months)

The mLSS is a 28-item instrument comprising 7 subscales, designed to assess the impact of cGVHD on a participant's symptoms. Participants were asked to indicate the degree of "bother" during the past 7 days due to symptoms in 7 domains (skin, eyes and mouth, breathing, eating and digestion, muscles and joints, energy, emotional distress) potentially affected by cGVHD. A mLSS responder is defined as a participant who achieved at least a 7-point improvement from baseline in the normalized mLSS score. This score is on a 0-100 scale, calculated as the average of the subscale scores.
before Cycle 7 Day 1 (up to 168 days); during study (up to 12.8 months)
ORR, as Defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD
Time Frame: up to 12.8 months

ORR was defined as the percentage of participants with a physician-assessed CR or a physician-assessed PR, evaluated as defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD. A participant was considered an objective responder if they had an overall response of CR or PR at any post-baseline visit. CR was defined as the resolution of all manifestations in each organ or site, and PR was defined as improvement in at least 1 organ or site without progression in any other organ or site.
up to 12.8 months
Duration of Response (DOR) in Participants With a Response in the First 6 Treatment Cycles
Time Frame: up to 13.8 months (maximum duration of treatment [12.8 months] plus 30 days)

Duration of response was defined as the time from the first response of either CR or PR until progression of cGVHD, start of another anti-GVHD treatment, or death from any cause, whichever was earlier. CR was defined as the resolution of all manifestations in each organ or site, and PR was defined as improvement in at least 1 organ or site without progression in any other organ or site. DOR was assessed for participants whose first CR or PR occurred during the first 6 treatment cycles. A response was followed until the response ended, regardless of whether the response ended during the first 6 treatment cycles or after Cycle 6.
up to 13.8 months (maximum duration of treatment [12.8 months] plus 30 days)
Organ-specific Response Rate
Time Frame: up to 12.8 months

Organ-specific response rate was defined as the percentage of participants with a CR or PR for the 9 individual organs based on the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD (skin, eyes, mouth, esophagus, upper gastrointestinal (GI) tract, lower GI, liver, lungs, and joints and fascia) in participants with each corresponding organ involvement at baseline.
up to 12.8 months
Percent Reduction in Average Daily Dose (or Equivalent) of Corticosteroids in the First 6 Treatment Cycles
Time Frame: up to the end of Cycle 6 (up to 168 days)

Participants may have been taking a corticosteroid upon study entry. At the discretion of the investigator, the corticosteroid dose may have been tapered after the first 4 weeks of study drug administration for participants who achieved either a PR or CR. Percent reduction was calculated as the ([difference between the baseline value and the post-baseline value]/divided by the baseline value) x 100.
up to the end of Cycle 6 (up to 168 days)
Percentage of Participants Who Discontinued Corticosteroid Use After Study Entry in the First 6 Treatment Cycles
Time Frame: up to the end of Cycle 6 (up to 168 days)

Participants may have been taking a corticosteroid upon study entry. At the discretion of the investigator, the corticosteroid dose may have been tapered after the first 4 weeks of study drug administration for participants who achieved either a PR or CR.
up to the end of Cycle 6 (up to 168 days)
Number of Participants With Any Treatment-emergent Adverse Event (TEAE)
Time Frame: up to 13.8 months

An adverse event was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it is considered drug related. An AE could therefore have been any unfavorable or unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study drug. For the purposes of analysis, all AEs were considered to be TEAEs unless the AE could unequivocally be defined as not treatment emergent.
up to 13.8 months
Number of Participants With Any ≥Grade 3 TEAE
Time Frame: up to 13.8 months

An adverse event was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not it is considered drug related. For the purposes of analysis, all AEs were considered to be TEAEs unless the AE could unequivocally be defined as not treatment emergent. The severity of AEs was assessed using Common Terminology Criteria for Adverse Events version 5.0 (CTCAE v5.0) Grades 1 through 5. Grade 1: mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; treatment not indicated. Grade 2: moderate; minimal, local, or noninvasive treatment indicated; limiting age-appropriate activities of daily living. Grade 3: severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care activities of daily living. Grade 4: life-threatening consequences; urgent treatment indicated. Grade 5: fatal.
up to 13.8 months
Change From Baseline in Karnofsky/Lansky Performance Status
Time Frame: up to 13.8 months

The Karnofsky/Lansky performance status allows participants to be classified as to their functional impairment on a scale from 0 to 100. The lower the score, the worse the survival for most serious illnesses. The score can be used to compare the effectiveness of different therapies and to assess the prognosis in individual participants. The Karnofsky scale is designed for participants aged 16 years and older, and the Lansky scale is designed for participants younger than 16 years. Change from baseline was calculated as the post-baseline value minus the baseline value.
up to 13.8 months
Percentage of Participants Positive for Anti-drug Antibodies (ADAs)
Time Frame: up to 12.8 months

Antibodies to axatilimab were evaluated in plasma samples collected from all participants.
up to 12.8 months
INCA034176 Concentration
Time Frame: up to 12.8 months

Plasma samples were collected for pharmacokinetic (PK) analysis.
up to 12.8 months

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 Biosciences Japan GK

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.
July 30, 2024

Primary Completion (Actual)

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.
August 6, 2025

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.
March 30, 2027

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).
February 9, 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.
February 9, 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.
February 16, 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.
October 5, 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.
October 1, 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.
October 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.
  • INCA34176-358

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

product manufactured in and exported from the U.S.

Yes

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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