Chronic Graft-versus-host-diseasePhase 3NCT06263478
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
A Phase 3 study testing INCA034176 in people with Chronic Graft-versus-host-disease, run by Incyte Biosciences Japan GK at 16 sites in Japan.
Trial summary
This study will be conducted to determine the clinical efficacy of axatilimab in Japanese participants with chronic graft-versus-host disease (cGVHD). Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Chronic Graft-versus-host-disease
- What is being tested?
- INCA034176
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 16 sitesJapan
- Study dates
- 30 Jul 2024to 30 Mar 2027
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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:
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
16 sites in 1 country.
16 sites in 1 country
Japan16 sites
Fujita Health University Hospital
Aichi, 470-1192
University of Fukui Hospital
Fukui, 910-1193
Kyushu University Hospital
Fukuoka
Hiroshima University Hospital
Hiroshima, 734-8551
Hokkaido University Hospital
Hokkaido, 060-8648
Kobe City Medical Center General Hospital
Hyōgo, 650-0047
Tokai University Hospital
Kanagawa, 259-1193
National Hospital Organization Kumamoto Medical Center
Kumamoto, 860-0008
Tohoku University Hospital
Miyagi, 980-8574
Japanese Red Cross Aichi Medical Center Nagoya Daiichi Hospital
Nagoya, 453-8511
Okayama University Hospital
Okayama, 700-8558
Osaka Prefectural Hospital Organization Osaka International Cancer Institute
Osaka, 541-8567
Osaka Metropolitan University Hospital
Osaka, 545-8586
Saitama Prefectural Children'S Medical Center
Saitama-shi, Saitama,, 330-8777
Tokyo Metropolitan Cancer and Infectious Diseases Center Komagome Hospital
Tokyo, 113-8677
National Center For Child Health and Development
Tokyo, 157-8535
About this study
Official title: 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).
- Conditions
- Chronic Graft-versus-host-disease
- Being tested
- INCA034176
- Keywords
- cGVHD
- Medical subject headings
- Bronchiolitis Obliterans Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
INCA034176Drug
Also called Axatilimab
IV infusion
| Study group | Role | What they receive |
|---|---|---|
| Axatilimab Dose | Experimental | Axatilimab at the protocol-defined dose. Receives: Drug: INCA034176 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
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.
Measured: up to the end of Cycle 6 (up to 168 days)
Secondary outcomes (11)
Percentage of Participants With a ≥7-point Improvement in Modified Lee Symptom Scale (mLSS) Score
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.
Measured: 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
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.
Measured: up to 12.8 months
Duration of Response (DOR) in Participants With a Response in the First 6 Treatment Cycles
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.
Measured: up to 13.8 months (maximum duration of treatment [12.8 months] plus 30 days)
Organ-specific Response Rate
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.
Measured: up to 12.8 months
Percent Reduction in Average Daily Dose (or Equivalent) of Corticosteroids in the First 6 Treatment Cycles
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.
Measured: 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
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.
Measured: up to the end of Cycle 6 (up to 168 days)
Number of Participants With Any Treatment-emergent Adverse Event (TEAE)
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.
Measured: up to 13.8 months
Number of Participants With Any ≥Grade 3 TEAE
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.
Measured: up to 13.8 months
Change From Baseline in Karnofsky/Lansky Performance Status
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.
Measured: up to 13.8 months
Percentage of Participants Positive for Anti-drug Antibodies (ADAs)
Antibodies to axatilimab were evaluated in plasma samples collected from all participants.
Measured: up to 12.8 months
INCA034176 Concentration
Plasma samples were collected for pharmacokinetic (PK) analysis.
Measured: up to 12.8 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Feb 2024
- Study start30 Jul 2024
- Primary completion6 Aug 2025
- Study completion30 Mar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Chronic Graft-versus-host-diseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Incyte Biosciences Japan GK
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Incyte Medical Monitor, Study director, Incyte Corporation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06263478
- Sponsor's protocol ID
- INCA34176-358
- Status last verified
- Oct 2026
- First submitted
- 9 Feb 2024
- Last updated
- 5 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
Study at a glance
From the registry record, last updated by the sponsor on 5 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06263478
- Acronym / sponsor ID
- INCA34176-358
- Conditions
- Chronic Graft-versus-host-disease
- Keywords
- cGVHD
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Incyte Biosciences Japan GKIndustry (pharma, biotech, devices)
- Study start
- 30 Jul 2024 (actual)
- Primary completion
- 6 Aug 2025 (actual)
- Study completion
- 30 Mar 2027 (estimated)
- First posted
- 16 Feb 2024 (actual)
- Results
- Posted 5 Oct 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will be conducted to determine the clinical efficacy of axatilimab in Japanese participants with chronic graft-versus-host disease (cGVHD).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Axatilimab Dose | Experimental | Drug: INCA034176Axatilimab at the protocol-defined dose. |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (17 listed)
- 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.
- 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.
- 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).
Key exclusion criteria (13 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| INCA034176Also: AxatilimabIV infusion | Drug | Axatilimab Dose |
Primary outcome
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.
11 secondary outcomes
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)
ORR, as Defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD
Time frame: 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)
Organ-specific Response Rate
Time frame: 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)
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)
Number of Participants With Any Treatment-emergent Adverse Event (TEAE)
Time frame: up to 13.8 months
Number of Participants With Any ≥Grade 3 TEAE
Time frame: up to 13.8 months
Change From Baseline in Karnofsky/Lansky Performance Status
Time frame: up to 13.8 months
Percentage of Participants Positive for Anti-drug Antibodies (ADAs)
Time frame: up to 12.8 months
INCA034176 Concentration
Time frame: up to 12.8 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Oct 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- A study to evaluate the Efficacy, Safety, and Pharmacokinetics of Axatilimab Monotherapy in Japanese Participants With Recurrent or Refractory Active Chronic Graft-Versus-Host DiseaseLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Incyte Medical Monitor study director
Incyte Corporation
16 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing INCA034176 in people with Chronic Graft-versus-host-disease, run by Incyte Biosciences Japan GK at 16 sites in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 5 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 5 Oct 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Oct 2026.