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Comparing Axatilimab to Best Available Therapy for Chronic Graft-versus-host Disease

A Phase 3, Randomized, Open-Label Study of Axatilimab Versus Best Available Therapy in Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy

Phase 3 Interventional Incyte Corporation · NCT06821542

This study is testing if a new treatment called Axatilimab works better than the best existing therapies for people aged 12 and older with chronic graft-versus-host disease who haven't improved after trying at least two other treatments.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment300 (estimated)
Ages12 Years and up
SexAll
SponsorIncyte Corporation Industry-sponsored
Drugs / interventionsrituximab, ruxolitinib, imatinib, ibrutinib, Axatilimab
Locations131 sites (Graz and 130 other locations)
Trial IDNCT06821542 on ClinicalTrials.gov

What this trial studies

This study aims to evaluate the effectiveness of Axatilimab compared to the best available therapy in patients suffering from chronic graft-versus-host disease (cGVHD) who have not responded to at least two prior systemic treatments. Participants will be aged 12 years and older and must have moderate to severe cGVHD requiring systemic immune suppression. The study will involve administering Axatilimab or a range of established therapies, with the goal of determining which treatment offers better outcomes for patients with this challenging condition.

Who should consider this trial

Good fit: Ideal candidates for this study are individuals aged 12 and older with active, moderate to severe chronic graft-versus-host disease who have undergone at least two lines of systemic therapy.

Not a fit: Patients who have not received prior systemic therapy or those with mild cGVHD may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new effective treatment option for patients with chronic graft-versus-host disease who have limited responses to existing therapies.

How similar studies have performed: Other studies have explored treatments for chronic graft-versus-host disease, but the specific use of Axatilimab in this context is a novel approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Age ≥ 12 years at the time of signing the ICF.
* Active, moderate to severe cGVHD, requiring systemic immune suppression.
* Participants with refractory or recurrent cGVHD who have received at least 2 lines of systemic therapy, including corticosteroids and ruxolitinib.
* Concomitant use of systemic corticosteroids is allowed. Participants on systemic corticosteroids must be on a stable dose of corticosteroids for at least 2 weeks prior to C1D1. Topical and inhaled corticosteroid agents are allowed.
* Participants must accept to be treated with one of the following BAT options on C1D1: CNI (cyclosporine or tacrolimus), ECP, MMF, an mTOR inhibitor (everolimus or sirolimus), rituximab, pentostatin, proteasome inhibitors, imatinib, or ibrutinib.
* History of allo-HCT from any donor HLA type (related or unrelated donor with any degree of HLA matching) using any graft source (bone marrow, peripheral blood stem cells, or cord blood). Recipients of myeloablative, nonmyeloablative, or reduced-intensity conditioning are eligible.

Exclusion Criteria:

* Receipt of more than 1 prior allo-HCT. Prior autologous HCT is allowed.
* Evidence of relapse of hematologic disease or treatment for relapse after the allo-SCT was performed, including DLI for the treatment of molecular relapse. Note: Participants who have received a scheduled DLI as part of their transplant procedure and not for management of malignancy relapse are eligible.
* Systemic treatment with CNIs or mTOR inhibitors started within 2 weeks prior to C1D1.
* Severe renal impairment, that is, estimated creatinine clearance \< 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.
* 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.
* History of acute or chronic pancreatitis.
* Active, symptomatic myositis.
* Pregnant or breastfeeding.

Other protocol-defined Inclusion/Exclusion Criteria may apply.

Where this trial is running

Graz and 130 other locations

+81 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Chronic Graft-versus-host-diseasecGVHD
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.