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Axatilimab versus best available therapy for children with chronic graft-versus-host disease after two or more prior treatments

A Phase 2, Randomized, Open-Label Study of Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft-Versus-Host Disease After at Least 2 Prior Lines of Systemic Therapy (AGAVE-256)

Phase 2 Interventional Incyte Corporation · NCT07124078

This trial will test axatilimab against the best available treatments in children aged 2–17 whose chronic graft-versus-host disease has not responded to at least two prior systemic therapies.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment60 (estimated)
Ages2 Years to 17 Years
SexAll
SponsorIncyte Corporation Industry-sponsored
Drugs / interventionsrituximab, ruxolitinib, imatinib, ibrutinib, methotrexate, Axatilimab
Locations40 sites (Duarte, California and 39 other locations)
Trial IDNCT07124078 on ClinicalTrials.gov

What this trial studies

This phase 2 interventional study enrolls pediatric participants aged 2 to under 18 with active moderate-to-severe chronic graft-versus-host disease that is refractory or recurrent after at least two prior systemic therapies, including corticosteroids and ruxolitinib. Participants will receive either axatilimab (INCA034176) or a selected best available therapy (such as a calcineurin inhibitor, ECP, mycophenolate mofetil, an mTOR inhibitor, rituximab, imatinib, methotrexate, or ibrutinib) with concomitant corticosteroids allowed if on a stable dose. The trial is sponsored by Incyte Corporation and will recruit at several U.S. pediatric transplant centers in California. Safety, tolerability, and clinical response measures will be collected to compare outcomes between the axatilimab and best-available-therapy arms.

Who should consider this trial

Good fit: Ideal candidates are children aged 2 to under 18 with active moderate-to-severe cGVHD who have failed at least two prior systemic therapies (including corticosteroids and ruxolitinib) and who can be treated with one of the allowed best-available therapy options.

Not a fit: Children with mild cGVHD, those younger than 2 or 18 and older, or patients who have not received at least two prior systemic therapies are unlikely to be eligible or benefit from this trial.

Why it matters

Potential benefit: If effective, axatilimab could offer a new treatment option to control symptoms and reduce steroid dependence for children with refractory cGVHD.

How similar studies have performed: Similar targeted agents and early adult studies in cGVHD have shown encouraging signals, but pediatric data are limited and this approach is still being tested in children.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Aged ≥ 2 to \< 18 years at the time of signing the informed consent.
* 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, imatinib, methotrexate, 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, GFR \< 30 mL/min/1.73 m2 as estimated using modified Schwartz formula, 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.
* Female adolescent participants who are pregnant or breastfeeding.

Other protocol-defined Inclusion/Exclusion Criteria may apply.

Where this trial is running

Duarte, California and 39 other locations

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.