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Axatilimab for Chronic Graft-Versus-Host Disease in Children

This study is looking at a treatment called Axatilimab for children aged 2 to 17 years old who have chronic graft-versus-host disease (cGVHD). cGVHD is a condition that can happen after a stem cell transplant where the donor's immune cells attack the patient's body. Participants in this study have moderate to severe cGVHD that hasn't improved with at least two other treatments, including corticosteroids and ruxolitinib. The study will compare Axatilimab to the best available treatment chosen by the doctor. The main goal is to see if participants have an "Objective Response" (meaning their cGVHD improves) after 6 months of treatment. The study plans to enroll 60 participants.

Study design
This study is comparing Axatilimab to the best available treatment for chronic GVHD in children. It aims to enroll 60 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The primary outcome is measured at 6 months, but the total follow-up duration is not specified.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07124078

A Study to Evaluate 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)

Recruiting
PHASE2Ages 2–17InterventionalTreatment
Incyte Corporation
~60 participants
Updated 2026-08-25 on ClinicalTrials.gov
What's tested:INCA034176Best available Treatment (BAT)

At a glance

Recruiting sites
31 of 41 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Objective Response (OR) at 6 months
Measured over 6 months
+1 more outcome measured

Conditions

Chronic Graft-versus-host-disease

Explore related trials

Graft Versus Host Disease

Where it's being run

41 sites across 13 states
United Kingdom9
Spain7
Germany6
Italy6
California3
North Carolina2
Belgium2
District of Columbia1

Study leadership

  • Incyte Medical Monitor · STUDY_DIRECTOR · Incyte Corporation

Who to contact

Incyte Corporation Call Center (US)
[email protected]
1.855.463.3463
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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Aged 2 to \< 18 years at the time of randomization.
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, ibrutinib, or pentostatin.
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

Receipt of more than 1 prior allo-HCT. Prior autologous HCT is allowed, including autologous CAR T-cell therapy given before the allo-SCT.
Documented evidence of relapse of the primary hematologic disease or receipt of treatment for relapse after allo-SCT, including DLI for treatment of any malignancy relapse, including molecular relapse. Autologous and allogeneic donor-derived CAR T-cell therapy after allo-SCT are not allowed. Note: Participants who received DLI solely for the management of mixed chimerism or as part of the planned transplant procedure and not for treatment of malignancy relapse (including molecular 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 or ALT \> 3 × ULN or 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.

What this trial measures

  • Objective Response (OR) at 6 months6 months

    Defined as complete response (CR) or partial response (PR) at 6 months (Cycle 7 Day 1, 28-day cycles) in the absence of new systemic therapy for cGVHD. Responses defined by the 2014 NIH consensus criteria.

  • United States (US): Best overall Response (BOR)Up to 6 months

    Defined as a best response of CR or PR in the first 6 months (up to and including Cycle 7 Day 1) in the absence of new systemic therapy for cGVHD.