Recruiting

AGAVE-256Axatilimab for Pediatric Chronic Graft-Versus-Host Disease

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What is being tested

INCA034176

+ Best available Treatment (BAT)

Drug
Who is being recruted

Bronchiolitis Obliterans Syndrome+8

+ Organizing Pneumonia

+ Bronchial Diseases

From 2 to 17 Years
+14 Eligibility Criteria
See all eligibility criteria
How is the trial designed

Treatment Study

Phase 2
Interventional
Study Start: March 2026
See protocol details

Summary

Principal SponsorIncyte Corporation
Study ContactIncyte Corporation Call Center (US)More contacts
Last updated: March 21, 2026
Sourced from a government-validated database.Claim as a partner

Study start date: March 31, 2026

Actual date on which the first participant was enrolled.

This study is focused on finding the best treatment for children and teens who have a condition called chronic graft-versus-host disease (cGVHD) and have already tried at least two other treatments. cGVHD can happen after a bone marrow or stem cell transplant, where the transplanted cells attack the body. This trial looks at how well a medicine called Axatilimab works compared to other treatments currently available. The goal is to see if Axatilimab can provide better relief from symptoms and improve quality of life for these young patients. Participants in this study will be randomly assigned to receive either Axatilimab or the best available therapy as determined by their doctor. Axatilimab is administered to see how it compares to existing treatments. Researchers will monitor the participants to evaluate the effectiveness of the treatments, focusing on how the symptoms of cGVHD change. The study does not mention specific outcomes being measured, but typically such trials look at improvements in symptoms and side effects. Understanding these results can help guide future treatment options for children with cGVHD.

Official TitleA 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)
NCT07124078
Principal SponsorIncyte Corporation
Study ContactIncyte Corporation Call Center (US)More contacts
Last updated: March 21, 2026
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

60 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 2 to 17 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Bronchiolitis Obliterans SyndromeOrganizing PneumoniaBronchial DiseasesBronchiolitisBronchiolitis ObliteransBronchitisGraft vs Host DiseaseImmune System DiseasesLung DiseasesLung Diseases, ObstructiveRespiratory Tract Diseases

Criteria

6 inclusion criteria required to participate
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.
Aged ≥ 2 to < 18 years at the time of signing the informed consent.
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8 exclusion criteria prevent from participating
Receipt of more than 1 prior allo-HCT. Prior autologous HCT is allowed.
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.
Systemic treatment with CNIs or mTOR inhibitors started within 2 weeks prior to C1D1.
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Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives

2 intervention groups are designated in this study

This study does not include a placebo group 

Treatment Groups

Group I

Experimental
Axatilimab at the protocol-defined dose.

Group II

Experimental
Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 38 locations

Recruiting

City of Hope Medical Center

Duarte, United StatesOpen City of Hope Medical Center in Google Maps
Recruiting

Memorial Sloan Kettering Cancer Center

New York, United States
Recruiting

Hospital Universitario Vall D'Hebron

Barcelona, Spain
Recruiting

Hospital Universitario La Paz

Madrid, Spain
Recruiting
38 Study Centers