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A Study of Axatilimab for Chronic Graft Versus Host Disease (cGVHD)

This study is testing a drug called axatilimab in people with chronic graft-versus-host disease (cGVHD). cGVHD is a condition where donor cells attack the recipient's body after a stem cell transplant. Axatilimab works by targeting a specific protein (CSF-1R) that helps certain immune cells (macrophages) grow and cause disease. The study aims to see how well axatilimab works, how safe it is, and if people can tolerate it at three different doses. You may be able to join if you are at least 2 years old, have active cGVHD, and have already tried at least two other treatments. The main goal is to see how many people respond to the treatment within the first 6 cycles (about 6 months). The current status of this study is unclear.

Study design
This is a Phase 2, open-label, randomized study involving up to 296 participants. Participants will be randomly assigned to receive one of three different doses of axatilimab.
What's involved
Participants will receive axatilimab treatment in 28-day cycles for up to 2 years.
Compensation
Not stated in the trial record.
Follow-up
The primary endpoint is measured at the first 6 cycles (up to Cycle 7 Day 1).

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

NCT04710576

A Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD)

Recruiting
PHASE2Ages 2+InterventionalTreatment
Syndax Pharmaceuticals
~296 participants
Updated 2026-06-16 on ClinicalTrials.gov
What's tested:Axatilimab

At a glance

Recruiting sites
13 of 121 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Overall Response Rate (ORR) in the First 6 Cycles as Defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-Versus-Host Disease (cGVHD)
Measured over First 6 cycles (up to Cycle 7 Day 1; each cycle = 4 weeks)

Conditions

Chronic Graft-versus-host-disease

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Chronic Graft Versus Host Disease

NCT04710576

Where you'd take part

This study runs at 121 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Barbara Ann Karmanos Cancer Institute

    Detroit, Michiganno site contact published

    Recruiting

  • Emory University

    Atlanta, Georgiano site contact published

    Recruiting

  • Fred Hutchinson Cancer Research Center

    Seattle, Washingtonno site contact published

    Recruiting

  • Intermountain Healthcare

    Salt Lake City, Utahno site contact published

    Recruiting

  • Massachusetts General Hospital

    Boston, Massachusettsno site contact published

    Recruiting

  • Oregon Health & Science University

    Portland, Oregonno site contact published

    Recruiting

  • The Ohio State University Comprehensive Cancer Center

    Columbus, Ohiono site contact published

    Recruiting

  • The University of Chicago Medical Center (UCMC)

    Chicago, Illinoisno site contact published

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Ellen Hooper, M.D. · STUDY_DIRECTOR · Syndax Pharmaceuticals

Who to contact

Syndax Pharmaceuticals
[email protected]
781-419-1400
Email the study team

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Eligibility criteria

Inclusion

Refractory disease defined as meeting any of the following criteria:
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 investigation therapy for cGVHD.
Participants who have not achieved a response within 3 months on their prior therapy for cGVHD and for whom the treating physician believes a new systemic therapy is required.
Recurrent cGVHD is active, symptomatic disease (after an initial response to prior therapy) as defined, based on the NIH 2014 consensus criteria, by organ-specific or global assessment or for which the physician believes that a new line of systemic therapy is required. 4. Participants may have persistent, active acute and cGVHD manifestations (overlap syndrome), as defined by 2014 NIH Consensus Development Project on Criteria for Clinical trials in cGVHD. 5. Karnofsky Performance Scale of ≥60 (if aged 16 years or older); Lansky Performance Score of ≥60 (if aged \<16 years) 6. Adequate organ and bone marrow functions evaluated during the 14 days prior to randomization. 7. Creatinine clearance (CrCl) ≥30 milliliter/minute based on the Cockcroft-Gault formula in adult participants and Schwartz formula in pediatric participants. 8. Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. 9. Concomitant use a of systemic corticosteroid is allowed but not required. Topical and inhaled corticosteroid agents are allowed. If a participant is taking corticosteroids at study randomization, they must be on a stable dose of corticosteroids for at least 2 weeks prior to Cycle 1 Day 1. 10. Concomitant use of CNI or mammalian target of repamycin (mTOR) inhibitors (sirolimus or everolimus) is allowed but not required. 11. Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and protocol. A parent/guardian should provide consent for pediatric participants unable to provide consent themselves; in addition, where applicable pediatric participants should sign their own assent form.

What this trial measures

  • Overall Response Rate (ORR) in the First 6 Cycles as Defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-Versus-Host Disease (cGVHD)First 6 cycles (up to Cycle 7 Day 1; each cycle = 4 weeks)

    The ORR was defined as the percentage of participants with objective response (complete response \[CR\] or partial response \[PR\]). CR was defined as 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.