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Evaluating Axatilimab with Ruxolitinib for Chronic Graft-Versus-Host Disease

A Phase 2, Open-Label, Randomized, Multicenter Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease

Phase 2 Interventional Incyte Corporation · NCT06388564

This study is testing whether a new treatment combining axatilimab and ruxolitinib can help people with newly diagnosed chronic graft-versus-host disease feel better after a stem cell transplant.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment120 (estimated)
Ages12 Years and up
SexAll
SponsorIncyte Corporation Industry-sponsored
Drugs / interventionsaxatilimab, ruxolitinib
Locations70 sites (Phoenix, Arizona and 69 other locations)
Trial IDNCT06388564 on ClinicalTrials.gov

What this trial studies

This study aims to assess the safety and efficacy of axatilimab in combination with ruxolitinib for patients with newly diagnosed chronic graft-versus-host disease (cGVHD). Participants will receive these treatments to evaluate their combined effect on managing cGVHD symptoms. The study will focus on individuals who require systemic therapy for moderate to severe cGVHD following an allogeneic stem cell transplant. The goal is to determine how well this combination therapy works compared to existing treatments.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 12 and older with newly diagnosed moderate or severe cGVHD requiring systemic therapy.

Not a fit: Patients who have received more than one prior allogeneic stem cell transplant or those with overlap cGVHD may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new effective option for patients suffering from chronic graft-versus-host disease.

How similar studies have performed: Other studies have shown promise in treating cGVHD with similar approaches, but this specific combination is being evaluated for the first time.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* ≥ 12 years of age at the time of informed consent.
* New-onset moderate or severe cGVHD, as defined by the 2014 NIH Consensus Development Project Criteria for Clinical Trials in cGVHD, requiring systemic therapy.
* History of 1 allo-SCT (any type of stem cell donor, any conditioning regimen, and source of hematopoietic stem cells).
* Adequate hematologic function independent of platelet transfusion and growth factors for at least 7 days prior to study entry: ANC ≥ 0.75 × 109/L and platelet count ≥ 20 × 109/L.
* Willingness to avoid pregnancy or fathering children.

Exclusion Criteria:

* Received more than 1 prior allo-SCT. Prior autologous HCT is allowed.
* Has overlap cGVHD, defined as simultaneous presence of features or characteristics of aGVHD in a patient with cGVHD.
* Received previous systemic treatment for cGVHD, including systemic corticosteroids and extracorporeal photopheresis.
* Received systemic corticosteroids within 2 weeks prior to C1D1, regardless of indication.
* Initiated systemic treatment with CNIs or mTOR inhibitors within 2 weeks prior to C1D1.
* Prior treatment with a JAK inhibitor within 8 weeks before randomization. Participants who received a JAK inhibitor for the treatment of aGVHD are eligible only if they achieved a response (CR or PR) to JAK inhibitor treatment and did not discontinue due to toxicity.
* Evidence of relapse of the primary hematologic disease or treatment for relapse after the allo-SCT was performed, including DLIs for the treatment of molecular relapse.
* History of acute or chronic pancreatitis.
* History of thromboembolic events (such as deep vein thrombosis, pulmonary embolism, stroke, myocardial infarction) in the 6 months prior to study entry.
* Active symptomatic myositis.
* Severe renal impairment, that is, estimated CrCl \< 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. Participants with CrCl of 30 to 59 mL/min on treatment with fluconazole are not eligible.
* 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.
* Currently active significant cardiac disease, such as uncontrolled arrhythmias, uncontrolled hypertension, or Class 3 or 4 congestive heart failure as defined by New York Heart Association, or a history of myocardial infarction or unstable angina within 6 months prior to randomization.
* Pregnant or breastfeeding.

Other protocol-defined Inclusion/Exclusion Criteria may apply.

Where this trial is running

Phoenix, Arizona and 69 other locations

+20 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.