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

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* 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 Criteria:

* 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.

Other protocol-defined Inclusion/Exclusion Criteria may apply.

Where it's running

• City of Hope Medical Center
  Duarte, California, United States Recruiting
• Lucile Packard Child Hospital End
  Palo Alto, California, United States Recruiting
• Childrens National Medical Center
  Washington D.C., District of Columbia, United States Recruiting
• Childrens Healthcare of Atlanta
  Atlanta, Georgia, United States Recruiting
• Memorial Sloan Kettering Cancer Center
  New York, New York, United States Recruiting
• Levine Cancer Institute
  Charlotte, North Carolina, United States Recruiting

Study contacts

• Study coordinator: Incyte Corporation Call Center (US)
• Email: medinfo@incyte.com
• Phone: 1.855.463.3463

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.