Recruiting
Phase 2

Axatilimab vs. Best Available Therapy

Sponsor:

Incyte Corporation

Code:

NCT07124078

Conditions

Chronic Graft-versus-host-disease

Eligibility Criteria

Sex: All

Age: 2 - 17

Healthy Volunteers: Not accepted

Interventions

INCA034176

Best available Treatment (BAT)

Study Details

Brief summary:

This study will be conducted to compare Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy.

Conditions

Chronic Graft-versus-host-disease

Study ID

NCT07124078

Start date

May 20, 2026

Status verified date

Aug, 2026

Completion date

Jul 31, 2029

Anticipated

Primary completion date

Jan 31, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2 - 17

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

60 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Axatilimab

Axatilimab at the protocol-defined dose.

experimental: Best available Treatment (BAT)

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.

Interventions

INCA034176

Axatilimab at the protocol-defined dose.

Best available Treatment (BAT)

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.

Primary outcome measure

  • Objective Response (OR) at 6 months [ Time Frame: 6 months ]
  • United States (US): Best overall Response (BOR) [ Time Frame: Up to 6 months ]

Central Contacts and Locations

Central contacts

Incyte Corporation Call Center (US)

1.855.463.3463medinfo@incyte.com

Incyte Corporation Call Center (ex-US)

+800 00027423eumedinfo@incyte.com

Locations

City of Hope Medical Center

Recruiting

Duarte, California, United States, 91010

Childrens National Medical Center

Recruiting

Washington D.C., District of Columbia, United States, 20010

Childrens Healthcare of Atlanta

Recruiting

Atlanta, Georgia, United States, 30329

Memorial Sloan Kettering Cancer Center

Recruiting

New York, New York, United States, 10065

Levine Cancer Institute

Recruiting

Charlotte, North Carolina, United States, 28203

More Information

Sponsor

Incyte Corporation

Last update posted

Aug 25, 2026

Last verified

Aug, 2026

Keywords

  • cGVHD

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Incyte Corporation on 2026-08-25.