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NCT Number: NCT07124078

A Study to Evaluate 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)

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.

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Key information

Conditions

Chronic Graft-versus-host-disease Bronchial Diseases Bronchiolitis Bronchiolitis Obliterans Bronchiolitis Obliterans Syndrome Bronchitis Graft vs Host Disease Immune System Diseases Lung Diseases Lung Diseases, Obstructive Organizing Pneumonia Respiratory Tract Diseases

Age range

2 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Hôpital Universitaire Des Enfants Reine Fabiola, Brussels, Belgium

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Aged ≥ 2 to < 18 years at the time of signing the informed consent.
  • 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, or ibrutinib.
  • 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.
  • Evidence of relapse of hematologic disease or treatment for relapse after the allo-SCT was performed, including DLI for the treatment of molecular relapse. Note: Participants who have received a scheduled DLI as part of their transplant procedure and not for management of malignancy 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 and/or ALT and 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.

Treatment and study plan

INCA034176

Drug

Axatilimab at the protocol-defined dose.

Other names: Axatilimab

Best Available Treatment (BAT)

Drug

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.

Other names: BAT could include:, • Calcineurin inhibitor (cyclosporine or tacrolimus), • Extracorporeal photopheresis (ECP), • Mycophenolate mofetil (MMF), • A mammalian target of rapamycin (mTOR) inhibitor (everolimus or sirolimus), • Rituximab, • Imatinib, • Methotrexate, • Ibrutinib

Primary outcomes

  1. Objective Response (OR) at 6 months

    Time frame: 6 months

    Defined for each treatment group as complete response (CR) or partial response (PR) at 6 months (Cycle 7 Day 1, 28-day cycles) in the absence of new systemic therapy for cGVHD. Responses defined by the 2014 NIH consensus criteria.

Secondary outcomes

  1. Pharmacokinetics Parameter (PK): Cmax of axatilimab

    Time frame: Up to 5 years

    Defined as maximum observed plasma concentration of axatilimab.

  2. Pharmacokinetics Parameter: Tmax of axatilimab

    Time frame: Up to 5 years

    Defined as the time to reach the maximum plasma concentration of axatilimab.

  3. Pharmacokinetics Parameter: Cmin of axatilimab

    Time frame: Up to 5 years

    Defined as the minimum observed plasma concentration of axatilimab.

  4. Pharmacokinetics Parameter: AUC(0-t) of axatilimab

    Time frame: Up to 5 years

    Defined as the area under the concentration-time curve up to the last measurable concentration of axatilimab.

  5. Pharmacokinetics Parameter: AUC 0-∞ of axatilimab

    Time frame: Up to 5 years

    Defined as the area under the concentration-time curve from 0 to infinity of axatilimab.

  6. Pharmacokinetics Parameter: CL of axatilimab

    Time frame: Up to 5 years

    Defined as the apparent oral dose clearance of axatilimab.

  7. Pharmacokinetics Parameter: Vz of axatilimab

    Time frame: Up to 5 years

    Defined as the apparent oral dose volume of distribution of axatilimab.

  8. Pharmacokinetics Parameter: t1/2 of axatilimab

    Time frame: Up to 5 years

    Defined as the apparent terminal phase disposition half-life of axatilimab.

  9. Best Overall Response (BOR)

    Time frame: Up to 5 years

    Defined as best response of CR or PR in the first 6 months (up to and including Cycle 7 Day 1), and at any timepoint up to the initiation of new systemic therapy for cGVHD.

  10. Overall Response at 12 months

    Time frame: 12 months

    Defined as CR or PR at 12 months in the absence of new systemic therapy for cGVHD.

  11. Duration of Response (DOR) (in responders only)

    Time frame: Up to 5 years

    Defined as the time from the date of first response (PR or CR) to the date of progression of cGVHD, initiation of new systemic treatment for cGVHD, or death from any cause, whichever comes first. An additional measure of response durability will consider DOR as the time from the date of first response to the date of new systemic therapy for cGVHD or death from any cause, whichever occurs first.

  12. Organ-specific response

    Time frame: Up to 5 years

    Organ-specific response as defined in the protocol.

  13. Percent reduction in daily corticosteroid dose at 6 months

    Time frame: 6 months

    Defined as Percent reduction in daily corticosteroid dose at Cycle 7 Day 1, and participants successfully tapered off all corticosteroids at Cycle 7 Day 1.

  14. Changes in parameters collected using the pediatric stem cell quality of life (Q0L) questionnaire (PedsQL Stem Cell Transplant Module)

    Time frame: Up to 5 years

  15. Number of participants with Treatment-emergent Adverse Events (TEAEs)

    Time frame: Up to 5 years and 30 days

    Defined as adverse events reported for the first time or worsening of a pre-existing event after the first dose of study treatment.

Study contacts

Contact information is provided by the study sponsor or research team.

Incyte Corporation Call Center (US)

CONTACT

[email protected]

1.855.463.3463

Incyte Corporation Call Center (ex-US)

CONTACT

[email protected]

+800 00027423

Sponsors and collaborators

Lead sponsor

Incyte Corporation

Industry

Registry information

Official study title

A 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)

Important dates

Study start
2026
Primary completion
2029
Study completion
2029
First posted
Aug 15, 2025
Registry last updated
Jun 2, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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