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)

NCT07124078 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: INCA034176-256
• Study Type: interventional
• Target: 60
• Locations: 6 countries
• Sites: 41

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

Keywords

cGVHD

Outcome Measures

Primary Outcomes (1)

• Objective Response (OR) at 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.

  6 months

Secondary Outcomes (15)

• Pharmacokinetics Parameter (PK): Cmax of axatilimab

  Up to 5 years

• Pharmacokinetics Parameter: Tmax of axatilimab

  Up to 5 years

• Pharmacokinetics Parameter: Cmin of axatilimab

  Up to 5 years

• Pharmacokinetics Parameter: AUC(0-t) of axatilimab

  Up to 5 years

• Pharmacokinetics Parameter: AUC 0-∞ of axatilimab

  Up to 5 years

Study Arms (2)

Axatilimab

EXPERIMENTAL

Axatilimab at the protocol-defined dose.

Drug: INCA034176

Best available Treatment (BAT)

EXPERIMENTAL

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.

Drug: Best available Treatment (BAT)

Interventions

INCA034176 DRUG

Axatilimab at the protocol-defined dose.

Also known as: Axatilimab

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.

Also known as: 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

Best available Treatment (BAT)

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Incyte Corporation: lead

Study Sites (41)

City of Hope Medical Center

Duarte, California, 91010, United States

RECRUITING

Children'S Hospital Los Angeles Specialt

Los Angeles, California, 90027, United States

NOT YET RECRUITING

Lucile Packard Child Hospital End

Palo Alto, California, 94304, United States

NOT YET RECRUITING

Childrens National Medical Center

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

RECRUITING

Adventhealth Pediatric Cellular Therapy and Blood and Marrow Transplant

Orlando, Florida, 32804, United States

NOT YET RECRUITING

Childrens Healthcare of Atlanta

Atlanta, Georgia, 30329, United States

NOT YET RECRUITING

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

RECRUITING

Levine Cancer Institute

Charlotte, North Carolina, 28203, United States

RECRUITING

Duke Cancer Center

Durham, North Carolina, 27710, United States

NOT YET RECRUITING

Childrens Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

NOT YET RECRUITING

Fred Hutchinson Cancer Research Center

Seattle, Washington, 98109, United States

NOT YET RECRUITING

Hôpital Universitaire Des Enfants Reine Fabiola

Brussels, Belgium

RECRUITING

Universite Catholique de Louvain (Ucl) - Cliniques Universitaires Saint-Luc

Woluwe-Saint-Lambert, 01200, Belgium

RECRUITING

Charite Campus Virchow

Berlin, 13353, Germany

NOT YET RECRUITING

Klinikum Der Johann Wolfgang Goethe-Universitaet

Frankfurt am Main, 60590, Germany

RECRUITING

Universitaetsklinikum Freiburg Zentrum Fuer Kinderheilkunde- Und Jugendmedizin Klinik Iv: Paediatris

Freiburg im Breisgau, 79106, Germany

NOT YET RECRUITING

Universitaetsklinikum Hamburg-Eppendorf

Hamburg, 20246, Germany

NOT YET RECRUITING

Medizinische Hochschule Hannover (Mhh) - Zentrum Fuer Kinderheilkunde Und Jugendmedizin - Klinik Fue

Hanover, 30625, Germany

NOT YET RECRUITING

University Hospital Regensburg

Regensburg, 93053, Germany

NOT YET RECRUITING

Irccs - Aou Di Bologna - Sant'Orsola Malpighi

Bologna, 40138, Italy

NOT YET RECRUITING

Irccs Istituto G. Gaslini, Universita Di Genova

Genova, 16147, Italy

RECRUITING

Fondazione Irccs San Gerardo Dei Tintori

Monza, 20900, Italy

NOT YET RECRUITING

Azienda Ospedaliera Universitaria Di Padova

Padua, 35128, Italy

RECRUITING

Fondazione Irccs Policlinico San Matteo Di Pavia Oncoematologia Pediatrica, Universita Di Pavia

Pavia, 27100, Italy

RECRUITING

Irccs Ospedale Pediatrico Bambino Gesu

Rome, 00165, Italy

RECRUITING

Hospital Universitario Vall D'Hebron

Barcelona, 08035, Spain

RECRUITING

Hospital Sant Joan de Deu Barcelona - Children'S Hospital

Esplugues de Llobregat, 08950, Spain

NOT YET RECRUITING

Hospital Infantil Unversitario Nino Jesus

Madrid, 28009, Spain

NOT YET RECRUITING

Hospital Universitario La Paz

Madrid, 28046, Spain

RECRUITING

Hospital Regional Universitario de Malaga- Hospital Materno Infantil

Málaga, 29011, Spain

RECRUITING

Hospital Universitario Virgen de La Arrixaca

Murcia, 30120, Spain

RECRUITING

Universitat de Valencia - Hospital Universitari I Politecnic La Fe de Valencia (Hospital La Fe Bulev

Valencia, 46026, Spain

RECRUITING

Birmingham Children'S Hospital - Birmingham Women'S and Children'S Nhs Foundation Trust

Birmingham, B4 6NH, United Kingdom

RECRUITING

Addenbrooke'S Hospital - Cambridge University Hospitals Nhs Foundation Trust

Cambridge, CB2 0QQ, United Kingdom

RECRUITING

The Royal Hospital For Children - Glasgow Health Board

Glasgow, G51 4TF, United Kingdom

RECRUITING

Leeds Childrens Hospital

Leeds, LS1 3EX, United Kingdom

RECRUITING

St Mary'S Hospital, Paddington - Imperial College Healthcare Nhs Trust

London, W2 1NY, United Kingdom

RECRUITING

Great Ormond Street Hospital Nhs Trust

London, WC1N 3JH, United Kingdom

RECRUITING

Royal Manchester Children'S Hospital - Manchester University Nhs Foundation Trust

Manchester, M139WL, United Kingdom

RECRUITING

Royal Victoria Infirmary - the Newcastle Upon Tyne Hospitals Nhs Foundation Trust

Newcastle, NE1 4LP, United Kingdom

RECRUITING

Sheffield Children'S Hospital - Sheffield Children'S Nhs Foundation Trust

Sheffield, S10 2TH, United Kingdom

RECRUITING

Related Links

• 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)

MeSH Terms

Conditions

Bronchiolitis Obliterans Syndrome

Interventions

axatilimab; Calcineurin Inhibitors; Cyclosporine; Tacrolimus; Photopheresis; Mycophenolic Acid; Everolimus; Sirolimus; Rituximab; Imatinib Mesylate; Methotrexate; ibrutinib

Condition Hierarchy (Ancestors)

Organizing Pneumonia; Bronchiolitis Obliterans; Bronchiolitis; Bronchitis; Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Graft vs Host Disease; Immune System Diseases

Intervention Hierarchy (Ancestors)

Enzyme Inhibitors; Molecular Mechanisms of Pharmacological Action; Pharmacologic Actions; Chemical Actions and Uses; Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins; Macrolides; Lactones; Organic Chemicals; PUVA Therapy; Ultraviolet Therapy; Phototherapy; Therapeutics; Extracorporeal Circulation; Surgical Procedures, Operative; Caproates; Acids, Acyclic; Carboxylic Acids; Fatty Acids; Lipids; Antibodies, Monoclonal, Murine-Derived; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Serum Globulins; Globulins; Benzamides; Amides; Benzoates; Acids, Carbocyclic; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Piperazines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Pyrimidines; Aminopterin; Pterins; Pteridines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Incyte Medical Monitor

  Incyte Corporation

  STUDY DIRECTOR

Central Study Contacts

Incyte Corporation Call Center (US)

CONTACT

1.855.463.3463; medinfo@incyte.com

Incyte Corporation Call Center (ex-US)

CONTACT

+800 00027423; eumedinfo@incyte.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 8, 2025
• First Posted: August 15, 2025
• Study Start: May 20, 2026
• Primary Completion (Estimated): January 31, 2029
• Study Completion (Estimated): July 31, 2029
• Last Updated: June 2, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: Data will be shared after the primary publication or 2 years after the study has ended for market authorized products and indications.
• Access Criteria: Data from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.incyteclinicaltrials.com website. For approved requests, the researchers will be granted access to anonymized data under the terms of a data sharing agreement.

Locations

IT (6); BE (2); ES (7); GB (9); DE (6); US (11)