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Clinical Trials in the USA / NCT07124078
Recruiting Phase 2

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 · tracked via the Priya Life Science USA tracker
Sponsor
Incyte Corporation
Phase
Phase 2
Started
2026-05-20
Last updated
2026-09-15

Condition(s) studied

Chronic Graft-versus-host-disease

Investigational drug(s) / intervention(s)

INCA034176Best available Treatment (BAT)

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.

Study 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.

Eligibility

Sex
ALL
Min age
2 Years
Max age
17 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (41)

FacilityCityRegionStatus
City of Hope Medical Center Duarte California Recruiting
Children'S Hospital Los Angeles Specialt Los Angeles California Not Yet Recruiting
Lucile Packard Child Hospital End Palo Alto California Recruiting
Childrens National Medical Center Washington D.C. District of Columbia Recruiting
Adventhealth Pediatric Cellular Therapy and Blood and Marrow Transplant Orlando Florida Not Yet Recruiting
Childrens Healthcare of Atlanta Atlanta Georgia Recruiting
Memorial Sloan Kettering Cancer Center New York New York Recruiting
Levine Cancer Institute Charlotte North Carolina Recruiting
Duke Cancer Center Durham North Carolina Not Yet Recruiting
Childrens Hospital of Pittsburgh Pittsburgh Pennsylvania Not Yet Recruiting
Fred Hutchinson Cancer Research Center Seattle Washington Recruiting
Hôpital Universitaire Des Enfants Reine Fabiola Brussels Belgium Recruiting
Universite Catholique de Louvain (Ucl) - Cliniques Universitaires Saint-Luc Woluwe-Saint-Lambert Belgium Recruiting
Charite Campus Virchow Berlin Germany Recruiting
Klinikum Der Johann Wolfgang Goethe-Universitaet Frankfurt am Main Germany Recruiting
Universitaetsklinikum Freiburg Kinder- Und Jugendklinik (Kjk) Freiburg im Breisgau Germany Recruiting
Universitaetsklinikum Hamburg-Eppendorf Hamburg Germany Recruiting
Medizinische Hochschule Hannover (Mhh) - Zentrum Fuer Kinderheilkunde Und Jugendmedizin - Klinik Fue Hanover Germany Not Yet Recruiting
University Hospital Regensburg Regensburg Germany Not Yet Recruiting
Irccs - Aou Di Bologna - Sant'Orsola Malpighi Bologna Italy Recruiting
Irccs Istituto G. Gaslini, Universita Di Genova Genova Italy Recruiting
Fondazione Irccs San Gerardo Dei Tintori Monza Italy Recruiting
Azienda Ospedaliera Universitaria Di Padova Padua Italy Recruiting
Fondazione Irccs Policlinico San Matteo Di Pavia Oncoematologia Pediatrica, Universita Di Pavia Pavia Italy Recruiting
Irccs Ospedale Pediatrico Bambino Gesu Rome Italy Recruiting
Hospital Universitario Vall D'Hebron Barcelona Spain Recruiting
Hospital Sant Joan de Deu Barcelona - Children'S Hospital Esplugues de Llobregat Spain Not Yet Recruiting
Hospital Infantil Unversitario Nino Jesus Madrid Spain Not Yet Recruiting
Hospital Universitario La Paz Madrid Spain Recruiting
Hospital Regional Universitario de Malaga- Hospital Materno Infantil Málaga Spain Recruiting
Hospital Universitario Virgen de La Arrixaca Murcia Spain Recruiting
Universitat de Valencia - Hospital Universitari I Politecnic La Fe de Valencia (Hospital La Fe Bulev Valencia Spain Recruiting
Birmingham Children'S Hospital - Birmingham Women'S and Children'S Nhs Foundation Trust Birmingham United Kingdom Recruiting
Addenbrooke'S Hospital - Cambridge University Hospitals Nhs Foundation Trust Cambridge United Kingdom Recruiting
The Royal Hospital For Children - Glasgow Health Board Glasgow United Kingdom Recruiting
Leeds Childrens Hospital Leeds United Kingdom Recruiting
St Mary'S Hospital, Paddington - Imperial College Healthcare Nhs Trust London United Kingdom Recruiting
Great Ormond Street Hospital Nhs Trust London United Kingdom Recruiting
Royal Manchester Children'S Hospital - Manchester University Nhs Foundation Trust Manchester United Kingdom Recruiting
Royal Victoria Infirmary - the Newcastle Upon Tyne Hospitals Nhs Foundation Trust Newcastle United Kingdom Recruiting

+ 1 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07124078 on ClinicalTrials.gov ↗ ← All trials in the USA