Actively Recruiting
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)
Led by Incyte Corporation · Updated on 2026-05-07
60
Participants Needed
40
Research Sites
165 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
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)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Aged 63 2 to < 18 years at the time of signing the informed consent.
- Active, moderate to severe chronic graft-versus-host disease requiring systemic immune suppression.
- Refractory or recurrent chronic graft-versus-host disease with at least 2 prior systemic therapies including corticosteroids and ruxolitinib.
- Stable dose of systemic corticosteroids for at least 2 weeks prior to treatment start; topical and inhaled corticosteroids allowed.
- Must agree to be treated with one of the specified best available therapy options on treatment day 1.
- History of allogeneic hematopoietic cell transplantation from any donor type with any graft source and conditioning regimen.
You will not qualify if you...
- More than 1 prior allogeneic hematopoietic cell transplant (prior autologous transplant allowed).
- Evidence of relapse of hematologic disease or treatment for relapse after allo-SCT, including donor lymphocyte infusion for relapse.
- Systemic treatment with calcineurin inhibitors or mTOR inhibitors started within 2 weeks prior to treatment start.
- Severe renal impairment with GFR < 30 mL/min/1.73 m2 or end-stage renal disease on dialysis.
- Impaired liver function defined as total bilirubin > 1.5 times upper limit of normal and/or ALT and AST > 3 times upper limit of normal without liver cGVHD.
- History of acute or chronic pancreatitis.
- Active, symptomatic myositis.
- Female adolescents who are pregnant or breastfeeding.
- Other protocol-defined criteria may apply.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 40 locations
1
City of Hope Medical Center
Duarte, California, United States, 91010
Actively Recruiting
2
Children'S Hospital Los Angeles Specialt
Los Angeles, California, United States, 90027
Not Yet Recruiting
3
Lucile Packard Child Hospital End
Palo Alto, California, United States, 94304
Not Yet Recruiting
4
Childrens National Medical Center
Washington D.C., District of Columbia, United States, 20010
Not Yet Recruiting
5
Adventhealth Pediatric Cellular Therapy and Blood and Marrow Transplant
Orlando, Florida, United States, 32804
Not Yet Recruiting
6
Childrens Healthcare of Atlanta
Atlanta, Georgia, United States, 30329
Not Yet Recruiting
7
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10065
Actively Recruiting
8
Levine Cancer Institute
Charlotte, North Carolina, United States, 28203
Actively Recruiting
9
Duke Cancer Center
Durham, North Carolina, United States, 27710
Not Yet Recruiting
10
Childrens Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
Not Yet Recruiting
11
Fred Hutchinson Cancer Research Center
Seattle, Washington, United States, 98109
Not Yet Recruiting
12
Hôpital Universitaire Des Enfants Reine Fabiola
Brussels, Belgium
Actively Recruiting
13
Universite Catholique de Louvain (Ucl) - Cliniques Universitaires Saint-Luc
Woluwe-Saint-Lambert, Belgium, 01200
Actively Recruiting
14
Charite Campus Virchow
Berlin, Germany, 13353
Not Yet Recruiting
15
Klinikum Der Johann Wolfgang Goethe-Universitaet
Frankfurt am Main, Germany, 60590
Actively Recruiting
16
Universitaetsklinikum Freiburg Zentrum Fuer Kinderheilkunde- Und Jugendmedizin Klinik Iv: Paediatris
Freiburg im Breisgau, Germany, 79106
Not Yet Recruiting
17
Universitaetsklinikum Hamburg-Eppendorf
Hamburg, Germany, 20246
Not Yet Recruiting
18
Medizinische Hochschule Hannover (Mhh) - Zentrum Fuer Kinderheilkunde Und Jugendmedizin - Klinik Fue
Hanover, Germany, 30625
Not Yet Recruiting
19
University Hospital Regensburg
Regensburg, Germany, 93053
Not Yet Recruiting
20
Irccs - Aou Di Bologna - Sant'Orsola Malpighi
Bologna, Italy, 40138
Not Yet Recruiting
21
Irccs Istituto G. Gaslini, Universita Di Genova
Genova, Italy, 16147
Not Yet Recruiting
22
Fondazione Irccs San Gerardo Dei Tintori
Monza, Italy, 20900
Not Yet Recruiting
23
Azienda Ospedaliera Universitaria Di Padova
Padua, Italy, 35128
Actively Recruiting
24
Fondazione Irccs Policlinico San Matteo Di Pavia Oncoematologia Pediatrica, Universita Di Pavia
Pavia, Italy, 27100
Not Yet Recruiting
25
Irccs Ospedale Pediatrico Bambino Gesu
Rome, Italy, 00165
Actively Recruiting
26
Hospital Universitario Vall D'Hebron
Barcelona, Spain, 08035
Actively Recruiting
27
Hospital Infantil Unversitario Nino Jesus
Madrid, Spain, 28009
Not Yet Recruiting
28
Hospital Universitario La Paz
Madrid, Spain, 28046
Actively Recruiting
29
Hospital Regional Universitario de Malaga- Hospital Materno Infantil
Málaga, Spain, 29011
Actively Recruiting
30
Hospital Universitario Virgen de La Arrixaca
Murcia, Spain, 30120
Actively Recruiting
31
Universitat de Valencia - Hospital Universitari I Politecnic La Fe de Valencia (Hospital La Fe Bulev
Valencia, Spain, 46026
Actively Recruiting
32
Birmingham Children'S Hospital - Birmingham Women'S and Children'S Nhs Foundation Trust
Birmingham, United Kingdom, B4 6NH
Actively Recruiting
33
Addenbrooke'S Hospital - Cambridge University Hospitals Nhs Foundation Trust
Cambridge, United Kingdom, CB2 0QQ
Actively Recruiting
34
The Royal Hospital For Children - Glasgow Health Board
Glasgow, United Kingdom, G51 4TF
Actively Recruiting
35
Leeds Childrens Hospital
Leeds, United Kingdom, LS1 3EX
Actively Recruiting
36
St Mary'S Hospital, Paddington - Imperial College Healthcare Nhs Trust
London, United Kingdom, W2 1NY
Actively Recruiting
37
Great Ormond Street Hospital Nhs Trust
London, United Kingdom, WC1N 3JH
Actively Recruiting
38
Royal Manchester Children'S Hospital - Manchester University Nhs Foundation Trust
Manchester, United Kingdom, M139WL
Actively Recruiting
39
Royal Victoria Infirmary - the Newcastle Upon Tyne Hospitals Nhs Foundation Trust
Newcastle, United Kingdom, NE1 4LP
Actively Recruiting
40
Sheffield Children'S Hospital - Sheffield Children'S Nhs Foundation Trust
Sheffield, United Kingdom, S10 2TH
Actively Recruiting
Research Team
I
Incyte Corporation Call Center (US)
CONTACT
I
Incyte Corporation Call Center (ex-US)
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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