Actively Recruiting
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)
Led by Incyte Corporation · Updated on 2026-06-02
60
Participants Needed
41
Research Sites
25 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying pediatric participants aged 2 to under 18 years who have chronic graft-versus-host disease (cGVHD) that is moderate to severe and requires systemic immune suppression. The study compares the drug axatilimab to the best available therapy (BAT) after participants have already received at least two prior systemic treatments, including corticosteroids and ruxolitinib. This phase 2 trial aims to evaluate the response to these treatments in children and teens with this condition. Participants will be randomly assigned to receive either axatilimab at a protocol-defined dose or the best available therapy chosen by the investigator. The BAT options include specific drugs such as cyclosporine, tacrolimus, extracorporeal photopheresis, mycophenolate mofetil, mTOR inhibitors, rituximab, imatinib, methotrexate, or ibrutinib, excluding experimental agents. Both treatment groups will be monitored over time to assess responses and side effects. During the study, participants will be evaluated for objective response at 6 months as the primary outcome, with additional assessments up to 5 years including drug pharmacokinetics, overall response at 12 months, duration of response, organ-specific responses, and quality of life changes. Safety will be monitored by tracking treatment-emergent adverse events. The total study duration allows for long-term follow-up to better understand the effects and potential benefits of the treatments.
CONDITIONS
Brief 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 2 to less than 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 after 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
- Willing to receive one of the specified best available therapy options on treatment start
- History of allogeneic hematopoietic stem cell transplant from any donor HLA type using any graft source and conditioning regimen
You will not qualify if you...
- More than 1 prior allogeneic hematopoietic stem cell transplant (prior autologous transplant allowed)
- Evidence of relapse of hematologic disease or treatment for relapse after transplant, excluding scheduled donor lymphocyte infusion not for malignancy relapse
- Systemic treatment with calcineurin inhibitors or mTOR inhibitors started within 2 weeks prior to treatment start
- Severe kidney impairment with GFR less than 30 mL/min/1.73 m2 or end-stage renal disease on dialysis
- Impaired liver function defined by elevated bilirubin or liver enzymes without liver chronic graft-versus-host disease
- History of acute or chronic pancreatitis
- Active, symptomatic muscle inflammation (myositis)
- Female adolescents who are pregnant or breastfeeding
- Other protocol-defined inclusion/exclusion criteria may apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 6 months
Participants receive either Axatilimab at the protocol-defined dose or the best available therapy selected by the investigator to treat chronic graft-versus-host disease.
Regular visits during treatment period as per protocol
Duration - Up to 5 years
Participants are monitored for response, safety, and long-term outcomes after treatment ends.
Periodic visits up to 5 years for assessments
Trial Site Locations
Total: 41 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
Actively 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
Actively 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
Actively 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 Sant Joan de Deu Barcelona - Children'S Hospital
Esplugues de Llobregat, Spain, 08950
Not Yet Recruiting
28
Hospital Infantil Unversitario Nino Jesus
Madrid, Spain, 28009
Not Yet Recruiting
29
Hospital Universitario La Paz
Madrid, Spain, 28046
Actively Recruiting
30
Hospital Regional Universitario de Malaga- Hospital Materno Infantil
Málaga, Spain, 29011
Actively Recruiting
31
Hospital Universitario Virgen de La Arrixaca
Murcia, Spain, 30120
Actively Recruiting
32
Universitat de Valencia - Hospital Universitari I Politecnic La Fe de Valencia (Hospital La Fe Bulev
Valencia, Spain, 46026
Actively Recruiting
33
Birmingham Children'S Hospital - Birmingham Women'S and Children'S Nhs Foundation Trust
Birmingham, United Kingdom, B4 6NH
Actively Recruiting
34
Addenbrooke'S Hospital - Cambridge University Hospitals Nhs Foundation Trust
Cambridge, United Kingdom, CB2 0QQ
Actively Recruiting
35
The Royal Hospital For Children - Glasgow Health Board
Glasgow, United Kingdom, G51 4TF
Actively Recruiting
36
Leeds Childrens Hospital
Leeds, United Kingdom, LS1 3EX
Actively Recruiting
37
St Mary'S Hospital, Paddington - Imperial College Healthcare Nhs Trust
London, United Kingdom, W2 1NY
Actively Recruiting
38
Great Ormond Street Hospital Nhs Trust
London, United Kingdom, WC1N 3JH
Actively Recruiting
39
Royal Manchester Children'S Hospital - Manchester University Nhs Foundation Trust
Manchester, United Kingdom, M139WL
Actively Recruiting
40
Royal Victoria Infirmary - the Newcastle Upon Tyne Hospitals Nhs Foundation Trust
Newcastle, United Kingdom, NE1 4LP
Actively Recruiting
41
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)
I
Incyte Corporation Call Center (ex-US)
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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