Chronic Graft-versus-host-diseasePhase 3AXemplify-357NCT06585774
A Study to Evaluate Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease
A Phase 3 study testing INCA034176 and Corticosteroids in people with Chronic Graft-versus-host-disease, run by Incyte Corporation at 131 sites in 13 countries.
Trial summary
This study will be conducted to compare the efficacy of axatilimab versus placebo in combination with corticosteroids as initial treatment for moderate or severe chronic graft-versus-host disease (cGVHD). Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Chronic Graft-versus-host-disease
- What is being tested?
- INCA034176 and Corticosteroids
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 240 participants
- Where
- 131 sitesin 13 countries
- Study dates
- 21 Jan 2025to 31 Mar 2030
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- ≥ 12 years of age at the time of informed consent.
- New-onset moderate or severe cGVHD, as defined by the 2014 NIH Consensus Development Project Criteria for Clinical Trials in cGVHD, requiring systemic therapy.
- 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.
- Adequate hematologic function with ANC ≥ 0.5 × 109/L independent of growth factors for at least 7 days prior to study entry.
- Willingness to avoid pregnancy or fathering children.
Who cannot join
- Received more than 1 prior allo-HCT. Prior autologous HCT is allowed.
- Has overlap cGVHD, defined as simultaneous presence of features or characteristics of aGVHD in a patient with cGVHD.
- Received more than 7 days of systemic corticosteroid treatment for cGVHD or unable to begin a prednisone dose ≥ 1.0 mg/kg per day (or methylprednisolone equivalent) for cGVHD.
- Received previous systemic treatment for cGVHD, including extracorporeal photopheresis.
- Systemic treatment with CNIs or mTOR inhibitors started within 2 weeks prior to C1D1.
- Prior treatment with CSF-1R targeted therapies.
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- Active, uncontrolled bacterial, fungal, parasitic, or viral infection.
- Evidence of relapse of the primary hematologic disease or treatment for relapse after the allo-HCT was performed, including DLIs for the treatment of molecular relapse.
- History of acute or chronic pancreatitis.
- Active symptomatic myositis.
- History or current diagnosis of cardiac disease indicating significant risk of safety for participation in the study, such as uncontrolled or significant cardiac disease.
- Severe renal impairment, that is, estimated CrCl < 30 mL/min measured or calculated by Cockcroft-Gault equation in adults and Schwartz formula in pediatric participants, or endstage 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.
- Pregnant or breastfeeding.
- Other protocol-defined Inclusion/Exclusion Criteria may apply.
Trial locations and contacts
131 sites in 13 countries. Contact the study team or a site near you to ask about taking part.
131 sites in 13 countries
United States37 sites, 37 recruiting
- Recruiting
University of Michigan
Ann Arbor, Michigan, 48109
- Recruiting
Emory University-Winship Cancer Institute
Atlanta, Georgia, 30322
- Recruiting
St David'S South Austin Medical Center
Austin, Texas, 78704
- Recruiting
John Hopkins Hospital
Baltimore, Maryland, 21287
- Recruiting
University of Alabama Birmingham
Birmingham, Alabama, 35294
- Recruiting
Massachusetts General Hospital
Boston, Massachusetts, 02114
- Recruiting
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
- Recruiting
Medical University of South Carolina
Charleston, South Carolina, 29425
- Recruiting
Northwestern Memorial Hospital
Chicago, Illinois, 60611
- Recruiting
University of Illinois
Chicago, Illinois, 60612
- Recruiting
Colorado Blood Cancer Institute
Denver, Colorado, 80218
- Recruiting
Henry Ford Hospital
Detroit, Michigan, 48202
- Recruiting
City of Hope Medical Center
Duarte, California, 91010
- Recruiting
Corewell Health Hematology Oncology
Grand Rapids, Michigan, 49503
- Recruiting
Prisma Health Upstate
Greenville, South Carolina, 29615
- Recruiting
Hackensack University Medical Center
Hackensack, New Jersey, 07601
- Recruiting
The University of Kansas Cancer Center
Kansas City, Kansas, 66160
- Recruiting
University of California San Diego Medical Center, Moores Cancer Center
La Jolla, California, 92037
- Recruiting
University of Southern California
Los Angeles, California, 90089
- Recruiting
Baptist Cancer Center
Memphis, Tennessee, 38120
- Recruiting
Miami Cancer Institute
Miami, Florida, 33176
- Recruiting
West Virginia University Cancer Institute
Morgantown, West Virginia, 26506
- Recruiting
Rutgers Cancer Institute of Nj
New Brunswick, New Jersey, 08903
- Recruiting
Orlando Health Cancer Institute Downtown Orlando
Orlando, Florida, 32806
- Recruiting
Memorial Cancer Institute
Pembroke Pines, Florida, 33026
- Recruiting
Jefferson University Hospitals
Philadelphia, Pennsylvania, 19107
- Recruiting
Upmc Hillman Cancer Center
Pittsburgh, Pennsylvania, 15232
- Recruiting
Oregon Health and Science University
Portland, Oregon, 97239
- Recruiting
Virginia Commonwealth University Massey Cancer Center
Richmond, Virginia, 23298
- Recruiting
University of Rochester Medical Center
Rochester, New York, 14642
- Recruiting
Intermountain Blood and Marrow Transplant
Salt Lake City, Utah, 84143
- Recruiting
Texas Transplant Institute
San Antonio, Texas, 78229
- Recruiting
UCSF
San Francisco, California, 94143
- Recruiting
Stony Brook University Medical Center
Stony Brook, New York, 11794
- Recruiting
University of Arizona Cancer Center-Out Pt.
Tucson, Arizona, 85719
- Recruiting
Childrens National Hospital
Washington D.C., District of Columbia, 20010
- Recruiting
Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, 27157
Italy17 sites, 17 recruiting
- Recruiting
Azienda Ospedaliero-Universitaria Di Alessandria Ss.Antonio E Biagio E Cesare Arrigo
Alessandria, 15121
- Recruiting
Azienda Ospedaliero Universitaria Delle Marche
Ancona, 60126
- Recruiting
Aou Policlinico Consorziale Di Bari
Bari, 70124
- Recruiting
Asst Degli Spedali Civili Di Brescia
Brescia, 25123
- Recruiting
Azienda Ospedaliero Universitaria Policlinico G.Rodolico San Marco
Catania, 95123
- Recruiting
Irccs Azienda Ospedaliera Universitaria San Martino
Genova, 16132
- Recruiting
Azienda Socio Sanitaria Territoriale Grande Ospedale Metropolitano Niguarda
Milan, 20162
- Recruiting
Ospedale San Raffaele - Milano
Milan, 20132
- Recruiting
Azienda Ospedaliera Di Rilievo Nazionale Antonio Cardarelli
Naples, 80131
- Recruiting
Azienda Ospedaliera Ospedali Riuniti Villa Sofia - Cervello
Palermo, 90146
- Recruiting
Irccs Fondazione Policlinico San Matteo
Pavia, 27100
- Recruiting
Grande Ospedale Metropolitano Bianchi-Melacrino-Morelli
Reggio Calabria, 89133
- Recruiting
Azienda Policlinico Umberto 1 Universita Sapienza Di Roma
Rome, 00161
- Recruiting
Ospedale Pediatrico Bambino Gesu Irccs
Rome, 00165
- Recruiting
I.R.C.C.S. Casa Sollievo Della Sofferenza
San Giovanni Rotondo, 71013
- Recruiting
Azienda Ospedaliera Card. G. Panico
Tricase, 73039
- Recruiting
Centro Ricerche Cliniche Di Verona
Verona, 37134
Germany16 sites, 16 recruiting
- Recruiting
University Medical Center Rwth Aachen
Aachen, D-52074
- Recruiting
Charite Universitatsmedizin Berlin
Berlin, 13353
- Recruiting
Universitaetsklinikum Bonn, University Hospital Bonn
Bonn, 53127
- Recruiting
University Clinic Carl Gustav Carus Technical University Dresden
Dresden, 01307
- Recruiting
University Hospital Duesseldorf
Düsseldorf, 40225
- Recruiting
Universitaetsklinikum Erlangen
Erlangen, 91054
- Recruiting
Universitatsklinikum Essen
Essen, 45147
- Recruiting
Universitatsklinikum Halle (Saale)
Halle, 06120
- Recruiting
Universitaetsklinikum Des Saarlandes
Homburg, 66421
- Recruiting
University Hospital Schleswig-Holstein Campus Kiel
Kiel, 24105
- Recruiting
Universitatsklinikum Leipzig
Leipzig, 04103
- Recruiting
Universitatsmedizin Der Johannes Gutenberg-Universitat Mainz Iii
Mainz, 55131
- Recruiting
Klinikum Der Philipps-Universitaet Marburg
Marburg, 35043
- Recruiting
Universitatsklinikum Munster
Münster, 48149
- Recruiting
University Hospital of Ulm, Universitatsklinikum Ulm
Ulm, 89081
- Recruiting
Universitatsklinikum Wurzburg Medizinische Klinik Und Poliklinik Ii
Würzburg, 97080
Japan13 sites, 13 recruiting
- Recruiting
Fujita Health University Hospital
Aichi, 470-1192
- Recruiting
Anjo Kosei Hospital
Aichi-ken, 446-8602
- Recruiting
Kyushu University Hospital
Fukuoka
- Recruiting
Hiroshima University Hospital
Hiroshima, 734-8551
- Recruiting
Kobe City Medical Center General Hospital
Hyōgo, 650-0047
- Recruiting
Tokai University Hospital
Isehara Kanagawa, 259-1193
- Recruiting
National Hospital Organization Kumamoto Medical Center
Kumamoto Kumamoto, 860-0008
- Recruiting
Gunma Saiseikai Maebashi Hospital
Maebashi, 371-0821
- Recruiting
Tohoku University Hospital
Miyagi, 980-8574
- Recruiting
Okayama University Hospital
Okayama Okayama, 700-0914
- Recruiting
Osaka Metropolitan University Hospital
Osaka, 545-8586
- Recruiting
Hokkaido University Hospital
Sapporo Hokkaido, 060-0814
- Recruiting
Tokyo Metropolitan Cancer and Infectious Diseases Center Komagome Hospital
Tokyo, 113-0021
Spain11 sites, 11 recruiting
- Recruiting
Hospital General Universitario Vall D Hebron
Barcelona, 08035
- Recruiting
Hospital de La Santa Creu I Sant Pau
Barcelona, 08041
- Recruiting
Hospital Universitario Donostia
Donostia / San Sebastian, 20014
- Recruiting
Institut Catala Doncologia Ico - Hospital Duran I Reynals Location
L'Hospitalet de Llobregat, 08906
- Recruiting
Hospital Universitario de Gran Canaria Doctor Negrin
Las Palmas de Gran Canaria, 35010
- Recruiting
Hospital General Universitario Gregorio Maranon
Madrid, 28007
- Recruiting
Hospital Puerta de Hierro
Majadahonda, 28222
- Recruiting
Hospital Clinico Universitario de Salamanca
Salamanca, 37007
- Recruiting
Hospital Universitario Virgen Del Rocio
Seville, 41013
- Recruiting
Hospital Universitari I Politecnic La Fe
Valencia, 46026
- Recruiting
Hospital Universitario Miguel Servet
Zaragoza, 50009
France9 sites, 9 recruiting
- Recruiting
Chu Amiens Picardie - Hopital Sud
Amiens, 80054
- Recruiting
Institut Paoli Calmettes
Marseille, 13273
- Recruiting
Hopitaux de Brabois
Nancy, 54511
- Recruiting
Centre Hospitalier Universitaire de Nantes (Chu de Nantes) - Hotel-Dieu
Nantes, 44903
- Recruiting
Chu de Nice - Hospital L Archet
Nice, 06202
- Recruiting
Hospital Saint Louis
Paris, 75010
- Recruiting
Hospital Saint Antoine
Paris, 75012
- Recruiting
Hospices Civils de Lyon Centre Hospitalier Lyon Sud
Pierre-Bénite, 69310
- Recruiting
Universitaire Du Cancer de Toulouse Institut Claudius Regaud Iuct-Oncopole
Toulouse, 31059
Canada8 sites, 8 recruiting
- Recruiting
Alberta'S Children Hospital
Calgary, Alberta, T3B 6A8
- Recruiting
Stollery Children'S Hospital
Edmonton, Alberta, T6G 2B7
- Recruiting
Queen Elizabeth Ii Health Sciences Centre
Halifax, Nova Scotia
- Recruiting
Juravinski Cancer Centre
Hamilton, Ontario, L8V 5C2
- Recruiting
Hospital Maisonneuve Rosemont
Montreal, Quebec, H1T 2M4
- Recruiting
Princess Margaret Cancer Center
Toronto, Ontario, M5G 2M9
- Recruiting
Bc Children'S Hospital
Vancouver, British Columbia, V6G1R8
- Recruiting
Vancouver General Hospital
Vancouver, V5Z1M9
United Kingdom8 sites, 8 recruiting
- Recruiting
Bristol Haematology and Oncology Centre
Bristol, BS2 8ED
- Recruiting
Queen Elizabeth University Hospital
Glasgow, G51 4TF
- Recruiting
St James University Hospital
Leeds, LS9 7TF
- Recruiting
Royal Marsden Hospital
London, SM2 5PT
- Recruiting
University College London Hospitals (Uclh)
London, WC1E 6AG
- Recruiting
Manchester University Nhs Foundation Trust
Manchester, M13 9WL.
- Recruiting
Nottingham University Hospitals
Nottingham, NG5 1PB
- Recruiting
Plymouth Hospitals Nhs Trust
Plymouth, PL6 8DH
Austria5 sites, 5 recruiting
- Recruiting
Landeskrankenhaus Universitatsklinikum Graz
Graz, A-8036
- Recruiting
Innsbruck University Hospital
Innsbruck, A-6020
- Recruiting
Ordensklinikum Linz Gmbh Elisabethinen
LINZ CET, A-4020
- Recruiting
Akh Und Medizinische Universitat Wien Universitatsklinik Fur Innere Medizin I
Vienna, 01090
- Recruiting
St. Anna Childrens Hospital
Vienna, 01090
Australia3 sites, 3 recruiting
- Recruiting
Royal Adelaide Hospital
Adelaide, South Australia, 05000
- Recruiting
Austin Health Medical Oncology and Clinical Haematology
Heidelberg, Victoria, 03084
- Recruiting
Royal Prince Alfred Hospital
Sydney, New South Wales, 02050
Netherlands2 sites, 2 recruiting
- Recruiting
Amsterdam University Medical Centre
Amsterdam, 1081 HV
- Recruiting
Maastricht University Medical Center
Maastricht, 6229 HX
About this study
Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease (AXemplify-357)
This study will be conducted to compare the efficacy of axatilimab versus placebo in combination with corticosteroids as initial treatment for moderate or severe chronic graft-versus-host disease (cGVHD).
- Conditions
- Chronic Graft-versus-host-disease
- Being tested
- INCA034176, Placebo, Corticosteroids
- Keywords
- cGVHD
- Medical subject headings
- Bronchiolitis Obliterans Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
INCA034176Drug
Also called Axatilimab
IV infusion
PlaceboDrug
IV infusion
CorticosteroidsDrug
Also called prednisone, methylprednisolone, prednisolone
Oral/IV Infusion
| Study group | Role | What they receive |
|---|---|---|
| Axatilimab + Corticosteroids | Experimental | Axatilimab and Corticosteroids at the protocol-defined dose. Receives: Drug: INCA034176; Drug: Corticosteroids |
| Placebo + Corticosteroids | Experimental | Matching placebo and Corticosteroids at the protocol-defined dose. Receives: Drug: Placebo; Drug: Corticosteroids |
What is being measured
The primary outcome is the main question the study is designed to answer.
Event Free Survival (EFS)
Defined from the date of randomization to the date of any predefined event, whichever occurs first.
Measured: Up to 3 years
Secondary outcomes (16)
Objective Response (OR)
Defined for each treatment group as CR or PR at 6 months Cycle 7 (28-day cycles), Day 1, in the absence of new systemic therapy for cGVHD. Responses defined by the 2014 NIH consensus criteria.
Measured: 6 months
Event Free Survival 2
Defined from the date of randomization to the date of any predefined event, whichever occurs first.
Measured: Up to 3 years
Proportion of participants with a ≥ 7-point improvement in mLSS total score
Measured: Up to 3 years
Overall Response
Defined as CR or PR at 12 months in the absence of new systemic therapy for cGVHD.
Measured: 12 Months
DOR (in responders only)
Defined as the time from the date of first response (PR or CR) to the date of progression of cGVHD from nadir in any organ, start of new systemic treatment for cGVHD, or death from any cause, whichever comes first.
Measured: Up to 3 years
Best Overall Response (BOR)
Defined as the best response of CR or PR at any timepoint up to the initiation of new therapy for cGVHD.
Measured: Up to 3 years
Overall Survival (OS)
Defined as the time from the date of randomization to the date of death due to any cause.
Measured: Up to 3 years
Nonrelapse mortality (NRM)
Defined as the time from the date of randomization to the date of death in the absence of primary hematologic disease relapse.
Measured: Up to 3 years
Failure-free survival (FFS)
Defined as the time from the date of randomization to the date of addition or initiation of another systemic therapy for cGVHD, relapse of underlying disease, or death due to any cause.
Measured: Up to 3 years
Relapse of hematologic diseases
Defined as the reappearance of symptoms of underlying disease.
Measured: Up to 3 years
Time to primary hematologic disease relapse
Defined as the time from the date of randomization to the date of relapse.
Measured: Up to 3 years
Percent reduction in daily corticosteroid dose
Measured: 6 months
Proportion of participants who tapered off all corticosteroids
Measured: 6 months
Number of participants with Treatment-emergent Adverse Events (TEAEs)
Defined as adverse events reported for the first time or worsening of a pre-existing event after the first dose of study treatment.
Measured: Up to 3 years and 30 days
Change from baseline in circulating monocyte number and phenotype (CD14/16)
Measured: Up to 3 years and 30 days
Change from baseline in soluble markers for bone resorption and formation, including bone-specific alkaline phosphatase (BAP) and C-terminal telopeptide (CTX)
Measured: Up to 3 years and 30 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2024
- Study start21 Jan 2025
- Primary completion28 Sep 2027 (estimated)
- Study completion31 Mar 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Graft-versus-host-diseaseStudies of the same condition, open and under way.
- More trials run by Incyte CorporationThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 240 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Incyte Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Albert Assad, MD, Study director, Incyte Corporation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06585774
- Sponsor's protocol ID
- INCA34176-357
- Other IDs
- 2023-510292-65-00, 2023
- Acronym
- AXemplify-357
- Status last verified
- Oct 2026
- First submitted
- 30 Aug 2024
- Last updated
- 9 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Incyte shares data with qualified external researchers after a research proposal is submitted. These requests are reviewed and approved by a review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. The trial data availability is according to the criteria and process described on https://www.incyte.com/our-company/compliance-and-transparency
- Expanded access
- Available