A Study to Evaluate Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease
AXemplify-357
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease (AXemplify-357)
2 other identifiers
interventional
240
12 countries
114
Brief 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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started Jan 2025
Longer than P75 for phase_3
114 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 30, 2024
CompletedFirst Posted
Study publicly available on registry
September 19, 2024
CompletedStudy Start
First participant enrolled
January 21, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 28, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
March 31, 2030
May 4, 2026
April 1, 2026
2.7 years
August 30, 2024
April 30, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Event Free Survival (EFS)
Defined from the date of randomization to the date of any predefined event, whichever occurs first.
Up to 3 years
Secondary Outcomes (16)
Objective Response (OR)
6 months
Event Free Survival 2
Up to 3 years
Proportion of participants with a ≥ 7-point improvement in mLSS total score
Up to 3 years
Overall Response
12 Months
DOR (in responders only)
Up to 3 years
- +11 more secondary outcomes
Study Arms (2)
Axatilimab + Corticosteroids
EXPERIMENTALAxatilimab and Corticosteroids at the protocol-defined dose.
Placebo + Corticosteroids
EXPERIMENTALMatching placebo and Corticosteroids at the protocol-defined dose.
Interventions
Oral/IV Infusion
Eligibility Criteria
You may qualify if:
- ≥ 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.
You may not qualify if:
- 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.
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (119)
University of Alabama Birmingham
Birmingham, Alabama, 35294, United States
University of California San Diego Medical Center, Moores Cancer Center
La Jolla, California, 92037, United States
University of Southern California
Los Angeles, California, 90089, United States
Colorado Blood Cancer Institute
Denver, Colorado, 80218, United States
Childrens National Hospital
Washington D.C., District of Columbia, 20010, United States
Miami Cancer Institute
Miami, Florida, 33176, United States
Orlando Health Cancer Institute Downtown Orlando
Orlando, Florida, 32806, United States
Memorial Cancer Institute
Pembroke Pines, Florida, 33026, United States
Emory University-Winship Cancer Institute
Atlanta, Georgia, 30322, United States
University of Illinois
Chicago, Illinois, 60612, United States
The University of Kansas Cancer Center
Kansas City, Kansas, 66160, United States
John Hopkins Hospital
Baltimore, Maryland, 21287, United States
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
Dana Farber Cancer Institute
Boston, Massachusetts, 02215, United States
University of Michigan
Ann Arbor, Michigan, 48109, United States
Henry Ford Hospital
Detroit, Michigan, 48202, United States
Corewell Health Hematology Oncology
Grand Rapids, Michigan, 49503, United States
Hackensack University Medical Center
Hackensack, New Jersey, 07601, United States
Rutgers Cancer Institute of Nj
New Brunswick, New Jersey, 08903, United States
University of Rochester Medical Center
Rochester, New York, 14642, United States
Stony Brook University Medical Center
Stony Brook, New York, 11794, United States
Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, 27157, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
Jefferson University Hospitals
Philadelphia, Pennsylvania, 19107, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
Prisma Health Upstate
Greenville, South Carolina, 29615, United States
Baptist Cancer Center
Memphis, Tennessee, 38120, United States
St David'S South Austin Medical Center
Austin, Texas, 78704, United States
Texas Transplant Institute
San Antonio, Texas, 78229, United States
Intermountain Blood and Marrow Transplant
Salt Lake City, Utah, 84143, United States
Virginia Commonwealth University Massey Cancer Center
Richmond, Virginia, 23298, United States
West Virginia University Cancer Institute
Morgantown, West Virginia, 26506, United States
Royal Prince Alfred Hospital
Sydney, New South Wales, 02050, Australia
Royal Adelaide Hospital
Adelaide, South Australia, 05000, Australia
Austin Health Medical Oncology and Clinical Haematology
Heidelberg, Victoria, 03084, Australia
Landeskrankenhaus Universitatsklinikum Graz
Graz, A-8036, Austria
Innsbruck University Hospital
Innsbruck, A-6020, Austria
Ordensklinikum Linz Gmbh Elisabethinen
LINZ CET, A-4020, Austria
Akh Und Medizinische Universitat Wien Universitatsklinik Fur Innere Medizin I
Vienna, 01090, Austria
St. Anna Childrens Hospital
Vienna, 01090, Austria
Alberta'S Children Hospital
Calgary, Alberta, T3B 6A8, Canada
Stollery Children'S Hospital
Edmonton, Alberta, T6G 2B7, Canada
Bc Children'S Hospital
Vancouver, British Columbia, V6G1R8, Canada
Queen Elizabeth Ii Health Sciences Centre
Halifax, Nova Scotia, Canada
Juravinski Cancer Centre
Hamilton, Ontario, L8V 5C2, Canada
Princess Margaret Cancer Center
Toronto, Ontario, M5G 2M9, Canada
Hospital Maisonneuve Rosemont
Montreal, Quebec, H1T 2M4, Canada
Vancouver General Hospital
Vancouver, V5Z1M9, Canada
Rigshospitalet
Copenhagen, 02100, Denmark
Chu Amiens Picardie - Hopital Sud
Amiens, 80054, France
Institut Paoli Calmettes
Marseille, 13273, France
Hopitaux de Brabois
Nancy, 54511, France
Centre Hospitalier Universitaire de Nantes (Chu de Nantes) - Hotel-Dieu
Nantes, 44903, France
Chu de Nice - Hospital L Archet
Nice, 06202, France
Hospital Saint Louis
Paris, 75010, France
Hospital Saint Antoine
Paris, 75012, France
Hospices Civils de Lyon Centre Hospitalier Lyon Sud
Pierre-Bénite, 69310, France
Universitaire Du Cancer de Toulouse Institut Claudius Regaud Iuct-Oncopole
Toulouse, 31059, France
University Medical Center Rwth Aachen
Aachen, D-52074, Germany
Charite Universitatsmedizin Berlin
Berlin, 13353, Germany
Universitaetsklinikum Bonn, University Hospital Bonn
Bonn, 53127, Germany
University Clinic Carl Gustav Carus Technical University Dresden
Dresden, 01307, Germany
University Hospital Duesseldorf
Düsseldorf, 40225, Germany
Universitaetsklinikum Erlangen
Erlangen, 91054, Germany
Universitatsklinikum Essen
Essen, 45147, Germany
Universitatsklinikum Halle (Saale)
Halle, 06120, Germany
University Hospital Schleswig-Holstein Campus Kiel
Kiel, 24105, Germany
Universitatsklinikum Leipzig
Leipzig, 04103, Germany
Universitatsmedizin Der Johannes Gutenberg-Universitat Mainz Iii
Mainz, 55131, Germany
Klinikum Der Philipps-Universitaet Marburg
Marburg, 35043, Germany
Universitatsklinikum Munster
Münster, 48149, Germany
St. James Hospital
Dublin, Ireland
Azienda Ospedaliero-Universitaria Di Alessandria Ss.Antonio E Biagio E Cesare Arrigo
Alessandria, 15121, Italy
Azienda Ospedaliero Universitaria Delle Marche
Ancona, 60126, Italy
Asst Degli Spedali Civili Di Brescia
Brescia, 25123, Italy
Irccs Azienda Ospedaliera Universitaria San Martino
Genova, 16132, Italy
Azienda Socio Sanitaria Territoriale Grande Ospedale Metropolitano Niguarda
Milan, 20162, Italy
Ospedale San Raffaele - Milano
Milan, 20132, Italy
Azienda Ospedaliera Di Rilievo Nazionale Antonio Cardarelli
Naples, 80131, Italy
Azienda Ospedaliera Ospedali Riuniti Villa Sofia - Cervello
Palermo, 90146, Italy
Irccs Fondazione Policlinico San Matteo
Pavia, 27100, Italy
Grande Ospedale Metropolitano Bianchi-Melacrino-Morelli
Reggio Calabria, 89133, Italy
Azienda Policlinico Umberto 1 Universita Sapienza Di Roma
Rome, 00161, Italy
Ospedale Pediatrico Bambino Gesu Irccs
Rome, 00165, Italy
I.R.C.C.S. Casa Sollievo Della Sofferenza
San Giovanni Rotondo, 71013, Italy
Azienda Ospedaliera Card. G. Panico
Tricase, 73039, Italy
Centro Ricerche Cliniche Di Verona
Verona, 37134, Italy
Fujita Health University Hospital
Aichi, 470-1192, Japan
Anjo Kosei Hospital
Aichi-ken, 446-8602, Japan
Kyushu University Hospital
Fukuoka, Japan
Hiroshima University Hospital
Hiroshima, 734-8551, Japan
Kobe City Medical Center General Hospital
Hyōgo, 650-0047, Japan
Tokai University Hospital
Isehara Kanagawa, 259-1193, Japan
National Hospital Organization Kumamoto Medical Center
Kumamoto Kumamoto, 860-0008, Japan
Gunma Saiseikai Maebashi Hospital
Maebashi, 371-0821, Japan
Tohoku University Hospital
Miyagi, 980-8574, Japan
Okayama University Hospital
Okayama Okayama, 700-0914, Japan
Osaka Metropolitan University Hospital
Osaka, 545-8586, Japan
Hokkaido University Hospital
Sapporo Hokkaido, 060-8648, Japan
Tokyo Metropolitan Cancer and Infectious Diseases Center Komagome Hospital
Tokyo, 113-0021, Japan
Amsterdam University Medical Centre
Amsterdam, 1081 HV, Netherlands
Maastricht University Medical Center
Maastricht, 6229 HX, Netherlands
Hospital General Universitario Vall D Hebron
Barcelona, 08035, Spain
Hospital de La Santa Creu I Sant Pau
Barcelona, 08041, Spain
Hospital Universitario Donostia
Donostia / San Sebastian, 20014, Spain
Institut Catala Doncologia Ico - Hospital Duran I Reynals Location
L'Hospitalet de Llobregat, 08906, Spain
Hospital Universitario de Gran Canaria Doctor Negrin
Las Palmas de Gran Canaria, 35010, Spain
Hospital General Universitario Gregorio Maranon
Madrid, 28007, Spain
Hospital Puerta de Hierro
Majadahonda, 28222, Spain
Hospital Universitario Virgen Del Rocio
Seville, 41013, Spain
Hospital Universitari I Politecnic La Fe
Valencia, 46026, Spain
Hospital Universitario Miguel Servet
Zaragoza, 50009, Spain
Bristol Haematology and Oncology Centre
Bristol, BS2 8ED, United Kingdom
Queen Elizabeth University Hospital
Glasgow, G51 4TF, United Kingdom
Royal Marsden Hospital
London, SM2 5PT, United Kingdom
University College London Hospitals (Uclh)
London, WC1E 6AG, United Kingdom
Manchester University Nhs Foundation Trust
Manchester, United Kingdom
Nottingham University Hospitals
Nottingham, NG5 1PB, United Kingdom
Plymouth Hospitals Nhs Trust
Plymouth, PL6 8DH, United Kingdom
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Albert Assad, MD
Incyte Corporation
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
- Expanded Access
- Yes
Study Record Dates
First Submitted
August 30, 2024
First Posted
September 19, 2024
Study Start
January 21, 2025
Primary Completion (Estimated)
September 28, 2027
Study Completion (Estimated)
March 31, 2030
Last Updated
May 4, 2026
Record last verified: 2026-04
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.
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