A Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD)
NCT ID: NCT04710576
Last Updated: 2025-09-03
Study Results
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View full resultsBasic Information
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ACTIVE_NOT_RECRUITING
PHASE2
241 participants
INTERVENTIONAL
2021-03-04
2027-12-31
Brief Summary
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Detailed Description
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Participants will be randomized to receive 1 of 3 different axatilimab treatment regimens in 28-day treatment cycles for up to 2 years.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Axatilimab Dose Cohort 1
Participants will be administered axatilimab 0.3 milligrams (mg)/kilogram (kg) intravenously (IV) every 2 weeks for up to 2 years.
Axatilimab
Axatilimab is a high-affinity antibody targeting the colony stimulating factor 1 receptor (CSF-1R). CSF-1R signaling has been demonstrated in nonclinical studies to be the key regulatory pathway involved in the expansion and infiltration of donor-derived macrophages that mediate the disease processes involved in cGVHD.
Axatilimab Dose Cohort 2
Participants will be administered axatilimab 1 mg/kg IV every 2 weeks for up to 2 years.
Axatilimab
Axatilimab is a high-affinity antibody targeting the colony stimulating factor 1 receptor (CSF-1R). CSF-1R signaling has been demonstrated in nonclinical studies to be the key regulatory pathway involved in the expansion and infiltration of donor-derived macrophages that mediate the disease processes involved in cGVHD.
Axatilimab Dose Cohort 3
Participants will be administered axatilimab 3 mg/kg IV every 4 weeks for up to 2 years.
Axatilimab
Axatilimab is a high-affinity antibody targeting the colony stimulating factor 1 receptor (CSF-1R). CSF-1R signaling has been demonstrated in nonclinical studies to be the key regulatory pathway involved in the expansion and infiltration of donor-derived macrophages that mediate the disease processes involved in cGVHD.
Interventions
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Axatilimab
Axatilimab is a high-affinity antibody targeting the colony stimulating factor 1 receptor (CSF-1R). CSF-1R signaling has been demonstrated in nonclinical studies to be the key regulatory pathway involved in the expansion and infiltration of donor-derived macrophages that mediate the disease processes involved in cGVHD.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Participants who are allogeneic hematopoietic stem cell transplantation (HSCT) recipients with active cGVHD requiring systemic immune suppression. Active cGVHD is defined as the presence of signs and symptoms of cGVHD per 2014 NIH Consensus Development Project on Criteria for Clinical trials in cGVHD.
3. Participants with refractory or recurrent active cGVHD despite at least 2 lines of systemic therapy.
* Refractory disease defined as meeting any of the following criteria:
* The development of 1 or more new sites of disease while being treated for cGVHD.
* Progression of existing sites of disease despite at least 1 month of standard or investigation therapy for cGVHD.
* Participants who have not achieved a response within 3 months on their prior therapy for cGVHD and for whom the treating physician believes a new systemic therapy is required.
* Recurrent cGVHD is active, symptomatic disease (after an initial response to prior therapy) as defined, based on the NIH 2014 consensus criteria, by organ-specific or global assessment or for which the physician believes that a new line of systemic therapy is required.
4. Participants may have persistent, active acute and cGVHD manifestations (overlap syndrome), as defined by 2014 NIH Consensus Development Project on Criteria for Clinical trials in cGVHD.
5. Karnofsky Performance Scale of ≥60 (if aged 16 years or older); Lansky Performance Score of ≥60 (if aged \<16 years)
6. Adequate organ and bone marrow functions evaluated during the 14 days prior to randomization.
7. Creatinine clearance (CrCl) ≥30 milliliter/minute based on the Cockcroft-Gault formula in adult participants and Schwartz formula in pediatric participants.
8. Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
9. Concomitant use a of systemic corticosteroid is allowed but not required. Topical and inhaled corticosteroid agents are allowed. If a participant is taking corticosteroids at study randomization, they must be on a stable dose of corticosteroids for at least 2 weeks prior to Cycle 1 Day 1.
10. Concomitant use of CNI or mammalian target of repamycin (mTOR) inhibitors (sirolimus or everolimus) is allowed but not required.
11. Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and protocol. A parent/guardian should provide consent for pediatric participants unable to provide consent themselves; in addition, where applicable pediatric participants should sign their own assent form.
Exclusion Criteria
1. Has acute GVHD without manifestations of cGVHD.
2. Any evidence (histologic, cytogenetic, molecular, hematologic, or mixed) of relapse of the underlying cancer or post-transplant lymphoproliferative disease at the time of screening.
3. History of acute or chronic pancreatitis.
4. History of myositis.
5. History or other evidence of severe illness, uncontrolled infection or any other conditions that would make the participant, in the opinion of the Investigator, unsuitable for the study.
6. Participants with acquired immune deficiency syndrome (AIDS).
7. Hepatitis B (defined as hepatitis B virus \[HBV\] surface antigen positive and HBV core antibody positive, with positive HBV deoxyribonucleic acid \[DNA\], or HBV positive core antibody alone with positive HBV DNA. Hepatitis C (defined as positive hepatitis C \[HCV\] antibody with positive HCV ribonucleic acid \[RNA\]).
8. Diagnosed with another malignancy (other than malignancy for which transplant was performed) within 3 years of randomization, unless previously treated with curative intent and approved by Sponsor's Medical Monitor (for example, completely resected basal cell or squamous cell carcinoma of the skin, resected in situ cervical malignancy, resected breast ductal carcinoma in situ, or low-risk prostate cancer after curative resection).
9. Female participant who is pregnant or breastfeeding.
10. Previous exposure to CSF1-R targeted therapies.
11. Taking agents for treatment of cGVHD other than corticosteroids or either a CNI or mTOR inhibitor is prohibited.
12. For approved or commonly used agents, other than corticosteroids, CNI and mTOR inhibitor, a washout of 2 weeks or 5 half-lives, whichever is shorter, is required at study enrollment.
13. Receiving another investigational treatment within 28 days of randomization.
14. Participants should not be participating in any other interventional study. Pediatric participants are encouraged to also participate in the ongoing developmental studies of the Pediatric cGVHD Symptom Scale (PCSS).
2 Years
ALL
No
Sponsors
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Syndax Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Vedran Radojcic, M.D.
Role: STUDY_DIRECTOR
Syndax Pharmaceuticals
Locations
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University of Alabama at Birmingham - Children's of Alabama
Birmingham, Alabama, United States
University of Alabama at Birmingham
Birmingham, Alabama, United States
City of Hope
Duarte, California, United States
University of Southern California Norris Comprehensive Cancer Center
Los Angeles, California, United States
University of California, Los Angeles (UCLA) - Medical Center
Los Angeles, California, United States
Stanford Cancer Center
Stanford, California, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
University of Florida (UF)
Gainesville, Florida, United States
Mayo Clinic - Jacksonville
Jacksonville, Florida, United States
University of Miami
Miami, Florida, United States
AdventHealth Orlando
Orlando, Florida, United States
Moffitt
Tampa, Florida, United States
Emory University
Atlanta, Georgia, United States
Northside Hospital
Atlanta, Georgia, United States
The University of Chicago Medical Center (UCMC)
Chicago, Illinois, United States
Indiana University Health Melvin and Bren Simon Cancer Center
Indianapolis, Indiana, United States
Franciscan Health Indianapolis
Indianapolis, Indiana, United States
Tulane University Medical Center
New Orleans, Louisiana, United States
Johns Hopkins Kimmel Cancer Center
Baltimore, Maryland, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Dana Farber Cancer Institute
Boston, Massachusetts, United States
University of Massachusetts Memorial Medical Center
Worcester, Massachusetts, United States
University of Michigan
Ann Arbor, Michigan, United States
Barbara Ann Karmanos Cancer Institute
Detroit, Michigan, United States
Henry Ford Hospital
Detroit, Michigan, United States
University of Minnesota
Minneapolis, Minnesota, United States
Mayo Clinic - Rochester
Rochester, Minnesota, United States
Washington University School of Medicine
St Louis, Missouri, United States
Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey, United States
Weill Medical College of Cornell University
New York, New York, United States
Stony Brook University Medical Center
Stony Brook, New York, United States
Wake Forest
Winston-Salem, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States
The Ohio State University Comprehensive Cancer Center
Columbus, Ohio, United States
The Cleveland Clinic Foundation
Lyndhurst, Ohio, United States
University of Oklahoma - Health Sciences Center
Oklahoma City, Oklahoma, United States
Oregon Health & Science University
Portland, Oregon, United States
University of Pittsburgh Medical Center - Hillman Cancer Center
Pittsburgh, Pennsylvania, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
MD Anderson Cancer Center
Houston, Texas, United States
Intermountain Healthcare
Salt Lake City, Utah, United States
University of Utah
Salt Lake City, Utah, United States
University of Virginia Medical Center
Charlottesville, Virginia, United States
Fred Hutchinson Cancer Research Center
Seattle, Washington, United States
University of Wisconsin - Carbone Cancer Center
Madison, Wisconsin, United States
Froedtert Hospital and the Medical College of Wisconsin
Milwaukee, Wisconsin, United States
The Royal Children's Hospital
Parkville, Victoria, Australia
Westmead Hospital
Westmead, , Australia
Universitaire Ziekenhuizen Leuven
Leuven, , Belgium
AZ Delta
Roeselare, , Belgium
Vancouver Coastal Health Authority
Vancouver, British Columbia, Canada
Princess Margaret Hospital
Toronto, Ontario, Canada
McGill University Health Center - Research Institute
Montreal, Quebec, Canada
CHU Sainte-Justine
Montreal, Quebec, Canada
CHU de Grenoble
La Tronche, Auvergne-Rhône-Alpes, France
Institut de cancérologie Strasbourg Europe (ICANS)
Strasbourg, Grand Est, France
IUCT-Oncopole
Toulouse, Haure-Garrone, France
CHU Amiens Picardie - Hopital Sud
Amiens, Hauts-de-France, France
CHRU de Lille - Hopital Claude Huriez
Lille, Hauts-de-France, France
CHRU de Nancy - Hôpitaux de Brabois
Nancy, , France
CHU de Nantes - Hôtel-Dieu
Nantes, , France
Hopital Saint Louis
Paris, , France
Hopital Pitie Salpetriere
Paris, , France
CHU Bordeaux - Hopital Haut-Leveque - Centre François Magendie
Pessac, , France
HCL Centre Hospitalier Lyon Sud
Pierre-Bénite, , France
Universitaetsklinikum Carl Gustav Carus Dresden
Dresden, , Germany
Universitaetsklinikum Jena
Jena, , Germany
Universitaetsklinikum Leipzig
Leipzig, , Germany
Universitaetsmedizin der Johannes Gutenberg - Universitaet Mainz
Mainz, , Germany
Universitaetsklinikum Muenster
Münster, , Germany
Universitatsklinikum Regensburg
Regensburg, , Germany
General Hospital of Thessaloniki G. Papanikolaou - Hematology Department, BMT Unit
Eksochi, Thessaloniki, Greece
University Hospital of West Attica - Attikon - Hematology Division
Athens, , Greece
University General Hospital of Patras
Pátrai, , Greece
Rambam Health Care Campus
Haifa, , Israel
Hadassah Medical Center Ein Karem
Jerusalem, , Israel
Chaim Sheba Medical Center
Ramat Gan, , Israel
Tel Aviv Sourasky Medical Center
Tel Aviv, , Israel
ASST degli Spedali Civili di Brescia
Brescia, , Italy
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano
Milan, , Italy
IRCCS Ospedale San Raffaele
Milan, , Italy
ASST di Monza-Ospedale San Gerardo
Monza, , Italy
Fondazione Monza e Brianza per il Bambino e la sua Mamma
Monza, , Italy
Fondazione IRCCS Policlinico San Matteo
Pavia, , Italy
Fondazione IRCCS Policlinico San Matteo
Pavia, , Italy
Fondazione Policlinica Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Roma, , Italy
AOU Citta della Salute e della Scienza di Torino - Ospedale Regina Margherita
Torino, , Italy
Citta della Salute e della Scienza di Torino - Ospedale le Molinette
Torino, , Italy
Narodowy Instytut Onkologii im. Marii Sklodowskiej-Curie - Panstwowy Instytut Badawczy, Oddzial w Gliwicach - Klinika Transplantacji Szpiku i Onkohematologii
Gliwice, , Poland
Instituto Portugues de Oncologia de Lisboa Francisco Gentil, E.P.E. (IPO-Lisboa)
Lisbon, , Portugal
Instituto Portugues de Oncologia do Porto Francisco Gentil, EPE
Porto, , Portugal
National University Hospital
Singapore, , Singapore
KK Women's and Children hospital
Singapore, , Singapore
Singapore General Hospital
Singapore, , Singapore
Pusan National University Hospital
Busan, , South Korea
Korea University Anam Hospital
Seoul, , South Korea
Seoul National University Hospital
Seoul, , South Korea
Severance Hospital
Seoul, , South Korea
Hospital Universitario Virgen del Rocio
Seville, Seville, Spain
Hospital Universitario Vall d'Hebron
Barcelona, , Spain
Hospital Clinic Barcelona
Barcelona, , Spain
Hospital Universitario Donostia
Donostia / San Sebastian, , Spain
Complejo Hospitalario Universitario de Granada - Hospital Universitario Virgen de las Nieves
Granada, , Spain
Hospital General Universitario Gregorio Maranon
Madrid, , Spain
Hospital Universitario Ramon y Cajal
Madrid, , Spain
Hospital Universitario La Paz
Madrid, , Spain
Hospital Universitario Puerta de Hierro
Majadahonda, , Spain
Hospital Clinico Universitario de Salamanca
Salamanca, , Spain
Hospital Universitario Marquis de Valdecilla
Santander, , Spain
Hospital Clinico Universitario de Valencia
Valencia, , Spain
Hospital Universitari i Politecnic La Fe
Valencia, , Spain
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, , Taiwan
China Medical University Hospital
Taichung, , Taiwan
National Taiwan University Hospital
Taipei, , Taiwan
Bristol Royal Hospital for Children
Bristol, , United Kingdom
University Hospital of Wales
Cardiff, , United Kingdom
Queen Elizabeth University Hospital
Glasgow, , United Kingdom
Hammersmith Hospital
London, , United Kingdom
King's College Hospital NHS Foundation Trust
London, , United Kingdom
Royal Marsden Foundation Trust
London, , United Kingdom
Countries
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References
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Wolff D, Cutler C, Lee SJ, Pusic I, Bittencourt H, White J, Hamadani M, Arai S, Salhotra A, Perez-Simon JA, Alousi A, Choe H, Kwon M, Bermudez A, Kim I, Socie G, Chhabra S, Radojcic V, O'Toole T, Tian C, Ordentlich P, DeFilipp Z, Kitko CL; AGAVE-201 Investigators. Axatilimab in Recurrent or Refractory Chronic Graft-versus-Host Disease. N Engl J Med. 2024 Sep 19;391(11):1002-1014. doi: 10.1056/NEJMoa2401537.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2024-512978-99-00
Identifier Type: CTIS
Identifier Source: secondary_id
SNDX-6352-0504
Identifier Type: -
Identifier Source: org_study_id
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