Clinical Research Directory

Browse clinical research sites, groups, and studies.

Sites Groups Studies

Back to Studies

ACTIVE NOT RECRUITING

NCT04710576

PHASE2

A Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD)

Sponsor: Syndax Pharmaceuticals

View on ClinicalTrials.gov

Summary

This is a Phase 2 study to evaluate the efficacy, safety, and tolerability of axatilimab at 3 different dose levels in participants with recurrent or refractory active chronic graft versus host disease (cGVHD) who have received at least 2 prior lines of systemic therapy.

Official title: AGAVE-201, A Phase 2, Open-label, Randomized, Multicenter Study to Evaluate the Efficacy, Safety and Tolerability of Axatilimab at 3 Different Doses in Patients With Recurrent or Refractory Active Chronic Graft Versus Host Disease Who Have Received at Least 2 Lines of Systemic Therapy

Key Details

Gender

All

Age Range

2 Years - Any

Study Type

INTERVENTIONAL

Enrollment

241

Start Date

2021-03-04

Completion Date

2027-09

Last Updated

2026-03-16

Healthy Volunteers

Conditions

Chronic Graft-versus-host-disease

Interventions

DRUG

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.

Inclusion Criteria: 1. Participants must be 2 years of age or older, at the time of signing the informed consent. 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: Participants are excluded from the study if any of the following criteria apply: 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).

Locations (121)

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

Clinical Research Directory

A Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD)

Summary

Key Details

Conditions

Interventions

Eligibility Criteria

Locations (121)