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A Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease

NCT06388564 · Incyte Corporation
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Official title
A Phase 2, Open-Label, Randomized, Multicenter Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease
About this study
This study will be conducted to determine the preliminary efficacy of axatilimab in combination with ruxolitinib and to assess the contribution of axatilimab to the combination treatment effect in participants with cGVHD.
Eligibility criteria
Inclusion Criteria: * ≥ 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 1 allo-SCT (any type of stem cell donor, any conditioning regimen, and source of hematopoietic stem cells). * Adequate hematologic function independent of platelet transfusion and growth factors for at least 7 days prior to study entry: ANC ≥ 0.75 × 109/L and platelet count ≥ 20 × 109/L. * Willingness to avoid pregnancy or fathering children. Exclusion Criteria: * Received more than 1 prior allo-SCT. Prior autologous HCT is allowed. * Has overlap cGVHD, defined as simultaneous presence of features or characteristics of aGVHD in a patient with cGVHD. * Received previous systemic treatment for cGVHD, including systemic corticosteroids and extracorporeal photopheresis. * Received systemic corticosteroids within 2 weeks prior to C1D1, regardless of indication. * Initiated systemic treatment with CNIs or mTOR inhibitors within 2 weeks prior to C1D1. * Prior treatment with a JAK inhibitor within 8 weeks before randomization. Participants who received a JAK inhibitor for the treatment of aGVHD are eligible only if they achieved a response (CR or PR) to JAK inhibitor treatment and did not discontinue due to toxicity. * Evidence of relapse of the primary hematologic disease or treatment for relapse after the allo-SCT was performed, including DLIs for the treatment of molecular relapse. * History of acute or chronic pancreatitis. * History of thromboembolic events (such as deep vein thrombosis, pulmonary embolism, stroke, myocardial infarction) in the 6 months prior to study entry. * Active symptomatic myositis. * 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 end-stage renal disease on dialysis. Participants with CrCl of 30 to 59 mL/min on treatment with fluconazole are not eligible. * 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. * Currently active significant cardiac disease, such as uncontrolled arrhythmias, uncontrolled hypertension, or Class 3 or 4 congestive heart failure as defined by New York Heart Association, or a history of myocardial infarction or unstable angina within 6 months prior to randomization. * Pregnant or breastfeeding. Other protocol-defined Inclusion/Exclusion Criteria may apply.
Study design
Enrollment target: 120 participants
Allocation: randomized
Masking: none
Age groups: child, adult, older_adult
Timeline
Starts: 2024-10-11
Estimated completion: 2029-12-01
Last updated: 2026-03-23
Interventions
Drug: AxatilimabDrug: RuxolitinibDrug: Corticosteroids
Primary outcomes
  • Objective Response Rate (6 months)
Sponsor
Incyte Corporation · industry
Contacts & investigators
ContactIncyte Corporation Call Center (US) · contact · medinfo@incyte.com · 1.855.463.3463
ContactIncyte Corporation Call Center (ex-US) · contact · eumedinfo@incyte.com · +800 00027423
InvestigatorIncyte Medical Monitor · study_director, Incyte Corporation
All locations (70)
Mayo Clinic HospitalRecruiting
Phoenix, Arizona, United States
City of Hope Medical CenterRecruiting
Duarte, California, United States
University of California-Los Angeles Medl Cntr-Oncology Center Bowyer ClinicRecruiting
Los Angeles, California, United States
Stanford Cancer CenterRecruiting
Stanford, California, United States
University of Colorado Cancer CenterRecruiting
Aurora, Colorado, United States
Colorado Blood Cancer InstituteRecruiting
Denver, Colorado, United States
Smilow Cancer Center-YaleRecruiting
New Haven, Connecticut, United States
Mayo Clinic JacksonvilleRecruiting
Jacksonville, Florida, United States
Northwestern UniversityRecruiting
Chicago, Illinois, United States
The University of Chicago MedicineRecruiting
Chicago, Illinois, United States
University of Maryland-Greenebaum Cancer CenterRecruiting
Baltimore, Maryland, United States
Massachusetts General HospitalWithdrawn
Boston, Massachusetts, United States
Washington UniversityRecruiting
St Louis, Missouri, United States
Fred and Pamela Buffett Cancer CenterRecruiting
Omaha, Nebraska, United States
Memorial Sloan Kettering Cancer CenterRecruiting
New York, New York, United States
Weill Cornell MedicineRecruiting
New York, New York, United States
Mount Sinai HospitalRecruiting
New York, New York, United States
Cleveland ClinicRecruiting
Cleveland, Ohio, United States
The Ohio State UniversityRecruiting
Columbus, Ohio, United States
University of Pennsylvania Abramson Cancer CenterRecruiting
Philadelphia, Pennsylvania, United States
Hillman Cancer CenterRecruiting
Pittsburgh, Pennsylvania, United States
Vanderbilt University Medical CenterRecruiting
Nashville, Tennessee, United States
Md Anderson Cancer CenterRecruiting
Houston, Texas, United States
Fred Hutchinson Cancer CenterRecruiting
Seattle, Washington, United States
University of WashingtonRecruiting
Seattle, Washington, United States
Froedtert & the Medical College of WisconsinRecruiting
Milwaukee, Wisconsin, United States
Az Sint-Jan Brugge - Oostende Av - Campus Sint-JanRecruiting
Bruges, Belgium
Universitair Ziekenhuis Antwerpen (Uza)Recruiting
Edegem, Belgium
Jessa ZiekenhuisRecruiting
Hasselt, Belgium
Universitair Ziekenhuis (Uz) LeuvenRecruiting
Leuven, Belgium
Universitaire Ziekenhuis Leuven - GasthuisbergRecruiting
Leuven, Belgium
Centre Hospitalier Universitaire (Chu) de LiegeRecruiting
Liège, Belgium
AZ DELTARecruiting
Roeselare, Belgium
Arthur J E Child Comprehensive Cancer CentreRecruiting
Calgary, Alberta, Canada
Princess Margaret Cancer Centre - University Health NetworkRecruiting
Toronto, Ontario, Canada
Chu Sainte-JustineRecruiting
Montreal, Quebec, Canada
Vancouver General HospitalRecruiting
Vancouver, Canada
Klinikum Der Johann Wolfgang Goethe UniversityRecruiting
Frankfurt am Main, Germany
Universitatklinikum FreiburgRecruiting
Freiburg im Breisgau, Germany
Universitatsklinikum Hamburg EppendorfRecruiting
Hamburg, Germany
Universitaetsklinikum JenaRecruiting
Jena, Germany
University Hospital MannheimRecruiting
Mannheim, Germany
Universitaetsklinikum RegensburgRecruiting
Regensburg, Germany
Azienda Socio Sanitaria Territoriale Papa Giovanni XxiiiRecruiting
Bergamo, Italy
Aou Policlinico S. Orsola-MalpighiRecruiting
Bologna, Italy
Fondazione Irccs Ca Granda Ospedale Maggiore Policlinico Di Milano Uo DermatologiaRecruiting
Milan, Italy
Ospedale Pediatrico Bambino Gesu IrccsRecruiting
Rome, Italy
Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro CuoreRecruiting
Rome, Italy
Irccs Istituto Clinico HumanitasRecruiting
Rozzano, Italy
A.O.U. Citta Della Salute E Della Scienza Di TorinoRecruiting
Torino, Italy
Hospital General Universitario Vall D HebronWithdrawn
Barcelona, Spain
Hospital Clinic Barcelona MainRecruiting
Barcelona, Spain
Hospital Universitario Virgen de La ArrixacaRecruiting
El Palmar, Spain
Hospital Universitario Virgen de Las NievesRecruiting
Granada, Spain
Ico Institut Catala D OncologiaRecruiting
Granvia de L'hospitalet 199-203, Spain
Hospital General Universitario Gregorio MaranonRecruiting
Madrid, Spain
Hospital Universitario Ramon Y CajalRecruiting
Madrid, Spain
Hospital Universitario 12 de OctubreRecruiting
Madrid, Spain
Hospital Regional Universitario de MalagaRecruiting
Málaga, Spain
Hospital Clinico Universitario de SalamancaRecruiting
Salamanca, Spain
Hospital Universitario Marques de ValdecillaRecruiting
Santander, Spain
Hospital Universitario Virgen Del RocioRecruiting
Seville, Spain
Hospital Clinico Universitario de ValenciaRecruiting
Valencia, Spain
Hospital Universitari I Politecnic La FeRecruiting
Valencia, Spain
Queen Elizabeth HospitalRecruiting
Birmingham, United Kingdom
Cambridge University Hospitals Nhs Foundation TrustRecruiting
Cambridge, United Kingdom
University Hospital of WalesRecruiting
Cardiff, United Kingdom
Queen Elizabeth University HospitalRecruiting
Glasgow, United Kingdom
St James University HospitalRecruiting
Leeds, United Kingdom
Clatterbridge Cancer CenterRecruiting
Liverpool, United Kingdom
A Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease · TrialPath