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 | Recruiting Phase 2 FDA Drug

• 2 other identifiers: INCA34176-2542022-502168-19-00
• Study Type: interventional
• Target: 120
• Locations: 7 countries
• Sites: 70

Brief Summary

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.

Conditions

Chronic Graft-versus-host-disease

Keywords

cGVHD

Outcome Measures

Primary Outcomes (1)

• Objective Response Rate

  Defined as Complete Response (CR) or Partial Response (PR) at 6 months in the absence of new systemic therapy for cGVHD. Response assessment will be based on the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD.

  6 months

Secondary Outcomes (10)

• Number of participants with Treatment-emergent Adverse Events (TEAEs)

  Up to 2 years and 30 days

• Duration of Response

  Up to 2 years

• Proportion of participants with a ≥ 7-point improvement in modified Lee symptom scale (mLSS) score

  Up to 2 years

• Best overall response in the first 6 months

  Up to 6 months

• OR at 12 months, defined as CR or PR at 12 months (C14D1) in the absence of new systemic therapy for cGVHD.

  12 months

Study Arms (3)

Treatment Group A

EXPERIMENTAL

Axatilimab will be administered at a protocol defined starting dose plus ruxolitinib at a protocol defined starting dose.

Drug: Axatilimab; Drug: Ruxolitinib

Treatment Group B

EXPERIMENTAL

Ruxolitinib will be administered at a protocol defined starting dose.

Drug: Ruxolitinib

Treatment Group C

EXPERIMENTAL

Corticosteroids alone will be administered at a protocol defined starting dose.

Drug: Corticosteroids

Interventions

Axatilimab DRUG

Axatilimab will be administered at protocol defined dose.

Treatment Group A

Ruxolitinib DRUG

Ruxolitinib will be administered at protocol defined dose.

Treatment Group A; Treatment Group B

Corticosteroids DRUG

Corticosteroids will be administered at protocol defined dose.

Also known as: prednisone, prednisolone, methylprednisolone

Treatment Group C

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

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 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Incyte Corporation: lead

Study Sites (70)

Mayo Clinic Hospital

Phoenix, Arizona, 85054, United States

RECRUITING

City of Hope Medical Center

Duarte, California, 91010, United States

RECRUITING

University of California-Los Angeles Medl Cntr-Oncology Center Bowyer Clinic

Los Angeles, California, 90095, United States

RECRUITING

Stanford Cancer Center

Stanford, California, 94305, United States

RECRUITING

University of Colorado Cancer Center

Aurora, Colorado, 80045, United States

RECRUITING

Colorado Blood Cancer Institute

Denver, Colorado, 80218, United States

RECRUITING

Smilow Cancer Center-Yale

New Haven, Connecticut, 06510, United States

RECRUITING

Mayo Clinic Jacksonville

Jacksonville, Florida, 32224, United States

RECRUITING

Northwestern University

Chicago, Illinois, 60611, United States

RECRUITING

The University of Chicago Medicine

Chicago, Illinois, 60637, United States

RECRUITING

University of Maryland-Greenebaum Cancer Center

Baltimore, Maryland, 21201, United States

RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, 02144, United States

WITHDRAWN

Washington University

St Louis, Missouri, 63110, United States

RECRUITING

Fred and Pamela Buffett Cancer Center

Omaha, Nebraska, 68198, United States

RECRUITING

Memorial Sloan Kettering Cancer Center

New York, New York, 10021, United States

RECRUITING

Weill Cornell Medicine

New York, New York, 10021, United States

RECRUITING

Mount Sinai Hospital

New York, New York, 10029, United States

RECRUITING

Cleveland Clinic

Cleveland, Ohio, 44195, United States

RECRUITING

The Ohio State University

Columbus, Ohio, 43210, United States

RECRUITING

University of Pennsylvania Abramson Cancer Center

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Hillman Cancer Center

Pittsburgh, Pennsylvania, 15232, United States

RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

RECRUITING

Md Anderson Cancer Center

Houston, Texas, 77030, United States

RECRUITING

Fred Hutchinson Cancer Center

Seattle, Washington, 98109, United States

RECRUITING

University of Washington

Seattle, Washington, 98195, United States

RECRUITING

Froedtert & the Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

RECRUITING

Az Sint-Jan Brugge - Oostende Av - Campus Sint-Jan

Bruges, 08000, Belgium

RECRUITING

Universitair Ziekenhuis Antwerpen (Uza)

Edegem, 02650, Belgium

RECRUITING

Jessa Ziekenhuis

Hasselt, 03500, Belgium

RECRUITING

Universitair Ziekenhuis (Uz) Leuven

Leuven, 03000, Belgium

RECRUITING

Universitaire Ziekenhuis Leuven - Gasthuisberg

Leuven, 03000, Belgium

RECRUITING

Centre Hospitalier Universitaire (Chu) de Liege

Liège, 04000, Belgium

RECRUITING

AZ DELTA

Roeselare, 08800, Belgium

RECRUITING

Arthur J E Child Comprehensive Cancer Centre

Calgary, Alberta, T3N 4N1, Canada

RECRUITING

Princess Margaret Cancer Centre - University Health Network

Toronto, Ontario, M5G 2M9, Canada

RECRUITING

Chu Sainte-Justine

Montreal, Quebec, H3T 1C5, Canada

RECRUITING

Vancouver General Hospital

Vancouver, V5Z1M9, Canada

RECRUITING

Klinikum Der Johann Wolfgang Goethe University

Frankfurt am Main, 60590, Germany

RECRUITING

Universitatklinikum Freiburg

Freiburg im Breisgau, 79106, Germany

RECRUITING

Universitatsklinikum Hamburg Eppendorf

Hamburg, 20251, Germany

RECRUITING

Universitaetsklinikum Jena

Jena, 07747, Germany

RECRUITING

University Hospital Mannheim

Mannheim, 68167, Germany

RECRUITING

Universitaetsklinikum Regensburg

Regensburg, 93053, Germany

RECRUITING

Azienda Socio Sanitaria Territoriale Papa Giovanni Xxiii

Bergamo, 24127, Italy

RECRUITING

Aou Policlinico S. Orsola-Malpighi

Bologna, 40138, Italy

RECRUITING

Fondazione Irccs Ca Granda Ospedale Maggiore Policlinico Di Milano Uo Dermatologia

Milan, 20122, Italy

RECRUITING

Ospedale Pediatrico Bambino Gesu Irccs

Rome, 00165, Italy

RECRUITING

Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro Cuore

Rome, 00168, Italy

RECRUITING

Irccs Istituto Clinico Humanitas

Rozzano, 20089, Italy

RECRUITING

A.O.U. Citta Della Salute E Della Scienza Di Torino

Torino, 10126, Italy

RECRUITING

Hospital General Universitario Vall D Hebron

Barcelona, 08035, Spain

WITHDRAWN

Hospital Clinic Barcelona Main

Barcelona, 08036, Spain

RECRUITING

Hospital Universitario Virgen de La Arrixaca

El Palmar, 30120, Spain

RECRUITING

Hospital Universitario Virgen de Las Nieves

Granada, 18014, Spain

RECRUITING

Ico Institut Catala D Oncologia

Granvia de L'hospitalet 199-203, 08908, Spain

RECRUITING

Hospital General Universitario Gregorio Maranon

Madrid, 28007, Spain

RECRUITING

Hospital Universitario Ramon Y Cajal

Madrid, 28034, Spain

RECRUITING

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

RECRUITING

Hospital Regional Universitario de Malaga

Málaga, 29011, Spain

RECRUITING

Hospital Clinico Universitario de Salamanca

Salamanca, 37007, Spain

RECRUITING

Hospital Universitario Marques de Valdecilla

Santander, 39008, Spain

RECRUITING

Hospital Universitario Virgen Del Rocio

Seville, 41013, Spain

RECRUITING

Hospital Clinico Universitario de Valencia

Valencia, 46010, Spain

RECRUITING

Hospital Universitari I Politecnic La Fe

Valencia, 46026, Spain

RECRUITING

Queen Elizabeth Hospital

Birmingham, B15 2TH, United Kingdom

RECRUITING

Cambridge University Hospitals Nhs Foundation Trust

Cambridge, CB2 0QQ, United Kingdom

RECRUITING

University Hospital of Wales

Cardiff, CF14 4XW, United Kingdom

RECRUITING

Queen Elizabeth University Hospital

Glasgow, G51 4TF, United Kingdom

RECRUITING

St James University Hospital

Leeds, LS9 7TF, United Kingdom

RECRUITING

Clatterbridge Cancer Center

Liverpool, L7 8YA, United Kingdom

RECRUITING

Related Links

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

MeSH Terms

Conditions

Bronchiolitis Obliterans Syndrome

Interventions

axatilimab; ruxolitinib; Adrenal Cortex Hormones; Prednisone; Prednisolone; Methylprednisolone

Condition Hierarchy (Ancestors)

Organizing Pneumonia; Bronchiolitis Obliterans; Bronchiolitis; Bronchitis; Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Graft vs Host Disease; Immune System Diseases

Intervention Hierarchy (Ancestors)

Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Pregnadienetriols

Study Officials

• Incyte Medical Monitor

  Incyte Corporation

  STUDY DIRECTOR

Central Study Contacts

Incyte Corporation Call Center (US)

CONTACT

1.855.463.3463; medinfo@incyte.com

Incyte Corporation Call Center (ex-US)

CONTACT

+800 00027423; eumedinfo@incyte.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: April 25, 2024
• First Posted: April 29, 2024
• Study Start: October 11, 2024
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): December 1, 2029
• Last Updated: March 23, 2026

Record last verified: 2026-03

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.

Locations

BE (7); CA (4); DE (6); US (26); IT (7); ES (14); GB (6)