A Study of Tacrolimus/Methotrexate/Ruxolitinib Versus Post-Transplant Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil in Non-Myeloablative/Reduced Intensity Conditioning Allogeneic Peripheral Blood Stem Cell Transplantation (BMT CTN 2203)

NCT ID: NCT06615050

Last Updated: 2025-11-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

572 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-04-02

Study Completion Date

2031-01-17

Brief Summary

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The purpose of this study is to assess Tacrolimus/Methotrexate/Ruxolitinib versus Post-Transplant Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil in Non-Myeloablative/Reduced Intensity Conditioning Allogeneic Peripheral Blood Stem Cell Transplantation

Detailed Description

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Conditions

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Graft-versus-host Disease (GVHD)

Keywords

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Graft-versus-host Disease (GVHD) Chronic GvHD (cGvHD) steroid-refractory ruxolitinib Janus kinase inhibitor

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Dose Finding Run-In Group 1: Tac/MTX/Ruxolitnib Dose 1

Tacrolimus/ methotrexate/ ruxolitinib (Tac/MTX/Rux) at the protocol defined doses.

Group Type EXPERIMENTAL

Tacrolimus (Tac)

Intervention Type DRUG

Tablet or intravenously (IV)

Methotrexate (MTX)

Intervention Type DRUG

Intravenously (IV)

Ruxolitinib (Rux)

Intervention Type DRUG

Tablet

Dose Finding Run-In Group 2: Tac/MTX/Ruxolitnib Dose 2

Tacrolimus/ methotrexate/ ruxolitinib (Tac/MTX/Rux) at the protocol defined doses.

Group Type EXPERIMENTAL

Tacrolimus (Tac)

Intervention Type DRUG

Tablet or intravenously (IV)

Methotrexate (MTX)

Intervention Type DRUG

Intravenously (IV)

Ruxolitinib (Rux)

Intervention Type DRUG

Tablet

Main Study Group A: Tac/MTX/Ruxolitnib

Tacrolimus/ methotrexate/ ruxolitinib (Tac/MTX/Rux) at the protocol defined doses.

Group Type EXPERIMENTAL

Tacrolimus (Tac)

Intervention Type DRUG

Tablet or intravenously (IV)

Methotrexate (MTX)

Intervention Type DRUG

Intravenously (IV)

Ruxolitinib (Rux)

Intervention Type DRUG

Tablet

Main Study Group B: PTCy/Tac/MMF

Post-transplant cyclophosphamide/ tacrolimus/ mycophenolate mofetil (PTCy/Tac/MMF) at the protocol defined doses.

Group Type ACTIVE_COMPARATOR

Tacrolimus (Tac)

Intervention Type DRUG

Tablet or intravenously (IV)

Cyclophosphamide

Intervention Type DRUG

Intravenously (IV)

Mycophenolate mofetil (MMF)

Intervention Type DRUG

Tablet or intravenously (IV)

Interventions

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Tacrolimus (Tac)

Tablet or intravenously (IV)

Intervention Type DRUG

Methotrexate (MTX)

Intravenously (IV)

Intervention Type DRUG

Ruxolitinib (Rux)

Tablet

Intervention Type DRUG

Cyclophosphamide

Intravenously (IV)

Intervention Type DRUG

Mycophenolate mofetil (MMF)

Tablet or intravenously (IV)

Intervention Type DRUG

Other Intervention Names

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Jakafi, INCB018424

Eligibility Criteria

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Inclusion Criteria

* Age 18.0 years or older at the time of enrollment.
* Participants undergoing allogeneic HCT for one of the following indications:

* Acute leukemia or chronic myelogenous leukemia with no circulating blasts and with less than 5% blasts in the bone marrow. Therapy related myeloid neoplasms are allowed.
* Myelodysplasia/chronic myelomonocytic leukemia with no circulating blasts and with less than 10% blasts in the bone marrow (higher blast percentage allowed in MDS due to lack of differences in outcomes with \< 5% versus 5-10% blasts in this disease). Therapy related myeloid neoplasms are allowed.
* Lymphoma \[follicular lymphoma, Hodgkin lymphoma, diffuse large B cell lymphoma, mantle cell lymphoma, peripheral T-cell lymphoma, angioimmunoblastic T-cell lymphoma and anaplastic large cell lymphoma\].
* Planned NMA/reduced intensity conditioning regimen.
* Participants must have a related or unrelated PBSC donor as follows:

* Sibling donor must be a 6/6 match for HLA-A and -B at intermediate (or higher) resolution, and -DRB1 at high resolution using DNA-based typing and must be willing to donate peripheral blood stem cells and meet institutional criteria for donation. HLA-matched parents and children may be used as donors.
* Unrelated donor must be a 7/8 or 8/8 match at HLA-A, -B, -C and -DRB1 at high resolution using DNA-based typing. Unrelated donor must be willing to donate peripheral blood stem cells and meet NMDP criteria for donation.
* Donor selection must comply with 21 CFR 1271.
* Cardiac function: Left ventricular ejection fraction at least 45%.
* Estimated creatinine clearance greater than 60 ml/min using the 2021 CKD-EPI formula or 24-hour urine creatinine clearance.
* Pulmonary function: DLCO corrected for hemoglobin at least 40% and FEV1 predicted at least 50%.
* Liver function: AST/ALT \< 3x ULN; Total bilirubin \< 2 mg/dL excluding Gilbert's syndrome or hemolysis.
* Karnofsky Performance Score of at least 60%.
* Female participants (unless postmenopausal for at least one year before the screening visit, or surgically sterilized), agree to practice two effective methods of contraception at the same time, or agree to completely abstain from heterosexual intercourse, from the time of signing the informed consent through 15 months post-transplant. Fertility preservation methods will be left to institutional standards.
* Male participants (even if surgically sterilized), of partners of women of childbearing potential must agree to one of the following: practice effective barrier contraception or abstain from heterosexual intercourse from the time of signing the informed consent through 15 months post-transplant.
* Plans for the use of targeted small molecule inhibitor post-transplant maintenance therapy must be disclosed upon enrollment and must be used irrespective of the outcome of the randomization. Planned use of investigational maintenance agents is not permitted. Planned hypomethylating agents as maintenance therapy is not permitted.
* Voluntary written consent obtained prior to the performance of any study-related procedure that is not a part of standard medical care, with the understanding that consent may be withdrawn by the participant at any time without prejudice to future medical care.

Exclusion Criteria

* Prior allogeneic transplant.
* Active CNS involvement by malignant cells.
* Participants with secondary AML arising from myeloproliferative neoplasms or overlap syndromes, including CMML and MDS/MPN syndromes; participants with secondary AML arising from myelodysplastic neoplasm are eligible.
* Participants with primary myelofibrosis.
* Participants with uncontrolled bacterial, viral, or fungal infections (currently taking medication and with progression or no clinical improvement) at time of enrollment.
* Active or inadequately treated latent infection with Mycobacterium tuberculosis (i.e., TB).
* Presence of clinically significant fluid collection (ascites, pleural or pericardial effusion) that interferes with methotrexate clearance or makes methotrexate use contraindicated.
* Participants seropositive for human immunodeficiency virus (HIV) with detectable viral load. HIV+ participants with an undetectable viral load on antiviral therapy are eligible.
* Evidence of uncontrolled hepatitis B virus (HBV) or hepatitis C virus (HCV). The study allows:

* Positive HBV serology with undetectable viral load and ongoing antiviral prophylaxis to prevent potential HBV reactivation.
* Positive HCV serology with quantitative PCR for plasma HCV RNA below the lower limit of detection, with or without concurrent antiviral HCV treatment.
* Arterial or venous thrombosis including DVT, PE, stroke, and myocardial infarction within six (6) months prior to enrollment or New York Heart Association (NYHA) Class III or IV heart failure, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia. Catheter-associated DVT is not exclusionary.
* Female participants who are pregnant (as per institutional practice) or lactating.
* Participants with a serious medical or psychiatric illness likely to interfere with participation in this clinical study.
* Participants with prior malignancies except resected non-melanoma skin cancer or treated cervical carcinoma in situ. Cancer treated with curative intent ≥ 5 years previously will be allowed. Cancer treated with curative intent \< 5 years previously must be reviewed and approved by the Protocol Officer or Chairs.
* Planned use of ATG or alemtuzumab in conditioning regimen.
* Planned use of prophylactic donor leukocyte infusions.
* Prior use of ruxolitinib.
* Prior use of immune checkpoint inhibitors (i.e., PD1, PDL1, CTLA4 modulators) within six (6) months prior to conditioning.
* For participants with 7/8 HLA-matched donors:

* Donor specific antibodies (DSAs) directed at the mismatched donor allele.
* Any use of desensitization protocols.
* Treatment with any other Investigational Medicinal Product (IMP) is not allowed while on study treatment. An IMP is defined as medications without any known FDA or EMA approved indications.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Blood and Marrow Transplant Clinical Trials Network

NETWORK

Sponsor Role collaborator

National Institutes of Health (NIH)

NIH

Sponsor Role collaborator

National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role collaborator

National Cancer Institute (NCI)

NIH

Sponsor Role collaborator

Incyte Corporation

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Incyte Medical Monitor

Role: STUDY_DIRECTOR

Incyte Corporation

Locations

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Stanford Cancer Center

Palo Alto, California, United States

Site Status RECRUITING

University of Miami

Miami, Florida, United States

Site Status RECRUITING

Moffitt Cancer Center

Tampa, Florida, United States

Site Status RECRUITING

Blood and Marrow Transplant Group of Georgia

Atlanta, Georgia, United States

Site Status RECRUITING

Indiana University Cancer Center

Indianapolis, Indiana, United States

Site Status RECRUITING

University of Kansas Hospital Authority

Kansas City, Kansas, United States

Site Status RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status RECRUITING

University of Michigan

Ann Arbor, Michigan, United States

Site Status RECRUITING

Karmanos Cancer Institute

Detroit, Michigan, United States

Site Status RECRUITING

Henry Ford Hospital

Detroit, Michigan, United States

Site Status RECRUITING

Washington University

St Louis, Missouri, United States

Site Status RECRUITING

Mount Sinai Hospital

New York, New York, United States

Site Status RECRUITING

Memorial Sloan Kettering

New York, New York, United States

Site Status RECRUITING

University of North Carolina At Chapel Hill

Chapel Hill, North Carolina, United States

Site Status RECRUITING

Levine Cancer Institute

Charlotte, North Carolina, United States

Site Status RECRUITING

Duke University Medical Center

Durham, North Carolina, United States

Site Status RECRUITING

Cleveland Clinic

Cleveland, Ohio, United States

Site Status RECRUITING

Ohio State University

Columbus, Ohio, United States

Site Status RECRUITING

Oregon Health & Science University

Portland, Oregon, United States

Site Status RECRUITING

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status RECRUITING

Vanderbilt Medical Center

Nashville, Tennessee, United States

Site Status RECRUITING

Baylor College of Medicine

Houston, Texas, United States

Site Status RECRUITING

Virginia Commonwealth University, North Hospital

Richmond, Virginia, United States

Site Status RECRUITING

Fred Hutchinson Cancer Research Center

Seattle, Washington, United States

Site Status RECRUITING

University of Wisconsin

Madison, Wisconsin, United States

Site Status RECRUITING

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Incyte Corporation Call Center (US)

Role: CONTACT

Phone: 1.855.463.3463

Email: [email protected]

Incyte Corporation Call Center (ex-US)

Role: CONTACT

Phone: +800 00027423

Email: [email protected]

Related Links

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https://www.incyteclinicaltrials.com/study/?id=INCB18424-370&SearchTerm=INCB18424-370&Latitude=&Longitude=&LocationName=&conditions=&Status=&phases=&AgeRanges=&gender=&StudyTypes=&AttachmentTypes=&MileRadius=100&PageIndex=0&PageSize=10&SortField=&SortOrder=&hasResults=

A Study of Tacrolimus/Methotrexate/Ruxolitinib Versus Post-Transplant Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil in Non-Myeloablative/Reduced Intensity Conditioning Allogeneic Peripheral Blood Stem Cell Transplantation (BMT CTN 2203)

Other Identifiers

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BMT CTN Protocol 2203

Identifier Type: OTHER

Identifier Source: secondary_id

INCB18424-370

Identifier Type: -

Identifier Source: org_study_id