Actively Recruiting

Phase 3
Age: 18Years +
All Genders
ID06615050

A Randomized Phase III Trial Comparing Tacrolimus/Methotrexate/Ruxolitinib Versus Post-Transplant Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil in Non-Myeloablative/Reduced Intensity Conditioning Allogeneic Peripheral Blood Stem Cell Transplantation

Led by Incyte Corporation · Updated on 2026-05-20

572

Participants Needed

30

Research Sites

N/A

Total Duration

On this page

Sponsors

I

Incyte Corporation

Lead Sponsor

B

Blood and Marrow Transplant Clinical Trials Network

Collaborating Sponsor

AI-Summary

What this Trial Is About

Researchers are evaluating two different drug combinations to prevent graft-versus-host disease (GVHD) in adults undergoing non-myeloablative or reduced intensity conditioning allogeneic peripheral blood stem cell transplantation (PBSC). The study compares the combination of Tacrolimus, Methotrexate, and Ruxolitinib with the combination of Post-Transplant Cyclophosphamide, Tacrolimus, and Mycophenolate Mofetil. This Phase III trial aims to find better ways to improve outcomes for patients receiving stem cell transplants for certain blood cancers and disorders. Participants receive one of the study drug regimens following their transplant. The experimental groups include three different doses of the Tacrolimus/Methotrexate/Ruxolitinib combination, while the active comparator group receives Post-Transplant Cyclophosphamide with Tacrolimus and Mycophenolate Mofetil. Drugs are administered by tablet or intravenous infusion as per protocol. The trial includes a dose-finding run-in period followed by the main randomized comparison of these treatment combinations. During the study, participants are closely monitored for up to 24 months after transplant to assess outcomes such as GVHD-free survival, incidence and severity of acute and chronic GVHD, time to blood cell recovery, donor cell engraftment, graft failure, relapse or progression of disease, mortality, infections, and treatment toxicities. Patient-reported outcomes including symptom scales, work productivity, and financial impact are also collected. Safety and efficacy are evaluated through regular clinical assessments and laboratory tests throughout the follow-up period.

CONDITIONS

Brief Title

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)

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Age 18 years or older at enrollment
  • Undergoing allogeneic hematopoietic cell transplantation for acute leukemia, chronic myelogenous leukemia, myelodysplasia, chronic myelomonocytic leukemia, or specified lymphomas
  • Receiving non-myeloablative or reduced intensity conditioning regimen
  • Have a related or unrelated peripheral blood stem cell donor meeting specified HLA matching criteria
  • Left ventricular ejection fraction of at least 45%
  • Estimated glomerular filtration rate greater than 60 ml/min/1.73 m2
  • Pulmonary function with DLCO corrected for hemoglobin at least 40% and FEV1 predicted at least 50%
  • Liver function with AST/ALT less than 3 times upper limit of normal and total bilirubin less than 2 mg/dL
  • Karnofsky Performance Score of at least 60%
  • Female participants agree to use two effective contraception methods or abstain from heterosexual intercourse unless postmenopausal or surgically sterilized
  • Male participants agree to use barrier contraception or abstain from heterosexual intercourse if partners of women of childbearing potential
  • Disclosure of planned post-transplant maintenance therapy excluding investigational agents and hypomethylating agents
  • Voluntary written informed consent obtained
Not Eligible

You will not qualify if you...

  • Prior allogeneic transplant
  • Active central nervous system involvement by malignant cells
  • Secondary AML arising from myeloproliferative neoplasms or overlap syndromes, except secondary AML from myelodysplastic neoplasm
  • Primary, post-Essential Thrombocythemia, and post-Polycythemia Vera myelofibrosis
  • Uncontrolled bacterial, viral, or fungal infections with progression or no improvement
  • Active or inadequately treated latent tuberculosis infection
  • Clinically significant fluid collections interfering with methotrexate use
  • HIV infection with detectable viral load (undetectable viral load allowed)
  • Uncontrolled hepatitis B or C virus infection (controlled cases with prophylaxis allowed)
  • Recent arterial or venous thrombosis or severe uncontrolled heart conditions
  • Pregnant or lactating females
  • Serious medical or psychiatric illness likely to interfere with participation
  • Prior malignancies except certain treated cancers
  • Planned use of ATG or alemtuzumab in conditioning
  • Planned prophylactic donor leukocyte infusions
  • Prior use of ruxolitinib or immune checkpoint inhibitors within 6 months
  • Presence of donor-specific antibodies or desensitization protocols for 7/8 HLA-matched donors
  • Treatment with other investigational medicinal products during study treatment

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Run-in Period

Duration - Duration as defined by protocol dosing schedules

Participants receive initial doses of Tacrolimus, Methotrexate, and Ruxolitinib to identify the appropriate dose level before starting the main treatment.

Visits according to dosing schedules

Treatment

Duration - Up to 24 months post-transplant

Participants receive assigned drug regimens post-transplant: either Tacrolimus/Methotrexate/Ruxolitinib or Post-transplant Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil to prevent graft-versus-host disease and support engraftment.

Multiple visits including baseline and follow-up visits during treatment

Follow-up

Duration - Up to 24 months post-transplant

Participants are monitored for outcomes such as graft-versus-host disease, relapse, survival, and treatment-related side effects after treatment ends.

Regular follow-up visits during this period

Trial Site Locations

Total: 30 locations

1

Stanford Cancer Center

Palo Alto, California, United States, 94304

Actively Recruiting

2

University of California San Francisco

San Francisco, California, United States, 94158

Actively Recruiting

3

University of Miami

Miami, Florida, United States, 33136

Actively Recruiting

4

Moffitt Cancer Center

Tampa, Florida, United States, 33612

Actively Recruiting

5

Blood and Marrow Transplant Group of Georgia

Atlanta, Georgia, United States, 30342

Actively Recruiting

6

Indiana University Cancer Center

Indianapolis, Indiana, United States, 46202

Actively Recruiting

7

University of Kansas Hospital Authority

Kansas City, Kansas, United States, 66160

Actively Recruiting

8

Massachusetts General Hospital

Boston, Massachusetts, United States, 02114

Actively Recruiting

9

Dana Farber Cancer Institute

Boston, Massachusetts, United States, 02215

Actively Recruiting

10

University of Michigan

Ann Arbor, Michigan, United States, 48109

Actively Recruiting

11

Karmanos Cancer Institute

Detroit, Michigan, United States, 48201

Actively Recruiting

12

Henry Ford Hospital

Detroit, Michigan, United States, 48202

Actively Recruiting

13

Washington University

St Louis, Missouri, United States, 63110

Actively Recruiting

14

Mount Sinai Hospital

New York, New York, United States, 10029

Actively Recruiting

15

Memorial Sloan Kettering

New York, New York, United States, 10065

Actively Recruiting

16

University of North Carolina At Chapel Hill

Chapel Hill, North Carolina, United States, 27514

Actively Recruiting

17

Levine Cancer Institute

Charlotte, North Carolina, United States, 28204

Actively Recruiting

18

Duke University Medical Center

Durham, North Carolina, United States, 27705

Actively Recruiting

19

Cleveland Clinic

Cleveland, Ohio, United States, 44195

Actively Recruiting

20

Ohio State University

Columbus, Ohio, United States, 43210

Actively Recruiting

21

Oregon Health & Science University

Portland, Oregon, United States, 97239

Actively Recruiting

22

University of Pennsylvania

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

23

Medical University of South Carolina

Charleston, South Carolina, United States, 29425

Actively Recruiting

24

Sarah Cannon

Nashville, Tennessee, United States, 37203

Actively Recruiting

25

Vanderbilt Medical Center

Nashville, Tennessee, United States, 37232

Actively Recruiting

26

Baylor College of Medicine

Houston, Texas, United States, 77030

Actively Recruiting

27

Virginia Commonwealth University, North Hospital

Richmond, Virginia, United States, 23298

Actively Recruiting

28

Fred Hutchinson Cancer Research Center

Seattle, Washington, United States, 98109

Actively Recruiting

29

University of Wisconsin

Madison, Wisconsin, United States, 53705

Actively Recruiting

30

Medical College of Wisconsin

Milwaukee, Wisconsin, United States, 53226

Actively Recruiting

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Research Team

I

Incyte Corporation Call Center (US)

I

Incyte Corporation Call Center (ex-US)

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

4

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