High Dose Ruxolitinib and Allogeneic Stem Cell Transplantation in Myelofibrosis Patients With Splenomegaly

March 9, 2026 updated by: M.D. Anderson Cancer Center
To learn if giving ruxolitinib and busulfan before a stem cell transplant can help to reduce spleen size and help the transplant to succeed.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Primary Objective

1) Compare the proportion of patients alive, disease free, engrafted, and without poor graft function at 100 days post-transplant with the historical rate of 45%.

Secondary Objectives:

  1. Overall survival
  2. Progression-free survival
  3. Graft vs host disease relapse free survival
  4. Relapse rate
  5. Non-relapse Mortality
  6. Time to Neutrophil and platelet engraftment
  7. Time to red cell transfusion independence
  8. Graft failure
  9. Acute and chronic GVHD
  10. Grade 3 -5 Toxicity
  11. Incidence of poor graft function5
  12. Need for growth factors (myeloid or thrombopoietic) at 100 days
  13. Spleen response around day -7, -1, 30, and 100 days
  14. Need for transfusions at 100 days
  15. Time to discontinuation of immunosuppressives

Exploratory Objectives:

  1. Immune reconstitution
  2. Cytokine profile

Study Type

Interventional

Enrollment (Estimated)

30

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • Md Anderson Cancer Center
        • Contact:
        • Principal Investigator:
          • Uday Popat, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Participants 18 years to less than or equal to 75 years.
  2. Able to provide written consent.
  3. Primary or secondary Myelofibrosis (may have received Jak inhibitors including ruxolitinib)
  4. Enlarged spleen by palpation or imaging. For the purpose of this study, splenomegaly is defined as any clinically palpable spleen or spleen larger than 12 cms on imaging.
  5. Has a fully matched (8/8:HLA A, B, C, DRB1) related or matched unrelated donor.

  6. Adequate renal function, including:

    a. Serum creatinine </= 1.5 mg/dL or estimated Glomerular Filtration Rate (eGFR using the CKI-EPI equation) >/= 40 ml/min/1.73 m2.

  7. Adequate liver function, including:

    1. ALT/AST </= 3 x ULN
    2. Direct bilirubin </= 1mg/dL
    3. No history of liver cirrhosis. No ascites.
  8. Female participants of childbearing potential must have negative results for a serum pregnancy test. Female participants must agree to not breastfeed during the study and for 3 months post-completion of the study therapy.

  9. Subjects who are of childbearing potential, sexually active, and at risk of pregnancy must agree to use a highly effective method of contraception for the duration of the active treatment and at least 3 months post-completion of the study therapy. Highly effective methods of contraception include the following:

    1. Hormonal contraception (i.e., birth control pills, injection, implant, transdermal patch, vaginal ring), Intrauterine device (IUD), Tubal Ligation or hysterectomy, Subject/Partner post vasectomy, Implantable or injectable contraceptives, and condoms plus spermicide. Not engaging in sexual activity for the total duration of the trial and the drug washout period is an acceptable practice; however periodic abstinence, the rhythm method, and the withdrawal method are not acceptable methods of birth control. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
    2. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of study agent administration. Men who are able to have children must use effective birth control while on the study. If the male participant fathers a child or suspects that he has fathered a child while on the study, he must immediately notify his doctor.

Exclusion Criteria:

  1. Positive beta HCG in females of child-bearing potential defined as not postmenopausal for 24 months or no previous surgical sterilization or lactating females.
  2. Ejection fraction <40%
  3. Corrected DLCO < 50%

  4. Evidence of other clinically significant uncontrolled condition(s) including, but not limited to:

    1. Uncontrolled and/or active systemic infection (viral, bacterial or fungal)
    2. Active hepatitis B virus (HBV), hepatitis C (HCV), HIV or TB infection or requiring treatment for the same.
    3. Thrombosis including MI, Stroke, PE, DVT in the past 6 months

Note: subjects with serologic evidence of prior vaccination to HBV (i.e. hepatitis B surface (HBs) antigen negative-, anti-HBs antibody positive and anti-hepatitis B core (HBc) antibody negative) or positive anti-HBc antibody from intravenous immunoglobulins (IVIG) may participate.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Ruxolitinib and Allogeneic Stem Cell Transplantation
Participants will be asked to receive study drugs and a stem cell transplant and attend study visits, at which various tests and procedures will be performed. Participants are expected to receive treatment for about 100 days, followed by a year of follow-up.
Drug: Cyclophosphamide
Given by IV
Other Names:
  • Cytoxan®
  • Neosar®
Drug: Fludarabine phosphate
Given by IV
Other Names:
  • Fludara®
  • Fludarabine
Drug: Mesna
Given by IV
Other Names:
  • Mesnex™
Drug: Ruxolitinib
Given by PO
Other Names:
  • Jakafi
  • INCB018424
  • INC424
Procedure: Allogeneic Stem Cell Transplantation
Given by Transplant
Other Names:
  • Autologous stem cell transplant
  • Cord Blood
Drug: Levetiracetam
Given by PO
Drug: Eltrombopag
Given by PO
Other Names:
  • Promacta®
Drug: Busulfan
Given by IV
Other Names:
  • Myleran®
  • Busulfex™
Drug: Romiplostim
Given by IV
Other Names:
  • Nplate
  • AMG 531
Drug: Tacrolimus
Given by IV or PO
Other Names:
  • Prograf®

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety and adverse events (AEs)
Time Frame: Through study completion; an average of 1 year.
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Through study completion; an average of 1 year.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

M.D. Anderson Cancer Center

Collaborators

Incyte Corporation

Investigators

  • Principal Investigator: Uday Popat, MD, M.D. Anderson Cancer Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 1, 2024

Primary Completion (Estimated)

January 1, 2027

Study Completion (Estimated)

January 1, 2029

Study Registration Dates

First Submitted

March 28, 2024

First Submitted That Met QC Criteria

April 2, 2024

First Posted (Actual)

April 3, 2024

Study Record Updates

Last Update Posted (Actual)

March 11, 2026

Last Update Submitted That Met QC Criteria

March 9, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2023-0899
  • NCI-2024-02814 (Other Identifier: NCI-CTRP Clinical Registry)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Myelofibrosis

Clinical Trials on Cyclophosphamide

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Saudi Arabia

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe