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SplenomegalyPhase 2NCT06345495

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

A Phase 2 study testing Ruxolitinib, Allogeneic Stem Cell Transplantation and Levetiracetam in people with Splenomegaly and Myelofibrosis, run by M.D. Anderson Cancer Center at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record

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Trial summary

To learn if giving ruxolitinib and busulfan before a stem cell transplant can help to reduce spleen size and help the transplant to succeed. Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Splenomegaly, Myelofibrosis
What is being tested?
Ruxolitinib, Allogeneic Stem Cell Transplantation and Levetiracetam
Could I receive a placebo?
No, everyone receives an active treatment
How many people
30 participants
Where
1 siteUnited States
Study dates
1 Oct 2024to 1 Jan 2029
Phase
Phase 2

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Participants 18 years to less than or equal to 75 years.
  • Able to provide written consent.
  • Primary or secondary Myelofibrosis (may have received Jak inhibitors including ruxolitinib)
  • 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.
  • Has a fully matched (8/8:HLA A, B, C, DRB1) related or matched unrelated donor.
  • Adequate renal function, including:
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  • Serum creatinine </= 1.5 mg/dL or estimated Glomerular Filtration Rate (eGFR using the CKI-EPI equation) >/= 40 ml/min/1.73 m2.
  • Adequate liver function, including:
  • ALT/AST </= 3 x ULN
  • Direct bilirubin </= 1mg/dL
  • No history of liver cirrhosis. No ascites.
  • 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.
  • 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:
  • 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.
  • 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.

Who cannot join

  • Positive beta HCG in females of child-bearing potential defined as not postmenopausal for 24 months or no previous surgical sterilization or lactating females.
  • Ejection fraction <40%
  • Corrected DLCO < 50%
  • Evidence of other clinically significant uncontrolled condition(s) including, but not limited to:
  • Uncontrolled and/or active systemic infection (viral, bacterial or fungal)
  • Active hepatitis B virus (HBV), hepatitis C (HCV), HIV or TB infection or requiring treatment for the same.
  • 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.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Uday Popat, MD contact

(713) 563-0812

upopat@mdanderson.org

United States1 site, 1 recruiting

  • MD Anderson Cancer Center

    Houston, Texas, 77030

    Uday Popat, MD, 713-563-0812, upopat@mdanderson.org

    Uday Popat, MD

    Recruiting

Other trials in United States

About this study

To learn if giving ruxolitinib and busulfan before a stem cell transplant can help to reduce spleen size and help the transplant to succeed.

Read the 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
Being tested
Ruxolitinib, Allogeneic Stem Cell Transplantation, Levetiracetam, Eltrombopag, Busulfan
Medical subject headings
Splenomegaly, Primary Myelofibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • RuxolitinibDrug

    Also called Jakafi, INCB018424, INC424

    Given by PO

  • Allogeneic Stem Cell TransplantationProcedure or surgery

    Also called Autologous stem cell transplant, Cord Blood

    Given by Transplant

  • LevetiracetamDrug

    Given by PO

  • EltrombopagDrug

    Also called Promacta®

    Given by PO

  • BusulfanDrug

    Also called Busulfex™, Myleran®

    Given by IV

  • RomiplostimDrug

    Also called AMG 531, Nplate

    Given by IV

  • Fludarabine phosphateDrug

    Also called Fludarabine, Fludara®

    Given by IV

  • CyclophosphamideDrug

    Also called Cytoxan®, Neosar®

    Given by IV

  • MesnaDrug

    Also called Mesnex™

    Given by IV

  • TacrolimusDrug

    Also called Prograf®

    Given by IV or PO

Study groups in NCT06345495
Study groupRoleWhat they receive
Ruxolitinib and Allogeneic Stem Cell TransplantationExperimentalParticipants 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.
Receives: Drug: Ruxolitinib; Procedure: Allogeneic Stem Cell Transplantation; Drug: Levetiracetam; Drug: Eltrombopag; Drug: Busulfan; Drug: Romiplostim; Drug: Fludarabine phosphate; Drug: Cyclophosphamide; Drug: Mesna; Drug: Tacrolimus

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safety and adverse events (AEs)

    Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0

    Measured: Through study completion; an average of 1 year.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Apr 2024
  2. Study start1 Oct 2024
  3. Primary completion1 Jan 2027 (estimated)
  4. Study completion1 Jan 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
30 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06345495
Sponsor's protocol ID
2023-0899
Other IDs
NCI-2024-02814
Status last verified
Sep 2026
First submitted
28 Mar 2024
Last updated
16 Sep 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

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