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

NCT06345495 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: 2023-0899NCI-2024-02814
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Splenomegaly; Myelofibrosis

Outcome Measures

Primary Outcomes (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

  Through study completion; an average of 1 year.

Study Arms (1)

Ruxolitinib and Allogeneic Stem Cell Transplantation

EXPERIMENTAL

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: Ruxolitinib; Procedure: Allogeneic Stem Cell Transplantation; Drug: Levetiracetam; Drug: Eltrombopag; Drug: Busulfan; Drug: Romiplostim; Drug: Fludarabine phosphate; Drug: Cyclophosphamide; Drug: Mesna; Drug: Tacrolimus

Interventions

Ruxolitinib DRUG

Given by PO

Also known as: Jakafi, INCB018424, INC424

Ruxolitinib and Allogeneic Stem Cell Transplantation

Levetiracetam DRUG

Given by PO

Ruxolitinib and Allogeneic Stem Cell Transplantation

Allogeneic Stem Cell Transplantation PROCEDURE

Given by Transplant

Also known as: Autologous stem cell transplant, Cord Blood

Ruxolitinib and Allogeneic Stem Cell Transplantation

Eltrombopag DRUG

Given by PO

Also known as: Promacta®

Ruxolitinib and Allogeneic Stem Cell Transplantation

Busulfan DRUG

Given by IV

Also known as: Busulfex™, Myleran®

Ruxolitinib and Allogeneic Stem Cell Transplantation

Romiplostim DRUG

Given by IV

Also known as: AMG 531, Nplate

Ruxolitinib and Allogeneic Stem Cell Transplantation

Fludarabine phosphate DRUG

Given by IV

Also known as: Fludarabine, Fludara®

Ruxolitinib and Allogeneic Stem Cell Transplantation

Cyclophosphamide DRUG

Given by IV

Also known as: Cytoxan®, Neosar®

Ruxolitinib and Allogeneic Stem Cell Transplantation

Mesna DRUG

Given by IV

Also known as: Mesnex™

Ruxolitinib and Allogeneic Stem Cell Transplantation

Tacrolimus DRUG

Given by IV or PO

Also known as: Prograf®

Ruxolitinib and Allogeneic Stem Cell Transplantation

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• a. 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• Incyte Corporation: collaborator

Study Sites (1)

MD Anderson Cancer Center

Houston, Texas, 77030, United States

RECRUITING

Related Links

• MD Anderson Cancer Center

MeSH Terms

Conditions

Splenomegaly; Primary Myelofibrosis

Interventions

ruxolitinib; Levetiracetam; eltrombopag; Busulfan; romiplostim; fludarabine phosphate; fludarabine; Cyclophosphamide; Mesna; Tacrolimus

Condition Hierarchy (Ancestors)

Hypertrophy; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms; Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Acetamides; Amides; Organic Chemicals; Acetates; Acids, Acyclic; Carboxylic Acids; Pyrrolidinones; Pyrrolidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Butylene Glycols; Glycols; Alcohols; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Phosphoramides; Organophosphorus Compounds; Sulfhydryl Compounds; Macrolides; Lactones

Study Officials

• Uday Popat, MD

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Uday Popat, MD

CONTACT

(713) 563-0812; upopat@mdanderson.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 28, 2024
• First Posted: April 3, 2024
• Study Start: October 1, 2024
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): January 1, 2029
• Last Updated: March 11, 2026

Record last verified: 2026-03

Locations

US (1)