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

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

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-10-01

Study Completion Date

2029-01-01

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Ruxolitinib Before, During and After Hematopoietic Cell Transplant in Older Patients With Myelofibrosis and Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes

NCT07228624

Myelodysplastic/Myeloproliferative Neoplasm Primary Myelofibrosis Secondary Myelofibrosis

RECRUITING PHASE2

Ruxolitinib Prior to Transplant in Patients With Myelofibrosis

NCT01790295

Primary Myelofibrosis Post Polycythemia Vera Myelofibrosis Post Essential Thrombocythemia Myelofibrosis

TERMINATED PHASE2

Ruxolitinib in Combination With Autotransplant

NCT02469974

Myelofibrosis MF

WITHDRAWN NA

Peritransplant Ruxolitinib for Patients With Primary and Secondary Myelofibrosis

NCT04384692

Primary Myelofibrosis Secondary Myelofibrosis

RECRUITING PHASE2

JAK Inhibitor Before Donor Stem Cell Transplant in Treating Patients With Primary or Secondary Myelofibrosis

NCT02251821

Primary Myelofibrosis Secondary Myelofibrosis

ACTIVE_NOT_RECRUITING PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Splenomegaly Myelofibrosis

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

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.

Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No