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

Myelofibrosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06345495
• Other study ID #: 2023-0899
• Secondary ID: NCI-2024-02814
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: September 30, 2024
• Est. completion date: January 1, 2029

Study information

• Verified date: May 2024
• Source: M.D. Anderson Cancer Center
• Contact: Uday Popat, MD
• Phone: (713) 563-0812
• Email: upopat[@]mdanderson.org
• Is FDA regulated: No
• Study type: Interventional

Clinical 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.

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

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 30
• Est. completion date: January 1, 2029
• Est. primary completion date: January 1, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

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

5. Has a fully matched (8/8:HLA A, B, C, DRB1) related or matched unrelated donor

6. Female participants of childbearing potential must have negative results for pregnancy test.

Exclusion Criteria:

1. Ejection fraction <40%

2. Creatinine clearance < 40ml/min measured or calculated by Cockroft Gault formula

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. Prior 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: participants 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.

Related Conditions & MeSH terms

• Myelofibrosis
• Primary Myelofibrosis
• Splenomegaly

Intervention

Drug:
• Ruxolitinib
Given by PO

Procedure:
• Allogeneic Stem Cell Transplantation
Given by Transplant

Drug:
• Levetiracetam
Given by PO
• Eltrombopag
Given by PO
• Busulfan
Given by IV
• Romiplostim
Given by IV
• Fludarabine phosphate
Given by IV
• Cyclophosphamide
Given by IV
• Mesna
Given by IV
• Tacrolimus
Given by IV or PO

Locations

Country	Name	City	State
United States	MD Anderson Cancer Center	Houston	Texas

Sponsors (2)

Lead Sponsor	Collaborator
M.D. Anderson Cancer Center	Incyte Corporation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.

External Links

•   MD Anderson Cancer Center