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

Launched by M.D. ANDERSON CANCER CENTER · Apr 2, 2024

Keywords

ClinConnect Summary

This clinical trial is investigating whether a high dose of a drug called ruxolitinib, combined with another medication called busulfan, can help reduce the size of the spleen and improve the success of stem cell transplants in patients with myelofibrosis. Myelofibrosis is a condition that affects blood cell production and can cause an enlarged spleen, known as splenomegaly. The trial aims to see if this treatment approach can make the transplant process more effective for patients.

To participate in the trial, individuals must be between 18 and 75 years old, have been diagnosed with myelofibrosis, and have a noticeably enlarged spleen. They also need to have a suitable donor for the stem cell transplant. Participants will be closely monitored throughout the study, and both men and women of childbearing potential must follow specific guidelines regarding contraception. This trial is currently recruiting participants, and those who join can expect to contribute to important research that may improve treatments for myelofibrosis in the future.

Gender

ALL

Eligibility criteria

• 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.