Using high doses of ruxolitinib and stem cell transplant for myelofibrosis patients with enlarged spleens

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study aims to evaluate the effectiveness of administering high doses of ruxolitinib and busulfan prior to allogeneic stem cell transplantation in patients with myelofibrosis and splenomegaly. The primary objective is to compare the survival and engraftment rates of participants at 100 days post-transplant against historical data. Secondary objectives include assessing overall survival, progression-free survival, and various complications related to the transplant process. The study also explores immune reconstitution and cytokine profiles as exploratory objectives.

Why it matters

Who can join: the full rules

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.

Where it's running

• MD Anderson Cancer Center
  Houston, Texas, United States Recruiting

Study contacts

• Principal investigator: Uday Popat, MD — M.D. Anderson Cancer Center
• Study coordinator: Uday Popat, MD
• Email: upopat@mdanderson.org
• Phone: (713) 563-0812

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Elritercept vs. placebo for adults with myelofibrosis and anemia on ruxolitinib

  This study tests whether elritercept can improve anemia in adults with myelofibrosis who are taking ruxolitinib, compared with placebo.

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• Ropeginterferon add-on to ruxolitinib in myelofibrosis

  This trial tests whether adding ropeginterferon alfa-2b to ongoing ruxolitinib treatment is safe and helpful for adults with myelofibrosis.

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• MPN PROGRESSion Registry — tracking symptoms, treatments, and disease progression in people with MPNs.

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  Recruiting at 2 sites MPN Research Foundation · Chicago, Illinois and 1 other locations · NCT07362225

• Nelfinavir to improve anemia in people with myelofibrosis by restoring iron delivery to the bone marrow

  This trial tests whether the antiviral drug nelfinavir can help people with myelofibrosis and anemia by raising hemoglobin and lowering blood markers of fibrosis.

  Recruiting Phase 1/2 University of California, Irvine · Orange, California · NCT07281781

• National registry for people with myelofibrosis and anemia in Germany.

  This project will collect health, treatment, and quality-of-life information from people in Germany with primary or secondary myelofibrosis and anemia to see how their care and symptoms change over time.

  Recruiting iOMEDICO AG · Multiple Locations · NCT06976918

• Combining ruxolitinib and ulixertinib for treating myelofibrosis

  This study is testing if combining two medications, ruxolitinib and ulixertinib, can help people with myelofibrosis who haven't had enough relief from ruxolitinib alone.

  Recruiting at 8 sites Phase 1/2 Memorial Sloan Kettering Cancer Center · Boston, Massachusetts and 8 other locations · NCT06773195

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.