Study on Using Ruxolitinib Around Transplant Time for Patients with Primary and Secondary Myelofibrosis

Sponsor: Fred Hutchinson Cancer Center

Sponsor score: 40
Inactive

ClinicalTrials ID:NCT04384692

Provider View

This clinical trial is exploring whether taking the drug ruxolitinib before, during, and after a stem cell transplant can help prevent complications and improve outcomes for patients with primary and secondary myelofibrosis.

Patient Parameters

Program Overview

This study is investigating if the drug ruxolitinib, when given before, during, and after a stem cell transplant, can help prevent a condition called graft versus host disease (GVHD) and improve the success of the transplant in patients with myelofibrosis. Myelofibrosis is a type of bone marrow cancer, and stem cell transplant is currently the only treatment that can potentially cure it. However, there is a risk of GVHD, where the donor's cells attack the patient's body. Ruxolitinib may help reduce this risk and improve patients' quality of life by decreasing inflammation and symptoms like night sweats and weight loss.

Description

This study is divided into two parts:

Part 1: Patients will start taking ruxolitinib by mouth 8 weeks before their stem cell transplant and continue until about 14 days before the transplant preparation begins. The dose will be gradually reduced until 4 days before the transplant, unless there are side effects or the disease worsens. If patients join another study for Part 2, their data and samples from Part 1 will be used in the new study.

Part 2: Patients will receive either a high-intensity or reduced-intensity preparation for the transplant, based on their health and doctor's advice:

  • High-Intensity Preparation: Patients will receive chemotherapy drugs cyclophosphamide and busulfan, and possibly fludarabine if using umbilical cord blood for the transplant.
  • Reduced-Intensity Preparation: Patients will receive chemotherapy drugs fludarabine and melphalan, and possibly total body irradiation if using umbilical cord blood.

After preparation, patients will undergo the stem cell transplant. To prevent GVHD, patients will continue taking ruxolitinib for about 7 months after the transplant, gradually reducing the dose over 2 months. They will also receive other medications like tacrolimus and methotrexate to help prevent GVHD. Patients using umbilical cord blood will receive additional medication, mycophenolate mofetil.

Patients will have regular scans and tests, including CT scans, echocardiograms, bone marrow biopsies, and blood tests, to monitor their health and the transplant's success. Follow-up visits will occur at 6 months, 1 year, and annually for up to 5 years after the transplant.

Eligibility Criteria

Inclusion Criteria

  • Must be 18 years or older.
  • Diagnosed with primary or secondary myelofibrosis.
  • Considered a candidate for stem cell transplant by a doctor.
  • Willing to start ruxolitinib within 6 months.
  • Must have taken ruxolitinib for at least 8 weeks before transplant preparation.
  • Good overall health with a performance status score of 70 or higher.
  • Adequate kidney and liver function based on specific blood tests.
  • Good lung and heart function.
  • Low comorbidity index score (less than 5).

Exclusion Criteria

  • Allergic to ruxolitinib or have significant kidney or liver problems.
  • Have an active, uncontrolled infection.
  • HIV positive.
  • Pregnant or trying to conceive.
  • History of a previous stem cell transplant.
  • Have more than 20% leukemia cells (blasts).
  • Have an uncontrolled viral or bacterial infection at the time of transplant.
  • Have had a serious fungal infection in the past 6 months without specialist approval.
  • No suitable donor for the transplant.

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