Pacritinib Before Transplant for Myeloproliferative Neoplasms (MPN)
Condition: Myeloproliferative Diseases
MeSH terms: Neoplasms; Myeloproliferative Disorders
Intervention: Pacritinib; Busulfan; Questionnaires; Phone Calls; Allogeneic Stem Cell Transplantation; Fludarabine
Intervention description: 200 mg by mouth twice a day for 60 days.; Busulfan AUC of 4000 microMol-min per day providing that pharmacokinetic can be done, otherwise Busulfan dose given as a fixed dose of 100 mg/m2 daily for four days.; Questionnaires completed at baseline, 1, 3, 6, and 12 months after transplant.; Phone calls made by study staff to participant on second and third week of each month.; Allogeneic stem cell transplantation (Allo TP) 60 days after starting Pacritinib but not more than 180 days.; Fludarabine taken along with Busulfan as per standard of care as preparative regimen for allogeneic stem cell transplantation (Allo TP).
Intervention MeSH terms: Fludarabine; Fludarabine phosphate; Busulfan
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 4.0
Sex: All
Minimum age: 18 Years
Maximum age: 70 Years
Healthy volunteers: No
Keywords: Myeloproliferative Diseases; Myeloproliferative Neoplasms; MPN; Myelofibrosis; Polycythemia vera; PV; Essential thrombocythemia; Allogeneic stem cell transplantation; Allo TP; Pacritinib; Busulfan; Busulfex; Myleran; Questionnaires; Surveys; Phone calls

Summary:

      The goal of this clinical research study is to learn if giving pacritinib before standard of
      care drugs followed by an allogeneic stem cell transplant can help to control
      myeloproliferative neoplasms. The safety of this therapy will also be studied.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Patients with Idiopathic Myelofibrosis or Myelofibrosis secondary to Polycythemia Vera
             or Essential Thrombocythemia.

          2. Patients 18 years to less than or equal to 70 years.

          3. Patients wanting to pursue transplant.

          4. Patients must have a Zubrod PS equal or less than 2.

          5. Calculated creatinine clearance greater than 50ml/min. using the Cockcroft-Gault
             equation.

          6. Ejection fraction equal or above 40%.

          7. Serum direct bilirubin less than 1 mg/dl (unless due to Gilbert's syndrome or
             hemolysis).

          8. SGPT equal or less than 4 x normal values.

          9. Corrected DLCO equal or above 50% of expected.

         10. Negative Beta HCG test in a woman with childbearing potential defined as not
             post-menopausal for 12 months or no previous surgical sterilization) and if fertile,
             males and females must agree to use contraceptives.

        Exclusion Criteria:

          1. Patients with low risk myelofibrosis.

          2. Uncontrolled life-threatening infections.

          3. HIV positive.

          4. Patients with active viral hepatitis.

          5. Prior treatment with Pacritinib.

          6. Prior stem cell transplant.

          7. QTc greater than 450 ms.

          8. CYP3A4 strong or moderate inhibitors/inducers in the past 7 days.