Asian Phase II Study of INC424 in Patients With Primary Myelofibrosis (MF), Post-PV MF or Post-ET MF
Condition: Primary Myelofibrosis (MF); Post-Polycythemia Vera (PV) MF; Post-Essential Thrombocythemia (ET) MF
MeSH terms: Polycythemia Vera; Primary Myelofibrosis; Polycythemia; Thrombocytosis; Thrombocythemia, Essential
Intervention: Ruxolitinib
Intervention description: INC424 Tablet for oral use, provided in 5 mg bottles. The dosage strength was 5 mg/tablet INC424 phosphate (free base equivalent).
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 120.0
Sex: All
Minimum age: 18 Years
Maximum age: 65 Years
Healthy volunteers: No
Keywords: Primary Myelofibrosis (MF); Post-Polycythemia Vera (PV) MF; Post-Essential Thrombocythemia (ET) MF; post-PV MF; post-ET MF; INC424; Ruxolitinib; Myelofibrosis; MF

Summary:

      The objective of this study was to determine the efficacy of INC424 as assessed by reduction
      in spleen volume in patients with primary myelofibrosis (MF), post-polycythemia vera (PV) MF,
      or post-essential thrombocythemia (ET) MF. The safety and tolerability of INC424 and the
      effects of INC424 on patient reported outcomes and the duration of response as assessed by
      reduction in spleen volume was also assessed.
    

Eligibility criteria:

        Inclusion Criteria:

          1. 18 years or older

          2. Diagnosis of primary myelofibrosis (MF), post-polycythemia vera (PV) MF, or
             post-essential thrombocythemia (ET) MF

          3. Enlarged spleen, measuring 5 cm or greater from the costal margin

          4. Must have two or more of the following risk factors:

               1. Over 65 years old

               2. Have the following symptoms often associated with MF: loss of weight, fever,
                  night sweats

               3. Have a low red blood cell count (anemia - hemoglobin < 10 g/dL)

               4. Have a high white blood cell count (history of white blood cell count >
                  25,000/uL)

               5. Have high circulating blasts (> or = 1%) as measured by blood tests

          5. Should have circulating blasts <10% (as measured by blood tests)

          6. Should be capable of self-care

          7. Should have adequate bone marrow reserve

          8. Should not have the option of stem cell transplantation

          9. Should discontinue any prior or ongoing treatment for myelofibrosis prior to entering
             the study

         10. Had no prior treatment with another JAK inhibitor

        Exclusion Criteria:

          1. Does not have adequate liver or kidney function (as measured by blood tests)

          2. Has an active infection (bacterial, viral, etc.)

          3. Has active hepatitis A, B, or C or positive for HIV

          4. Has another cancer that needs active intervention

          5. Had a history of bleeding disorder

          6. Had a history of very low platelet counts (as measured by blood tests) not related to
             treatment of MF

          7. Had radiation of the spleen within 1 year of joining the study

          8. Does not have adequate heart function

          9. Sufficient time has elapsed between stopping previous treatment for MF and joining the
             study

         10. Females who are pregnant or breast-feeding

         11. Not able to sign informed consent

         12. Has any other active medical conditions that the doctor deems may compromise your
             safety or ability to join in the study