Myelofibrosis (MF)
Conditions
Interventions
Pacritinib 200 mg (2 capsules) BID orally, at the same time of day, with or without food
Sponsors
Ito Masayuki
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Primary MF, post-polycythemia vera MF, or post-essential thrombocythemia MF 2. Platelet count of -5 cm below the lower costal margin in the midclavicular line as assessed by physical examination 5. TSS of >-10 on the MPN-SAF TSS 2.0 or a single symptom score of >-5 or two symptoms of >-3, including only the symptoms of left upper quadrant pain, bone pain, itching, or night sweats. The TSS criteria need only to be met on a single day. 6. Age >-18 years 7. Eastern Cooperative Oncology Group performance status 0 to 2 8. Provision of signed informed consent
Exclusion criteria
Exclusion criteria: 1. Life expectancy <6 months 2. Completed allogeneic stem cell transplant, or are eligible for and willing to complete other approved available therapy including allogeneic stem cell transplant 3. History of splenectomy or planning to undergo splenectomy 4. Splenic irradiation within the last 6 months 5. Previously treated with pacritinib 6. Treatment with any MF-directed therapy within 14 days prior to treatment Day 1 7. Prior treatment with more than one JAK2 inhibitor Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. To compare the efficacy of pacritinib with that of P/C therapy, as assessed by the proportion of participants achieving a >-35% SVR from baseline at Week 24 as measured by MRI (preferred) or CT scans. 2. To compare the efficacy of pacritinib with P/C therapy, as assessed by the proportion of participants achieving a >-50% reduction in TSS from baseline at Week 24 | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. To compare the percentage of participants who self-assess as ''very much improved'' or ''much improved'' as measured by the Patient Global Impression of Change (PGIC) in participants treated with pacritinib versus those treated with P/C 2. To compare the OS of participants treated with pacritinib versus those treated with P/C 3. To compare the safety of pacritinib versus P/C therapy | — |
Countries
Australia, Belarus, Bosnia and Herzegovina, Bulgaria, Canada, Czechia, Federal Republic of Brazil, France, Georgia, Hungary, India, Israel, Italy, Japan, Kazakhstan, Korea, Poland, Romania, Russian Federation, Serbia, Spain, Ukraine, United Kingdom, United States
Contacts
Public ContactMasayuki Ito
Swedish Orphan Biovitrum Japan Co., Ltd
Outcome results
None listed