Primary Myelofibrosis, Post-essential thrombocythemia myelofibrosis with Severe Thrombocytopenia (Platelet Counts <50,000/µL), Post polycythaemia vera myelofibrosis MedDRA version: 20.0 Level: PT Classification code 10077161 Term: Primary myelofibrosis System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) MedDRA version: 21.1 Level: LLT Classification code 10074691 Term: Post polycythaemia vera myelofibrosis System Organ Class: 10029104 - Neoplasms
Conditions
Interventions
Product Name: Pacritinib
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Pacritinib
CAS Number: 937272-79-2
Current Sponsor code: SB1518
Other descriptive name: PACRITINIB
Concentration unit:
Sponsors
CTI BioPharma Corp.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.PMF, PPV-MF, or PET-MF (as defined by Tefferi and Vardiman 2008) 2.Platelet count of 10 mg ofruxolitinib on any day during that interval. The 90- or 180-day period may overlap with the Screening period but may not extend into the washout period (14 days prior to treatment Day 1). 7.Age =18 years 8.Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2 9.Peripheral blast count of =65 years) yes F.1.3.1 Number of subjects for this age range 209
Exclusion criteria
Exclusion criteria: 1. Life expectancy 450 ms or other factors that increase the risk for QT interval prolongation (e.g., heart failure, hypokalemia [defined as serum potassium <3.0 mEq/L that is persistent and refractory to correction], or history of long QT interval syndrome) 16.New York Heart Association Class II, III, or IV congestive heart failure 17.Any active GI or metabolic condition that could interfere with absorption of oral medication 18.Active or uncontrolled inflammatory or chronic functional bowel disorder such as Crohn’s disease, inflammatory bowel disease, chronic diarrhea, or chronic constipation 19.Other malignancy within 3 years prior to treatment Day 1, other than curatively treated basal cell or squamous cell skin or corneal cancer; curatively treated carcinoma in situ of the cervix; organ-confined prostate cancer with prostate-specific antigen (PSA) <20 ng/mL and National Comprehensive Cancer Network risk of Very Low, Low, or Favorable Intermediate; curatively treated non-metastatic prostate cancer with negative PSA; or in situ breast carcinoma after complete surgical resection 20.Uncontrolled intercurrent illness, including, but not limited
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is to compare the efficacy of pacritinib with that of P/C therapy, as assessed by the proportion of patients achieving a =35% SVR between baseline and Week 24 as measured by MRI (preferred) or CT scans.;Secondary Objective: The secondary objectives of the study are as follows: 1. To compare the efficacy of pacritinib with P/C therapy, as assessed by the proportion of patients achieving a =50% reduction in TSS between baseline and Week 24 2. To compare the percentage of patients who self-assess as “very much improved” or “much improved” as measured by the Patient Global Impression of Change (PGIC) in patients treated with pacritinib versus those treated with P/C 3. To compare the OS of patients treated with pacritinib versus those treated with P/C 4. To compare the safety of pacritinib versus P/C therapy;Primary end point(s): The primary efficacy endpoint is the spleen volume reduction response rate defined as the percentage of patients who achieve at least 35% reduction in spleen volume from baseline as measured by MRI (preferred) or CT scan at Week 24. Patients who do not have a Week 24 spleen assessment will be included as non-responders in this analysis.;Timepoint(s) of evaluation of this end point: The primary analysis of spleen volume reduction will occur using data from the first 168 patients after they have been followed for 24 weeks. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The secondary efficacy endpoint of Total Symptom Score (TSS) response rate is defined as the percentage of patients with at least 50% reduction in TSS from baseline at Week 24. The TSS endpoint score will be calculated as the sum of scores for six items from the MPN SAF TSS 2.0 instrument: satiety, abdominal discomfort, night sweats, itching, bone pain, and pain under ribs on the left side. The secondary endpoint of Patient Global Impression of Change response rate is defined as the percentage of patients who self-assess as “very much improved” or “much improved” at Week 24. The secondary endpoint of Overall Survival is defined as the time between randomization and death. ;Timepoint(s) of evaluation of this end point: Week 24 | — |
Countries
Belarus, Canada, Czech Republic, France, Georgia, Germany, Hungary, Israel, Italy, Korea, Republic of, Poland, Russian Federation, Serbia, Spain, Ukraine, United Kingdom, United States
Contacts
Public ContactRegulatory Affairs
CTI BioPharma Corp.
Outcome results
None listed