myelofibrosis myeloproliferative diseases
Conditions
Interventions
The Pacritinib group:
The start dose is 4 x 100 mg capsules Pacritinib, once per day orally, at the
same time of day, with or without food.
A maximum of two dose reductions is allowed. The first dose
Sponsors
CTI BioPharma Corp.
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: 1). Intermediate 1 or 2 or high-risk PMF, PPV-MF, or PET-MF (Passamonti et al 2010) 2). Palpable splenomegaly * 5 cm below LCM in midclavicular line by physical examination 3). Total Symptom Score (TSS) * 13 on the MPN-SAF-TSS2.0, not including te inactivity question. 4). Age * 18 years old 5). ECOG performance status 0-3 6). Peripheral blast count 500/*L 8). Patients who are platelet or red blood cell transfusion-dependent are eligible 9). Adequate liver and renal function, defined by liver transaminases (AST/SGOT and ALT/SGPT) * 3 × ULN (AST/ALT * 5 × ULN if transaminase elevation is related to MF), direct bilirubin * 4 x ULN, and creatinine * 2.5 mg/dL 10). At least 6 months from prior splenic irradiation 11). At least 12 months from prior 32P therapy 12). At least 1 week since prior treatment (most recent dose) with a potent CYP3A4 inhibitor 13). At least 4 weeks since any experimental treatment for PMF, PPV-MF, or PET-MF 14). At least 2 weeks since any treatment for PMF, PPV-MF, or PET-MF 15). If fertile, both males and females must agree to use effective birth control. Women of childbearing potential must use highly effective methods (defined as those resulting in a failure rate of
Exclusion criteria
Exclusion criteria: 1). Any GI or metabolic condition that could interfere with absorption of oral medication 2). Life expectancy 450 ms or other factors that increase the risk for QT prolongation (eg, heart failure, serum potassium
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Study objective: Primary efficacy objective: - The proportion of patients achieving a * 35% reduction in spleen volume from baseline to Week 24 by MRI or CT. | — |
Secondary
| Measure | Time frame |
|---|---|
| The key secundary objective is the proportion of patients with 50% reduction in total score from baseline to Week 24 on the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF TSS2.0). Other secundary objectives are: - Proportion of patients with a baseline platelet count 35% reduction in spleen volume from baseline to Week 24 as measured by MRI or CT - Proportion of patients with a baseline platelet count 50% reduction in the TSS from baseline to Week 24 - Proportion of patients with a baseline platelet count 35% reduction in spleen volume from baseline to Week 24 as measured by MRI or CT - Proportion of patients with a baseline platelet count 50% reduction in in the TSS from baseline to Week 24 | — |
Countries
Netherlands
Outcome results
None listed