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| Name | Class |
|---|---|
| CTI BioPharma | INDUSTRY |
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To evaluate the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of pacritinib in Asian subjects with myelofibrosis (MF), which includes primary MF (PMF), post-polycythemia vera MF (PPV-MF) or post-essential thrombocythemia MF (PET-MF).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Pacritinib | Experimental | Oral administration |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Pacritinib | Biological | QD (Once a day) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of participants achieving a ≥35% reduction in spleen volume | Measured by MRI or CT scan | Baseline to Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of participants with ≥50% reduction in total symptom score (TSS) | Measured by the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) TSS 2.0 | Baseline to Week 24 |
| Proportion of participants with baseline platelet count <100,000/μL achieving ≥35% reduction in spleen volume |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
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| ID | Term |
|---|---|
| D055728 | Primary Myelofibrosis |
| ID | Term |
|---|---|
| D009196 | Myeloproliferative Disorders |
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| ID | Term |
|---|---|
| C561234 | 11-(2-pyrrolidin-1-ylethoxy)-14,19-dioxa-5,7,26-triazatetracyclo(19.3.1.1(2,6).1(8,12))heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene |
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Measured by MRI or CT scan |
| Baseline to Week 24 |
| Proportion of participants with baseline platelet count <100,000/μL achieving ≥50% reduction in total symptom score (TSS) | Baseline to Week 24 |
| Proportion of participants with baseline platelet count <50,000/μL achieving ≥35% reduction in spleen volume | Measured by MRI or CT scan | Baseline to Week 24 |
| Proportion of participants with baseline platelet count <50,000/μL achieving ≥50% reduction in total symptom score (TSS) | Baseline to Week 24 |
| Clinically significant adverse events (AEs) | Throughout the study period of approximately 5 years |
| Clinically significant changes in laboratory results | Throughout the study period of approximately 5 years |
| Clinically significant changes in vital signs | Throughout the study period of approximately 5 years |
| Clinically significant changes in electrocardiograms (ECGs) | Throughout the study period of approximately 5 years |
| Pacritinib pharmacokinetic (PK) parameter: maximum observed concentration (Cmax) | Baseline; weeks 3, 12 & 24 |
| Pacritinib pharmacokinetic (PK) parameter: Time of maximum observed concentration (Tmax) | Baseline; weeks 3, 12 & 24 |
| Pacritinib pharmacokinetic (PK) parameter: minimum observed concentration (Cmin) | Baseline; weeks 3, 12 & 24 |
| Pacritinib pharmacokinetic (PK) parameter: area under the concentration curve (AUC) | Baseline; weeks 3, 12 & 24 |
| Pacritinib pharmacokinetic (PK) parameter: apparent volume of distribution (V/F) | Baseline; weeks 3, 12 & 24 |
| Pharmacodynamic parameter: Maximum observed effect (Emax) | Baseline; weeks 3, 12 & 24 |
| Pharmacodynamic parameter: time of maximum observed effect (tEmax) | Baseline; weeks 3, 12 & 24 |
| Pharmacodynamic parameter: area under the effect curve (AUEC) | Baseline; weeks 3, 12 & 24 |