A Randomized Controlled Phase 3 Study of Oral Pacritinib Versus Best Available Therapy in Patients With Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis
18 Years
N/A
Both
Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis
Trial Information
A Randomized Controlled Phase 3 Study of Oral Pacritinib Versus Best Available Therapy in Patients With Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis
Inclusion Criteria:
- Intermediate -1 or -2 or high-risk Myelofibrosis (per Passamonti et al 2010)
- Palpable splenomegaly ≥ 5 cm on physical examination
- Score ≥ 3 for at least two symptoms on the MPN-SAF TSS or score ≥ 4 for at least one
symptom on MPN-SAF TSS other than fatigue
- Patients who are platelet or red blood cell transfusion-dependent are eligible
- Adequate white blood cell counts (with low blast counts), liver function, and renal
function
- No spleen radiation therapy for 6-12 months
- Last therapy for myelofibrosis was 2-4 weeks ago, including any erythropoietic or
thrombopoietic agent
- Not pregnant, not lactating, and agree to use effective birth control
Exclusion Criteria:
- Prior treatment with a JAK2 inhibitor
- History of (or plans to undergo) spleen removal surgery or allogeneic stem cell
transplant
- Ongoing gastrointestinal medical condition such as Crohn's disease, Inflammatory
bowel disease, chronic diarrhea, or constipation
- Cardiovascular disease, including recent history or currently clinically symptomatic
and uncontrolled: congestive heart failure, arrhythmia, angina, QTc prolongation or
other QTc risk factors, myocardial infarction
- Other malignancy within last 3 years other than certain limited skin, cervical,
prostate, breast, or bladder cancers
- Other ongoing, uncontrolled illnesses (including HIV infection and active hepatitis
A, B, or C), psychiatric disorder, or social situation that would prevent good care
on this study
- Life expectancy < 6 months
Type of Study:
Interventional
Study Design:
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
Efficacy
Outcome Description:
To compare the efficacy of pacritinib with that of best available therapy (BAT) in patients with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF); the efficacy measure for this analysis is the proportion of patients achieving a ≥ 35% reduction in spleen volume from baseline to week 24 by magnetic resonance imaging (MRI) or computed tomography (CT)
Outcome Time Frame:
Baseline to Week 24
Safety Issue:
No
Principal Investigator
James Dean, MD, PhD
Investigator Role:
Study Director
Investigator Affiliation:
Cell Therapeutics
Authority:
United States: Food and Drug Administration
Study ID:
PERSIST-1 (PAC325)
NCT ID:
NCT01773187
Start Date:
December 2012
Completion Date:
August 2017
Related Keywords:
- Primary Myelofibrosis
- Post-Polycythemia Vera Myelofibrosis
- Post-Essential Thrombocythemia Myelofibrosis
- Myelofibrosis
- Post-Polycythemia Vera Myelofibrosis
- Post-Essential Thrombocythemia Myelofibrosis
- Primary Myelofibrosis
- Polycythemia
- Polycythemia Vera
- Thrombocythemia, Essential
- Thrombocythemia
- Myeloproliferative Disorders
- Bone Marrow Disease
- Hematologic Diseases
- Blood Platelet Disorders
- Hemorrhagic Disorders
- Splenomegaly
- Pacritinib
- MPN-SAF
- MPN-SAF TSS
- Anemia
- Myeloproliferative
- Myeloproliferative Neoplasm
- Spleen
- Spleen volume
- Thrombocytopenia
- SB1518
- Primary Myelofibrosis
- Polycythemia
- Polycythemia Vera
- Thrombocythemia, Essential
- Thrombocytosis
Name | Location |
|---|---|
| CTI Investigational Site 10001 | Morristown, New Jersey 07962 |
