Pacritinib Versus Best Available Therapy to Treat Patients With Myelofibrosis and Thrombocytopenia (PAC326)
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ClinicalTrials.gov Identifier: NCT02055781 |
Recruitment Status :
Terminated
First Posted : February 5, 2014
Results First Posted : November 18, 2021
Last Update Posted : November 18, 2021
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Primary Myelofibrosis Post-polycythemia Vera Myelofibrosis Post-essential Thrombocythemia Myelofibrosis | Drug: Pacritinib Drug: Best Available Therapy | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 311 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Randomized Controlled Phase 3 Study of Oral Pacritinib Versus Best Available Therapy in Patients With Thrombocytopenia and Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis |
Actual Study Start Date : | February 2014 |
Actual Primary Completion Date : | April 2016 |
Actual Study Completion Date : | April 2016 |
Arm | Intervention/treatment |
---|---|
Experimental: Pacritinib, Once Daily
Pacritinib 400 mg QD
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Drug: Pacritinib |
Experimental: Pacritinib, Twice Daily
Pacritinib 200 mg BID
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Drug: Pacritinib |
Active Comparator: Best Available Therapy
BAT includes any physician-selected treatment for myelofibrosis, such as approved JAK2 inhibitors administered according to package insert for patients with thrombocytopenia, and may include any treatment received before study entry.
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Drug: Best Available Therapy |
- Spleen Volume Reduction [ Time Frame: Baseline to Week 24 ]Proportion of patients achieving a ≥ 35% reduction in spleen volume from baseline to week 24 as measured by magnetic resonance imaging (MRI) or computed tomography (CT).
- Total Symptom Score (TSS) Reduction [ Time Frame: Baseline to Week 24 ]Proportion of patients achieving a ≥ 50% reduction in the total symptom score from baseline to Week 24 on the Myeloproliferative Neoplasm Symptom Assessment Form 2.0 (MPN-SAF TSS 2.0). Responses (on a scale from 0 [absent] to 10 [worst imaginable]) to questions about symptoms (tiredness, early satiety, abdominal discomfort, night sweats, pruritus, bone pain, and pain under the ribs on the left side) were used to calculate the TSS.
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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Intermediate -1 or -2 or high-risk Myelofibrosis (per Passamonti et al 2010)
- Thrombocytopenia (platelet count ≤ 100,000/µL) at any time after signing informed consent
- Palpable splenomegaly ≥ 5 cm on physical examination
- Total Symptom Score ≥ 13 on the MPN-SAF TSS 2.0, not including the inactivity question
- 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
- At least 6 months from prior splenic irradiation
- At least 1-4 weeks since prior myelofibrosis therapy, including any erythropoietic or thrombopoietic agent
- Not pregnant, not lactating, and agree to use effective birth control
- Able and willing to undergo frequent MRI or CT assessments and complete symptom assessments using a patient-reported outcome instrument
Exclusion Criteria:
- Prior treatment with more than 2 JAK2 inhibitors or with pacritinib
- There is no maximum cumulative prior JAK2 inhibitor treatment
- 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
- Active bleeding that requires hospitalization during the screening period
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02055781
Study Director: | Simran Singh | Sr. Director, Head of Clinical Operations |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | CTI BioPharma |
ClinicalTrials.gov Identifier: | NCT02055781 |
Other Study ID Numbers: |
PERSIST-2 (PAC326) |
First Posted: | February 5, 2014 Key Record Dates |
Results First Posted: | November 18, 2021 |
Last Update Posted: | November 18, 2021 |
Last Verified: | October 2021 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Myelofibrosis Post-Polycythemia Vera Myelofibrosis Post-Essential Thrombocythemia Myelofibrosis Primary Myelofibrosis Polycythemia Vera Essential Thrombocythemia Bone Marrow Disease Hematologic Diseases Hemorrhagic Disorders |
Splenomegaly Pacritinib MPN-SAF MPN-SAF TSS Anemia Myeloproliferative Neoplasm Spleen volume Thrombocytopenia SB1518 |
Polycythemia Vera Thrombocytopenia Primary Myelofibrosis Polycythemia Thrombocytosis Thrombocythemia, Essential Blood Platelet Disorders Hematologic Diseases Cytopenia |
Myeloproliferative Disorders Bone Marrow Diseases Bone Marrow Neoplasms Hematologic Neoplasms Neoplasms by Site Neoplasms Blood Coagulation Disorders Hemorrhagic Disorders |