Clinical trial · Interventional
Pacritinib Versus Best Available Therapy to Treat Patients With Myelofibrosis and Thrombocytopenia
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
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
Phase 3, randomized, controlled study to evaluate the safety and efficacy of oral pacritinib compared to Best Available Therapy (BAT) in patients with thrombocytopenia and primary or secondary myelofibrosis.
Conditions
Conditions (3)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Post-essential Thrombocythemia Myelofibrosis | — | UNRESOLVED | — |
| Post-polycythemia Vera Myelofibrosis | Polycythemia Vera, Post-Polycythemic Myelofibrosis Phase | ALIAS | 0.90 |
| Primary Myelofibrosis | Primary Myelofibrosis | ONTOLOGY_EXACT | 0.98 |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Best Available Therapy | Drug | — | UNRESOLVED |
| Pacritinib | Drug | Pacritinib | ALIAS |
Design
Arms and outcomes
Arms (3)
- type
- EXPERIMENTAL
- label
- Pacritinib, Once Daily
- description
- Pacritinib 400 mg QD
- interventionNames
- Drug: Pacritinib
- type
- EXPERIMENTAL
- label
- Pacritinib, Twice Daily
- description
- Pacritinib 200 mg BID
- interventionNames
- Drug: Pacritinib
- type
- ACTIVE_COMPARATOR
- label
- Best Available Therapy
- description
- 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.
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
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
References
Publications (4)
- BACKGROUNDMascarenhas J, Hoffman R, Talpaz M, Gerds AT, Stein B, Gupta V, Szoke A, Drummond M, Pristupa A, Granston T, Daly R, Al-Fayoumi S, Callahan JA, Singer JW, Gotlib J, Jamieson C, Harrison C, Mesa R, Verstovsek S. Pacritinib vs Best Available Therapy, Including Ruxolitinib, in Patients With Myelofibrosis: A Randomized Clinical Trial. JAMA Oncol. 2018 May 1;4(5):652-659. doi: 10.1001/jamaoncol.2017.5818. PMID 29522138
- DERIVEDAjufo H, Bewersdorf JP, Harrison C, Palandri F, Mascarenhas J, Palmer J, Gerds A, Kiladjian JJ, Buckley S, Derkach A, Roman-Torres K, Rampal RK. Pacritinib Response Is Associated With Overall Survival in Myelofibrosis: PERSIST-2 Landmark Analysis of Survival. Eur J Haematol. 2025 Feb;114(2):238-247. doi: 10.1111/ejh.14321. Epub 2024 Oct 14. PMID 39400386
- DERIVEDVerstovsek S, Mesa R, Talpaz M, Kiladjian JJ, Harrison CN, Oh ST, Vannucchi AM, Rampal R, Scott BL, Buckley SA, Craig AR, Roman-Torres K, Mascarenhas JO. Retrospective analysis of pacritinib in patients with myelofibrosis and severe thrombocytopenia. Haematologica. 2022 Jul 1;107(7):1599-1607. doi: 10.3324/haematol.2021.279415. PMID 34551507
- DERIVEDTremblay D, Mesa R, Scott B, Buckley S, Roman-Torres K, Verstovsek S, Mascarenhas J. Pacritinib demonstrates spleen volume reduction in patients with myelofibrosis independent of JAK2V617F allele burden. Blood Adv. 2020 Dec 8;4(23):5929-5935. doi: 10.1182/bloodadvances.2020002970. PMID 33275766