Clinical trial · Interventional
Pacritinib Versus Best Available Therapy to Treat Myelofibrosis
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
NCT01773187CI-TRIAL-00047388terminatedPhase 3Results postedClinicalTrials.gov clinicaltrialsProvenance
- 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 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 (2)
- type
- EXPERIMENTAL
- label
- Pacritinib
- description
- Pacritinib 400 mg QD
- interventionNames
- Drug: Pacritinib
- type
- ACTIVE_COMPARATOR
- label
- Best Available Therapy
- description
- BAT includes any physician-selected treatment for primary or secondary myelofibrosis with the exclusion of JAK inhibitors (inhibitors of Janus kinases)
- interventionNames
- Drug: Best Available Therapy
Primary outcomes (1)
- measure
- Spleen Volume Reduction
- timeFrame
- Baseline to Week 24
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) * 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 * 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
References
Publications (2)
- BACKGROUNDMesa RA, Vannucchi AM, Mead A, Egyed M, Szoke A, Suvorov A, Jakucs J, Perkins A, Prasad R, Mayer J, Demeter J, Ganly P, Singer JW, Zhou H, Dean JP, Te Boekhorst PA, Nangalia J, Kiladjian JJ, Harrison CN. Pacritinib versus best available therapy for the treatment of myelofibrosis irrespective of baseline cytopenias (PERSIST-1): an international, randomised, phase 3 trial. Lancet Haematol. 2017 May;4(5):e225-e236. doi: 10.1016/S2352-3026(17)30027-3. Epub 2017 Mar 20. PMID 28336242
- 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