Pacritinib Versus Best Available Therapy to Treat Myelofibrosis

NCT ID: NCT01773187

Last Updated: 2020-09-29

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 327 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-01-31
• Study Completion Date: 2016-04-30

Brief Summary

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.

Detailed Description

Multicenter, randomized, controlled, phase 3 trial comparing the safety and efficacy of pacritinib with that of BAT in patients with primary or secondary myelofibrosis. Approximately 322 eligible patients will be randomized in a 2:1 allocation to pacritinib (400mg QD) or BAT (includes any physician-selected treatment for myelofibrosis with the exclusion of JAK inhibitors (inhibitors of Janus kinases)). Spleen volume will be measured by MRI or CT at baseline and every 12 weeks thereafter. An independent radiology facility (IRF), blind to treatment assignments, will measure spleen volumes. Patients will also be followed for safety, Leukemia Free Survival (LFS), Overall Survival (OS), frequency of red blood cell (RBC) and platelet transfusions, and other exploratory endpoints. An Independent Data Monitoring Committee (IDMC) will evaluate the safety of pacritinib.

Conditions

Primary Myelofibrosis; Post-polycythemia Vera Myelofibrosis; Post-essential Thrombocythemia Myelofibrosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Pacritinib

Pacritinib 400 mg QD

Group Type: EXPERIMENTAL

Pacritinib

Intervention Type: DRUG

Best Available Therapy

BAT includes any physician-selected treatment for primary or secondary myelofibrosis with the exclusion of JAK inhibitors (inhibitors of Janus kinases)

Group Type: ACTIVE_COMPARATOR

Best Available Therapy

Intervention Type: DRUG

Interventions

Pacritinib

Intervention Type: DRUG

Best Available Therapy

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

CTI BioPharma

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Beth Ziemba

Role: STUDY_DIRECTOR

VP, Pharmacovigilance, Clinical Operations, QA

Locations

CTI Investigational Site 10002

Scottsdale, Arizona, United States

CTI Investigational Site 10004

Omaha, Nebraska, United States

CTI Investigational Site 10001

Morristown, New Jersey, United States

CTI Investigational Site 10003

Greenville, South Carolina, United States

CTI Investigational Site 61006

Box Hill, , Australia

CTI Investigational Site 61001

Coffs Harbour, , Australia

CTI Investigational Site 61005

Geelong, , Australia

CTI Investigational Site 61003

Gosford, , Australia

CTI Investigational Site 61004

Hobart, , Australia

CTI Investigational Site 61002

Milton, , Australia

CTI Investigational Site 32002

Antwerp, , Belgium

CTI Investigational Site 32003

Antwerp, , Belgium

CTI Investigational Site 32001

Bruges, , Belgium

CTI Investigational Site 32005

Brussels, , Belgium

CTI Investigational Site 32004

La Louvière, , Belgium

CTI Investigational Site 42003

Brno, , Czechia

CTI Investigational Site 42001

Olomouc, , Czechia

CTI Investigational Site 42002

Pilsen, , Czechia

CTI Investigational Site 42004

Prague, , Czechia

CTI Investigational Site 33005

Amiens, , France

CTI Investigational Site 33006

Caen, , France

CTI Investigational Site 33011

Grenoble, , France

CTI Investigational Site 33012

Lens, , France

CTI Investigational Site 33007

Lille, , France

CTI Investigational Site 33001

Nîmes, , France

CTI Investigational Site 33004

Paris, , France

CTI Investigational Site 33008

Paris, , France

CTI Investigational Site 33009

Pessac, , France

CTI Investigational Site 33010

Pierre-Bénite, , France

CTI Investigational Site 33003

Strasbourg, , France

CTI Investigational Site 33002

Toulouse, , France

CTI Investigational Site 49006

Berlin, , Germany

CTI Investigational Site 49007

Berlin, , Germany

CTI Investigational Site 49001

Cologne, , Germany

CTI Investigational Site 49003

Dresden, , Germany

CTI Investigational Site 49008

Essen, , Germany

CTI Investigational Site 49002

Freiburg im Breisgau, , Germany

CTI Investigational Site 49005

Mainz, , Germany

CTI Investigational Site 49004

München, , Germany

CTI Investigational Site 49009

Münster, , Germany

CTI Investigational Site 36002

Budapest, , Hungary

CTI Investigational Site 36005

Debrecen, , Hungary

CTI Investigational Site 36006

Gyula, , Hungary

CTI Investigational Site 36003

Kaposvár, , Hungary

CTI Investigational Site 36004

Kecskemét, , Hungary

CTI Investigational Site 36001

Szeged, , Hungary

CTI Investigational Site 36008

Szolnok, , Hungary

CTI Investigational Site 36007

Szombathely, , Hungary

CTI Investigational Site 39003

Bologna, , Italy

CTI Investigational Site 39001

Florence, , Italy

CTI Investigational Site 39005

Milan, , Italy

CTI Investigational Site 39004

Monza, , Italy

CTI Investigational Site 39002

Padua, , Italy

CTI Investigational Site 39008

Reggio Emilia, , Italy

CTI Investigational Site 39006

Rimini, , Italy

CTI Investigational Site 31001

Amsterdam, , Netherlands

CTI Investigational Site 31002

Maastricht, , Netherlands

CTI Investigational Site 31003

Rotterdam, , Netherlands

CTI Investigational Site 31004

Utrecht, , Netherlands

CTI Investigational Site 64001

Christchurch, , New Zealand

CTI Investigational Site 64004

Dunedin, , New Zealand

CTI Investigational Site 64002

Hamilton, , New Zealand

CTI Investigational Site 64003

Takapuna, , New Zealand

CTI Investigational Site 70011

Izhevsk, , Russia

CTI Investigational Site 70008

Moscow, , Russia

CTI Investigational Site 70009

Moscow, , Russia

CTI Investigational Site 70002

Petrozavodsk, , Russia

CTI Investigational Site 70001

Saint Petersburg, , Russia

CTI Investigational Site 70004

Saint Petersburg, , Russia

CTI Investigational Site 70010

Saint Petersburg, , Russia

CTI Investigational Site 70005

Samara, , Russia

CTI Investigational Site 70006

Sochi, , Russia

CTI Investigational Site 70007

Volgograd, , Russia

CTI Investigational Site 44004

Birmingham, , United Kingdom

CTI Investigational Site 44008

Bournemouth, , United Kingdom

CTI Investigational Site 44002

Cambridge, , United Kingdom

CTI Investigational Site 44003

Cardiff, , United Kingdom

CTI Investigational Site 44001

London, , United Kingdom

CTI Investigational Site 44007

London, , United Kingdom

CTI Investigational Site 44006

Manchester, , United Kingdom

CTI Investigational Site 44005

Oxford, , United Kingdom

Countries

United States; Australia; Belgium; Czechia; France; Germany; Hungary; Italy; Netherlands; New Zealand; Russia; United Kingdom

References

Mesa 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.

Reference Type: BACKGROUND

PMID: 28336242 (View on PubMed)

Tremblay 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.

Reference Type: DERIVED

PMID: 33275766 (View on PubMed)

Other Identifiers

PERSIST-1 (PAC325)

Identifier Type: -

Identifier Source: org_study_id