Pacritinib Versus Best Available Therapy to Treat Myelofibrosis

NCT01773187 | Terminated Phase 3 Results DMC FDA Drug

• 1 other identifier: PERSIST-1 (PAC325)
• Study Type: interventional
• Target: 327
• Locations: 12 countries
• Sites: 81

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.

Conditions

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

Keywords

Myelofibrosis; Post-Polycythemia Vera Myelofibrosis; Post-Essential Thrombocythemia Myelofibrosis; Primary Myelofibrosis; Polycythemia Vera; Essential Thrombocythemia; Bone Marrow Disease; Hematologic Disease; Splenomegaly; Pacritinib; MPN-SAF; MPN-SAF TSS; Anemia; Myeloproliferative Neoplasm; Spleen volume; Thrombocytopenia; SB1518

Outcome Measures

Primary Outcomes (1)

• Spleen Volume Reduction

  Number 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)

  Baseline to Week 24

Secondary Outcomes (1)

• Total Symptom Score (TSS) Reduction

  Baseline to Week 24

Study Arms (2)

Pacritinib

EXPERIMENTAL

Pacritinib 400 mg QD

Drug: Pacritinib

Best Available Therapy

ACTIVE COMPARATOR

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

Drug: Best Available Therapy

Interventions

Pacritinib DRUG

Pacritinib

Best Available Therapy DRUG

Best Available Therapy

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• CTI BioPharma: lead

Study Sites (81)

CTI Investigational Site 10002

Scottsdale, Arizona, 85259, United States

Location

CTI Investigational Site 10004

Omaha, Nebraska, 68198, United States

Location

CTI Investigational Site 10001

Morristown, New Jersey, 07962, United States

Location

CTI Investigational Site 10003

Greenville, South Carolina, 29601, United States

Location

CTI Investigational Site 61006

Box Hill, Australia

Location

CTI Investigational Site 61001

Coffs Harbour, Australia

Location

CTI Investigational Site 61005

Geelong, Australia

Location

CTI Investigational Site 61003

Gosford, Australia

Location

CTI Investigational Site 61004

Hobart, Australia

Location

CTI Investigational Site 61002

Milton, Australia

Location

CTI Investigational Site 32002

Antwerp, Belgium

Location

CTI Investigational Site 32003

Antwerp, Belgium

Location

CTI Investigational Site 32001

Bruges, Belgium

Location

CTI Investigational Site 32005

Brussels, Belgium

Location

CTI Investigational Site 32004

La Louvière, Belgium

Location

CTI Investigational Site 42003

Brno, Czechia

Location

CTI Investigational Site 42001

Olomouc, Czechia

Location

CTI Investigational Site 42002

Pilsen, Czechia

Location

CTI Investigational Site 42004

Prague, Czechia

Location

CTI Investigational Site 33005

Amiens, France

Location

CTI Investigational Site 33006

Caen, France

Location

CTI Investigational Site 33011

Grenoble, France

Location

CTI Investigational Site 33012

Lens, France

Location

CTI Investigational Site 33007

Lille, France

Location

CTI Investigational Site 33001

Nîmes, France

Location

CTI Investigational Site 33004

Paris, France

Location

CTI Investigational Site 33008

Paris, France

Location

CTI Investigational Site 33009

Pessac, France

Location

CTI Investigational Site 33010

Pierre-Bénite, France

Location

CTI Investigational Site 33003

Strasbourg, France

Location

CTI Investigational Site 33002

Toulouse, France

Location

CTI Investigational Site 49006

Berlin, Germany

Location

CTI Investigational Site 49007

Berlin, Germany

Location

CTI Investigational Site 49001

Cologne, Germany

Location

CTI Investigational Site 49003

Dresden, Germany

Location

CTI Investigational Site 49008

Essen, Germany

Location

CTI Investigational Site 49002

Freiburg im Breisgau, Germany

Location

CTI Investigational Site 49005

Mainz, Germany

Location

CTI Investigational Site 49004

München, Germany

Location

CTI Investigational Site 49009

Münster, Germany

Location

CTI Investigational Site 36002

Budapest, Hungary

Location

CTI Investigational Site 36005

Debrecen, Hungary

Location

CTI Investigational Site 36006

Gyula, Hungary

Location

CTI Investigational Site 36003

Kaposvár, Hungary

Location

CTI Investigational Site 36004

Kecskemét, Hungary

Location

CTI Investigational Site 36001

Szeged, Hungary

Location

CTI Investigational Site 36008

Szolnok, Hungary

Location

CTI Investigational Site 36007

Szombathely, Hungary

Location

CTI Investigational Site 39003

Bologna, Italy

Location

CTI Investigational Site 39001

Florence, Italy

Location

CTI Investigational Site 39005

Milan, Italy

Location

CTI Investigational Site 39004

Monza, Italy

Location

CTI Investigational Site 39002

Padua, Italy

Location

CTI Investigational Site 39008

Reggio Emilia, Italy

Location

CTI Investigational Site 39006

Rimini, Italy

Location

CTI Investigational Site 31001

Amsterdam, Netherlands

Location

CTI Investigational Site 31002

Maastricht, Netherlands

Location

CTI Investigational Site 31003

Rotterdam, Netherlands

Location

CTI Investigational Site 31004

Utrecht, Netherlands

Location

CTI Investigational Site 64001

Christchurch, New Zealand

Location

CTI Investigational Site 64004

Dunedin, New Zealand

Location

CTI Investigational Site 64002

Hamilton, New Zealand

Location

CTI Investigational Site 64003

Takapuna, New Zealand

Location

CTI Investigational Site 70011

Izhevsk, Russia

Location

CTI Investigational Site 70008

Moscow, Russia

Location

CTI Investigational Site 70009

Moscow, Russia

Location

CTI Investigational Site 70002

Petrozavodsk, Russia

Location

CTI Investigational Site 70001

Saint Petersburg, Russia

Location

CTI Investigational Site 70004

Saint Petersburg, Russia

Location

CTI Investigational Site 70010

Saint Petersburg, Russia

Location

CTI Investigational Site 70005

Samara, Russia

Location

CTI Investigational Site 70006

Sochi, Russia

Location

CTI Investigational Site 70007

Volgograd, Russia

Location

CTI Investigational Site 44004

Birmingham, United Kingdom

Location

CTI Investigational Site 44008

Bournemouth, United Kingdom

Location

CTI Investigational Site 44002

Cambridge, United Kingdom

Location

CTI Investigational Site 44003

Cardiff, United Kingdom

Location

CTI Investigational Site 44001

London, United Kingdom

Location

CTI Investigational Site 44007

London, United Kingdom

Location

CTI Investigational Site 44006

Manchester, United Kingdom

Location

CTI Investigational Site 44005

Oxford, United Kingdom

Location

Related Publications (2)

• 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.

  PMID: 28336242 BACKGROUND

• 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.

  PMID: 33275766 DERIVED

MeSH Terms

Conditions

Primary Myelofibrosis; Polycythemia Vera; Thrombocythemia, Essential; Bone Marrow Diseases; Hematologic Diseases; Splenomegaly; Anemia; Myeloproliferative Disorders; Thrombocytopenia

Interventions

11-(2-pyrrolidin-1-ylethoxy)-14,19-dioxa-5,7,26-triazatetracyclo(19.3.1.1(2,6).1(8,12))heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene

Condition Hierarchy (Ancestors)

Hemic and Lymphatic Diseases; Bone Marrow Neoplasms; Hematologic Neoplasms; Neoplasms by Site; Neoplasms; Blood Coagulation Disorders; Thrombocytosis; Blood Platelet Disorders; Hemorrhagic Disorders; Hypertrophy; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms; Cytopenia

Results Point of Contact

• Title: Beth Ziemba
• Organization: CTI BioPharma Corp.

bziemba@ctibiopharma.com

206-272-4347

Study Officials

• Beth Ziemba

  VP, Pharmacovigilance, Clinical Operations, QA

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 18, 2013
• First Posted: January 23, 2013
• Study Start: January 1, 2013
• Primary Completion: January 1, 2015
• Study Completion: April 1, 2016
• Last Updated: September 29, 2020
• Results First Posted: September 29, 2020

Record last verified: 2020-09

Locations

NZ (4); GB (8); DE (9); RU (10); AU (6); FR (12); CZ (4); US (4); IT (7); NL (4); HU (8); BE (5)