Pacritinib Versus Best Available Therapy to Treat Myelofibrosis

September 25, 2020 updated by: CTI BioPharma

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

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

327

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Box Hill, Australia
        • CTI Investigational Site 61006
      • Coffs Harbour, Australia
        • CTI Investigational Site 61001
      • Geelong, Australia
        • CTI Investigational Site 61005
      • Gosford, Australia
        • CTI Investigational Site 61003
      • Hobart, Australia
        • CTI Investigational Site 61004
      • Milton, Australia
        • CTI Investigational Site 61002
  • Belgium
      • Antwerp, Belgium
        • CTI Investigational Site 32002
      • Antwerp, Belgium
        • CTI Investigational Site 32003
      • Brugge, Belgium
        • CTI Investigational Site 32001
      • Bruxelles, Belgium
        • CTI Investigational Site 32005
      • La Louviere, Belgium
        • CTI Investigational Site 32004
  • Czechia
      • Brno, Czechia
        • CTI Investigational Site 42003
      • Olomouc, Czechia
        • CTI Investigational Site 42001
      • Plzen, Czechia
        • CTI Investigational Site 42002
      • Prague, Czechia
        • CTI Investigational Site 42004
  • France
      • Amiens, France
        • CTI Investigational Site 33005
      • Caen, France
        • CTI Investigational Site 33006
      • Grenoble, France
        • CTI Investigational Site 33011
      • Lens, France
        • CTI Investigational Site 33012
      • Lille, France
        • CTI Investigational Site 33007
      • Nimes Cedex, France
        • CTI Investigational Site 33001
      • Paris, France
        • CTI Investigational Site 33004
      • Paris, France
        • CTI Investigational Site 33008
      • Pessac, France
        • CTI Investigational Site 33009
      • Pierre Benite, France
        • CTI Investigational Site 33010
      • Strasbourg, France
        • CTI Investigational Site 33003
      • Toulouse, France
        • CTI Investigational Site 33002
  • Germany
      • Berlin, Germany
        • CTI Investigational Site 49006
      • Berlin, Germany
        • CTI Investigational Site 49007
      • Dresden, Germany
        • CTI Investigational Site 49003
      • Essen, Germany
        • CTI Investigational Site 49008
      • Freiburg, Germany
        • CTI Investigational Site 49002
      • Koln, Germany
        • CTI Investigational Site 49001
      • Mainz, Germany
        • CTI Investigational Site 49005
      • Munchen, Germany
        • CTI Investigational Site 49004
      • Munster, Germany
        • CTI Investigational Site 49009
  • Hungary
      • Budapest, Hungary
        • CTI Investigational Site 36002
      • Debrecen, Hungary
        • CTI Investigational Site 36005
      • Gyula, Hungary
        • CTI Investigational Site 36006
      • Kaposvar, Hungary
        • CTI Investigational Site 36003
      • Kecskemet, Hungary
        • CTI Investigational Site 36004
      • Szeged, Hungary
        • CTI Investigational Site 36001
      • Szolnok, Hungary
        • CTI Investigational Site 36008
      • Szombathely, Hungary
        • CTI Investigational Site 36007
  • Italy
      • Bologna, Italy
        • CTI Investigational Site 39003
      • Firenze, Italy
        • CTI Investigational Site 39001
      • Milano, Italy
        • CTI Investigational Site 39005
      • Monza, Italy
        • CTI Investigational Site 39004
      • Padova, Italy
        • CTI Investigational Site 39002
      • Reggio Emilia, Italy
        • CTI Investigational Site 39008
      • Rimini, Italy
        • CTI Investigational Site 39006
  • Netherlands
      • Amsterdam, Netherlands
        • CTI Investigational Site 31001
      • Maastricht, Netherlands
        • CTI Investigational Site 31002
      • Rotterdam, Netherlands
        • CTI Investigational Site 31003
      • Utrecht, Netherlands
        • CTI Investigational Site 31004
  • New Zealand
      • Christchurch, New Zealand
        • CTI Investigational Site 64001
      • Dunedin, New Zealand
        • CTI Investigational Site 64004
      • Hamilton, New Zealand
        • CTI Investigational Site 64002
      • Takapuna, New Zealand
        • CTI Investigational Site 64003
  • Russian Federation
      • Izhevsk, Russian Federation
        • CTI Investigational Site 70011
      • Moscow, Russian Federation
        • CTI Investigational Site 70008
      • Moscow, Russian Federation
        • CTI Investigational Site 70009
      • Petrozavodsk, Russian Federation
        • CTI Investigational Site 70002
      • Saint Petersburg, Russian Federation
        • CTI Investigational Site 70010
      • Samara, Russian Federation
        • CTI Investigational Site 70005
      • Sochi, Russian Federation
        • CTI Investigational Site 70006
      • St. Petersburg, Russian Federation
        • CTI Investigational Site 70001
      • St. Petersburg, Russian Federation
        • CTI Investigational Site 70004
      • Volgograd, Russian Federation
        • CTI Investigational Site 70007
  • United Kingdom
      • Birmingham, United Kingdom
        • CTI Investigational Site 44004
      • Bournemouth, United Kingdom
        • CTI Investigational Site 44008
      • Cambridge, United Kingdom
        • CTI Investigational Site 44002
      • Cardiff, United Kingdom
        • CTI Investigational Site 44003
      • London, United Kingdom
        • CTI Investigational Site 44001
      • London, United Kingdom
        • CTI Investigational Site 44007
      • Manchester, United Kingdom
        • CTI Investigational Site 44006
      • Oxford, United Kingdom
        • CTI Investigational Site 44005
  • United States
    • Arizona
      • Scottsdale, Arizona, United States, 85259
        • CTI Investigational Site 10002
    • Nebraska
      • Omaha, Nebraska, United States, 68198
        • CTI Investigational Site 10004
    • New Jersey
      • Morristown, New Jersey, United States, 07962
        • CTI Investigational Site 10001
    • South Carolina
      • Greenville, South Carolina, United States, 29601
        • CTI Investigational Site 10003

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Pacritinib
Pacritinib 400 mg QD
Drug: Pacritinib
Active Comparator: Best Available Therapy
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Spleen Volume Reduction
Time Frame: Baseline to Week 24
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Total Symptom Score (TSS) Reduction
Time Frame: Baseline to Week 24
Number of patients with >= 50% reduction in total 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.
Baseline to Week 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

CTI BioPharma

Investigators

  • Study Director: Beth Ziemba, VP, Pharmacovigilance, Clinical Operations, QA

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 1, 2013

Primary Completion (Actual)

January 1, 2015

Study Completion (Actual)

April 1, 2016

Study Registration Dates

First Submitted

January 18, 2013

First Submitted That Met QC Criteria

January 18, 2013

First Posted (Estimate)

January 23, 2013

Study Record Updates

Last Update Posted (Actual)

September 29, 2020

Last Update Submitted That Met QC Criteria

September 25, 2020

Last Verified

September 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PERSIST-1 (PAC325)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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