search
Back to results

Pacritinib Versus Best Available Therapy to Treat Myelofibrosis

Primary Purpose

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

Status
Terminated
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Pacritinib
Best Available Therapy
Sponsored by
CTI BioPharma
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Primary Myelofibrosis focused on measuring 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

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • CTI Investigational Site 10002
  • CTI Investigational Site 10004
  • CTI Investigational Site 10001
  • CTI Investigational Site 10003
  • CTI Investigational Site 61006
  • CTI Investigational Site 61001
  • CTI Investigational Site 61005
  • CTI Investigational Site 61003
  • CTI Investigational Site 61004
  • CTI Investigational Site 61002
  • CTI Investigational Site 32002
  • CTI Investigational Site 32003
  • CTI Investigational Site 32001
  • CTI Investigational Site 32005
  • CTI Investigational Site 32004
  • CTI Investigational Site 42003
  • CTI Investigational Site 42001
  • CTI Investigational Site 42002
  • CTI Investigational Site 42004
  • CTI Investigational Site 33005
  • CTI Investigational Site 33006
  • CTI Investigational Site 33011
  • CTI Investigational Site 33012
  • CTI Investigational Site 33007
  • CTI Investigational Site 33001
  • CTI Investigational Site 33004
  • CTI Investigational Site 33008
  • CTI Investigational Site 33009
  • CTI Investigational Site 33010
  • CTI Investigational Site 33003
  • CTI Investigational Site 33002
  • CTI Investigational Site 49006
  • CTI Investigational Site 49007
  • CTI Investigational Site 49003
  • CTI Investigational Site 49008
  • CTI Investigational Site 49002
  • CTI Investigational Site 49001
  • CTI Investigational Site 49005
  • CTI Investigational Site 49004
  • CTI Investigational Site 49009
  • CTI Investigational Site 36002
  • CTI Investigational Site 36005
  • CTI Investigational Site 36006
  • CTI Investigational Site 36003
  • CTI Investigational Site 36004
  • CTI Investigational Site 36001
  • CTI Investigational Site 36008
  • CTI Investigational Site 36007
  • CTI Investigational Site 39003
  • CTI Investigational Site 39001
  • CTI Investigational Site 39005
  • CTI Investigational Site 39004
  • CTI Investigational Site 39002
  • CTI Investigational Site 39008
  • CTI Investigational Site 39006
  • CTI Investigational Site 31001
  • CTI Investigational Site 31002
  • CTI Investigational Site 31003
  • CTI Investigational Site 31004
  • CTI Investigational Site 64001
  • CTI Investigational Site 64004
  • CTI Investigational Site 64002
  • CTI Investigational Site 64003
  • CTI Investigational Site 70011
  • CTI Investigational Site 70008
  • CTI Investigational Site 70009
  • CTI Investigational Site 70002
  • CTI Investigational Site 70010
  • CTI Investigational Site 70005
  • CTI Investigational Site 70006
  • CTI Investigational Site 70001
  • CTI Investigational Site 70004
  • CTI Investigational Site 70007
  • CTI Investigational Site 44004
  • CTI Investigational Site 44008
  • CTI Investigational Site 44002
  • CTI Investigational Site 44003
  • CTI Investigational Site 44001
  • CTI Investigational Site 44007
  • CTI Investigational Site 44006
  • CTI Investigational Site 44005

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

Pacritinib

Best Available Therapy

Arm Description

Pacritinib 400 mg QD

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

Outcomes

Primary Outcome Measures

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)

Secondary Outcome Measures

Total Symptom Score (TSS) Reduction
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.

Full Information

First Posted
January 18, 2013
Last Updated
September 25, 2020
Sponsor
CTI BioPharma
search

1. Study Identification

Unique Protocol Identification Number
NCT01773187
Brief Title
Pacritinib Versus Best Available Therapy to Treat Myelofibrosis
Official Title
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
Study Type
Interventional

2. Study Status

Record Verification Date
September 2020
Overall Recruitment Status
Terminated
Study Start Date
January 2013 (Actual)
Primary Completion Date
January 2015 (Actual)
Study Completion Date
April 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
CTI BioPharma

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
327 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Pacritinib
Arm Type
Experimental
Arm Description
Pacritinib 400 mg QD
Arm Title
Best Available Therapy
Arm Type
Active Comparator
Arm Description
BAT includes any physician-selected treatment for primary or secondary myelofibrosis with the exclusion of JAK inhibitors (inhibitors of Janus kinases)
Intervention Type
Drug
Intervention Name(s)
Pacritinib
Intervention Type
Drug
Intervention Name(s)
Best Available Therapy
Primary Outcome Measure Information:
Title
Spleen Volume Reduction
Description
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)
Time Frame
Baseline to Week 24
Secondary Outcome Measure Information:
Title
Total Symptom Score (TSS) Reduction
Description
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.
Time Frame
Baseline to Week 24

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Beth Ziemba
Organizational Affiliation
VP, Pharmacovigilance, Clinical Operations, QA
Official's Role
Study Director
Facility Information:
Facility Name
CTI Investigational Site 10002
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85259
Country
United States
Facility Name
CTI Investigational Site 10004
City
Omaha
State/Province
Nebraska
ZIP/Postal Code
68198
Country
United States
Facility Name
CTI Investigational Site 10001
City
Morristown
State/Province
New Jersey
ZIP/Postal Code
07962
Country
United States
Facility Name
CTI Investigational Site 10003
City
Greenville
State/Province
South Carolina
ZIP/Postal Code
29601
Country
United States
Facility Name
CTI Investigational Site 61006
City
Box Hill
Country
Australia
Facility Name
CTI Investigational Site 61001
City
Coffs Harbour
Country
Australia
Facility Name
CTI Investigational Site 61005
City
Geelong
Country
Australia
Facility Name
CTI Investigational Site 61003
City
Gosford
Country
Australia
Facility Name
CTI Investigational Site 61004
City
Hobart
Country
Australia
Facility Name
CTI Investigational Site 61002
City
Milton
Country
Australia
Facility Name
CTI Investigational Site 32002
City
Antwerp
Country
Belgium
Facility Name
CTI Investigational Site 32003
City
Antwerp
Country
Belgium
Facility Name
CTI Investigational Site 32001
City
Brugge
Country
Belgium
Facility Name
CTI Investigational Site 32005
City
Bruxelles
Country
Belgium
Facility Name
CTI Investigational Site 32004
City
La Louviere
Country
Belgium
Facility Name
CTI Investigational Site 42003
City
Brno
Country
Czechia
Facility Name
CTI Investigational Site 42001
City
Olomouc
Country
Czechia
Facility Name
CTI Investigational Site 42002
City
Plzen
Country
Czechia
Facility Name
CTI Investigational Site 42004
City
Prague
Country
Czechia
Facility Name
CTI Investigational Site 33005
City
Amiens
Country
France
Facility Name
CTI Investigational Site 33006
City
Caen
Country
France
Facility Name
CTI Investigational Site 33011
City
Grenoble
Country
France
Facility Name
CTI Investigational Site 33012
City
Lens
Country
France
Facility Name
CTI Investigational Site 33007
City
Lille
Country
France
Facility Name
CTI Investigational Site 33001
City
Nimes Cedex
Country
France
Facility Name
CTI Investigational Site 33004
City
Paris
Country
France
Facility Name
CTI Investigational Site 33008
City
Paris
Country
France
Facility Name
CTI Investigational Site 33009
City
Pessac
Country
France
Facility Name
CTI Investigational Site 33010
City
Pierre Benite
Country
France
Facility Name
CTI Investigational Site 33003
City
Strasbourg
Country
France
Facility Name
CTI Investigational Site 33002
City
Toulouse
Country
France
Facility Name
CTI Investigational Site 49006
City
Berlin
Country
Germany
Facility Name
CTI Investigational Site 49007
City
Berlin
Country
Germany
Facility Name
CTI Investigational Site 49003
City
Dresden
Country
Germany
Facility Name
CTI Investigational Site 49008
City
Essen
Country
Germany
Facility Name
CTI Investigational Site 49002
City
Freiburg
Country
Germany
Facility Name
CTI Investigational Site 49001
City
Koln
Country
Germany
Facility Name
CTI Investigational Site 49005
City
Mainz
Country
Germany
Facility Name
CTI Investigational Site 49004
City
Munchen
Country
Germany
Facility Name
CTI Investigational Site 49009
City
Munster
Country
Germany
Facility Name
CTI Investigational Site 36002
City
Budapest
Country
Hungary
Facility Name
CTI Investigational Site 36005
City
Debrecen
Country
Hungary
Facility Name
CTI Investigational Site 36006
City
Gyula
Country
Hungary
Facility Name
CTI Investigational Site 36003
City
Kaposvar
Country
Hungary
Facility Name
CTI Investigational Site 36004
City
Kecskemet
Country
Hungary
Facility Name
CTI Investigational Site 36001
City
Szeged
Country
Hungary
Facility Name
CTI Investigational Site 36008
City
Szolnok
Country
Hungary
Facility Name
CTI Investigational Site 36007
City
Szombathely
Country
Hungary
Facility Name
CTI Investigational Site 39003
City
Bologna
Country
Italy
Facility Name
CTI Investigational Site 39001
City
Firenze
Country
Italy
Facility Name
CTI Investigational Site 39005
City
Milano
Country
Italy
Facility Name
CTI Investigational Site 39004
City
Monza
Country
Italy
Facility Name
CTI Investigational Site 39002
City
Padova
Country
Italy
Facility Name
CTI Investigational Site 39008
City
Reggio Emilia
Country
Italy
Facility Name
CTI Investigational Site 39006
City
Rimini
Country
Italy
Facility Name
CTI Investigational Site 31001
City
Amsterdam
Country
Netherlands
Facility Name
CTI Investigational Site 31002
City
Maastricht
Country
Netherlands
Facility Name
CTI Investigational Site 31003
City
Rotterdam
Country
Netherlands
Facility Name
CTI Investigational Site 31004
City
Utrecht
Country
Netherlands
Facility Name
CTI Investigational Site 64001
City
Christchurch
Country
New Zealand
Facility Name
CTI Investigational Site 64004
City
Dunedin
Country
New Zealand
Facility Name
CTI Investigational Site 64002
City
Hamilton
Country
New Zealand
Facility Name
CTI Investigational Site 64003
City
Takapuna
Country
New Zealand
Facility Name
CTI Investigational Site 70011
City
Izhevsk
Country
Russian Federation
Facility Name
CTI Investigational Site 70008
City
Moscow
Country
Russian Federation
Facility Name
CTI Investigational Site 70009
City
Moscow
Country
Russian Federation
Facility Name
CTI Investigational Site 70002
City
Petrozavodsk
Country
Russian Federation
Facility Name
CTI Investigational Site 70010
City
Saint Petersburg
Country
Russian Federation
Facility Name
CTI Investigational Site 70005
City
Samara
Country
Russian Federation
Facility Name
CTI Investigational Site 70006
City
Sochi
Country
Russian Federation
Facility Name
CTI Investigational Site 70001
City
St. Petersburg
Country
Russian Federation
Facility Name
CTI Investigational Site 70004
City
St. Petersburg
Country
Russian Federation
Facility Name
CTI Investigational Site 70007
City
Volgograd
Country
Russian Federation
Facility Name
CTI Investigational Site 44004
City
Birmingham
Country
United Kingdom
Facility Name
CTI Investigational Site 44008
City
Bournemouth
Country
United Kingdom
Facility Name
CTI Investigational Site 44002
City
Cambridge
Country
United Kingdom
Facility Name
CTI Investigational Site 44003
City
Cardiff
Country
United Kingdom
Facility Name
CTI Investigational Site 44001
City
London
Country
United Kingdom
Facility Name
CTI Investigational Site 44007
City
London
Country
United Kingdom
Facility Name
CTI Investigational Site 44006
City
Manchester
Country
United Kingdom
Facility Name
CTI Investigational Site 44005
City
Oxford
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
28336242
Citation
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.
Results Reference
background
PubMed Identifier
33275766
Citation
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.
Results Reference
derived

Learn more about this trial

Pacritinib Versus Best Available Therapy to Treat Myelofibrosis

We'll reach out to this number within 24 hrs