Primary MyelofibrosisPhase 3NCT01773187
Pacritinib Versus Best Available Therapy to Treat Myelofibrosis
A Phase 3 study testing Pacritinib and Best Available Therapy in people with Primary Myelofibrosis and Post-polycythemia Vera Myelofibrosis, run by CTI BioPharma at 81 sites in 12 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis
- What is being tested?
- Pacritinib and Best Available Therapy
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 327 participants
- Where
- 81 sitesin 12 countries
- Study dates
- Jan 2013to Apr 2016
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
81 sites in 12 countries.
81 sites in 12 countries
France12 sites
CTI Investigational Site 33005
Amiens
CTI Investigational Site 33006
Caen
CTI Investigational Site 33011
Grenoble
CTI Investigational Site 33012
Lens
CTI Investigational Site 33007
Lille
CTI Investigational Site 33001
Nîmes
CTI Investigational Site 33004
Paris
CTI Investigational Site 33008
Paris
CTI Investigational Site 33009
Pessac
CTI Investigational Site 33010
Pierre-Bénite
CTI Investigational Site 33003
Strasbourg
CTI Investigational Site 33002
Toulouse
Russia10 sites
CTI Investigational Site 70011
Izhevsk
CTI Investigational Site 70008
Moscow
CTI Investigational Site 70009
Moscow
CTI Investigational Site 70002
Petrozavodsk
CTI Investigational Site 70001
Saint Petersburg
CTI Investigational Site 70004
Saint Petersburg
CTI Investigational Site 70010
Saint Petersburg
CTI Investigational Site 70005
Samara
CTI Investigational Site 70006
Sochi
CTI Investigational Site 70007
Volgograd
Germany9 sites
CTI Investigational Site 49006
Berlin
CTI Investigational Site 49007
Berlin
CTI Investigational Site 49001
Cologne
CTI Investigational Site 49003
Dresden
CTI Investigational Site 49008
Essen
CTI Investigational Site 49002
Freiburg im Breisgau
CTI Investigational Site 49005
Mainz
CTI Investigational Site 49004
München
CTI Investigational Site 49009
Münster
Hungary8 sites
CTI Investigational Site 36002
Budapest
CTI Investigational Site 36005
Debrecen
CTI Investigational Site 36006
Gyula
CTI Investigational Site 36003
Kaposvár
CTI Investigational Site 36004
Kecskemét
CTI Investigational Site 36001
Szeged
CTI Investigational Site 36008
Szolnok
CTI Investigational Site 36007
Szombathely
United Kingdom8 sites
CTI Investigational Site 44004
Birmingham
CTI Investigational Site 44008
Bournemouth
CTI Investigational Site 44002
Cambridge
CTI Investigational Site 44003
Cardiff
CTI Investigational Site 44001
London
CTI Investigational Site 44007
London
CTI Investigational Site 44006
Manchester
CTI Investigational Site 44005
Oxford
Italy7 sites
CTI Investigational Site 39003
Bologna
CTI Investigational Site 39001
Florence
CTI Investigational Site 39005
Milan
CTI Investigational Site 39004
Monza
CTI Investigational Site 39002
Padova
CTI Investigational Site 39008
Reggio Emilia
CTI Investigational Site 39006
Rimini
Australia6 sites
CTI Investigational Site 61006
Box Hill
CTI Investigational Site 61001
Coffs Harbour
CTI Investigational Site 61005
Geelong
CTI Investigational Site 61003
Gosford
CTI Investigational Site 61004
Hobart
CTI Investigational Site 61002
Milton
Belgium5 sites
CTI Investigational Site 32002
Antwerp
CTI Investigational Site 32003
Antwerp
CTI Investigational Site 32001
Bruges
CTI Investigational Site 32005
Brussels
CTI Investigational Site 32004
La Louvière
United States4 sites
CTI Investigational Site 10003
Greenville, South Carolina, 29601
CTI Investigational Site 10001
Morristown, New Jersey, 07962
CTI Investigational Site 10004
Omaha, Nebraska, 68198
CTI Investigational Site 10002
Scottsdale, Arizona, 85259
Czechia4 sites
CTI Investigational Site 42003
Brno
CTI Investigational Site 42001
Olomouc
CTI Investigational Site 42002
Pilsen
CTI Investigational Site 42004
Prague
Netherlands4 sites
CTI Investigational Site 31001
Amsterdam
CTI Investigational Site 31002
Maastricht
CTI Investigational Site 31003
Rotterdam
CTI Investigational Site 31004
Utrecht
New Zealand4 sites
CTI Investigational Site 64001
Christchurch
CTI Investigational Site 64004
Dunedin
CTI Investigational Site 64002
Hamilton
CTI Investigational Site 64003
Takapuna
About this study
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
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.
Read the 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
- Being tested
- Pacritinib, Best Available Therapy
- 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
- Medical subject headings
- Primary Myelofibrosis, Polycythemia Vera, Thrombocythemia, Essential, Bone Marrow Diseases, Hematologic Diseases, Splenomegaly, Anemia, Myeloproliferative Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PacritinibDrug
Best Available TherapyDrug
| Study group | Role | What they receive |
|---|---|---|
| Pacritinib | Experimental | Pacritinib 400 mg QD Receives: 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) Receives: Drug: Best Available Therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: Baseline to Week 24
Secondary outcomes (1)
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.
Measured: Baseline to Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jan 2013 (estimated)
- Study startJan 2013
- Primary completionJan 2015
- Study completionApr 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Primary MyelofibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 327 participants (actual)
Sponsor and investigators
- Lead sponsor
- CTI BioPharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Beth Ziemba, Study director, VP, Pharmacovigilance, Clinical Operations, QA
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01773187
- Sponsor's protocol ID
- PERSIST-1 (PAC325)
- Status last verified
- Sep 2020
- First submitted
- 18 Jan 2013
- Last updated
- 29 Sep 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
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.
PubMed 28336242Tremblay 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.
PubMed 33275766
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01773187
- Acronym / sponsor ID
- PERSIST-1 (PAC325)
- 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
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 327 participants (actual)
- Lead sponsor
- CTI BioPharmaIndustry (pharma, biotech, devices)
- Study start
- Jan 2013 (actual)
- Primary completion
- Jan 2015 (actual)
- Study completion
- Apr 2016 (actual)
- First posted
- 23 Jan 2013 (estimated)
- Results
- Posted 29 Sep 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Pacritinib | Experimental | Drug: PacritinibPacritinib 400 mg QD |
| Best Available Therapy | Active comparator | Drug: Best Available TherapyBAT includes any physician-selected treatment for primary or secondary myelofibrosis with the exclusion of JAK inhibitors (inhibitors of Janus kinases) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Pacritinib | Drug | Pacritinib |
| Best Available Therapy | Drug | Best Available Therapy |
Primary outcome
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)
1 secondary outcome
Total Symptom Score (TSS) Reduction
Time frame: Baseline to Week 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Sep 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 28336242Mesa 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.
- Derived from this study: PubMed 33275766Tremblay 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Beth Ziemba study director
VP, Pharmacovigilance, Clinical Operations, QA
81 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Pacritinib and Best Available Therapy in people with Primary Myelofibrosis and Post-polycythemia Vera Myelofibrosis, run by CTI BioPharma at 81 sites in 12 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 29 Sep 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 81 sites in France, Russia, Germany, Hungary, United Kingdom and 7 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 29 Sep 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Spleen Volume Reduction (time frame: Baseline to Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01773187, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Sep 2020; AzTrials last refreshed its copy on 7 Nov 2026.