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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Pacritinib | Experimental | Pacritinib 400 mg QD |
|
| 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) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Pacritinib | Drug |
| ||
| Best Available Therapy |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Beth Ziemba | VP, Pharmacovigilance, Clinical Operations, QA | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| CTI Investigational Site 10002 | Scottsdale | Arizona | 85259 | United States | ||
| CTI Investigational Site 10004 |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 28336242 | Background | 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. | |
| 33275766 |
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| ID | Title | Description |
|---|---|---|
| FG000 | Pacritinib | Pacritinib 400 mg QD |
| FG001 | Best Available Therapy | BAT includes any physician-selected treatment for primary or secondary myelofibrosis with the exclusion of JAK inhibitors (inhibitors of Janus kinases). |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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|
| Baseline to Week 24 |
| Omaha |
| Nebraska |
| 68198 |
| United States |
| CTI Investigational Site 10001 | Morristown | New Jersey | 07962 | United States |
| CTI Investigational Site 10003 | Greenville | South Carolina | 29601 | 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 | Padova | 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 |
| Derived |
| 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. |
| COMPLETED |
|
| NOT COMPLETED |
|
Represents Intent To Treat population (number of randomized patients)
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| ID | Title | Description |
|---|---|---|
| BG000 | Pacritinib | Pacritinib 400 mg QD |
| BG001 | Best Available Therapy | BAT includes any physician-selected treatment for primary or secondary myelofibrosis with the exclusion of JAK inhibitors (inhibitors of Janus kinases). |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | Number | participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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) | Posted | Count of Participants | Participants | Baseline to Week 24 |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | The original version (MPN-SAF TSS) was administered to the first 179 subjects enrolled in the study. The subsequent 148 enrolled subjects were administered the MPN-SAF TSS 2.0 and comprise the ITT population for this endpoint. | Posted | Count of Participants | Participants | Baseline to Week 24 |
|
|
Time of signing informed consent through 30 days after the last study treatment.
The data display threshold for SAEs is set to 1% or more, that of AEs is set to 5% or more.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Pacritinib | Pacritinib 400 mg QD | 27 | 220 | 117 | 220 | 207 | 220 |
| EG001 | Best Available Therapy | BAT includes any physician-selected treatment for primary or secondary myelofibrosis with the exclusion of JAK inhibitors (inhibitors of Janus kinases). | 3 | 106 | 33 | 106 | 81 | 106 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Basal cell Carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (16.0) | Systematic Assessment |
| |
| Squarmous cell Carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (16.0) | Systematic Assessment |
| |
| Acute myeloid leukaemia | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (16.0) | Systematic Assessment |
| |
| Squamous cell carcinoma of skin | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (16.0) | Systematic Assessment |
| |
| Cardiac failure congestive | Cardiac disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Anemia | Blood and lymphatic system disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Febrile Neutropenia | Blood and lymphatic system disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Disease Progression | General disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Multi-organ failure | General disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Abdominal Pain | Gastrointestinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Renal Failure Acute | Renal and urinary disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
| |
| Lobar pneumonia | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
| |
| Lower Respiratory Tract Infection | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
| |
| Sepis | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
| |
| Cardiac Failure | Cardiac disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Clonal evolution | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (16.0) | Systematic Assessment |
| |
| Endometrial cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (16.0) | Systematic Assessment |
| |
| Epstein-Barr virus associated lymphoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (16.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hypertension | Vascular disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Asthernia | General disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (16.0) | Systematic Assessment |
| |
| Electrocardiogram QT prolonged | Investigations | MedDRA (16.0) | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Oropharyngeal | Respiratory, thoracic and mediastinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Rush | Skin and subcutaneous tissue disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Arthralgia | Skin and subcutaneous tissue disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Pain in extremity | Skin and subcutaneous tissue disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (16.0) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (16.0) | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Beth Ziemba | CTI BioPharma Corp. | 206-272-4347 | bziemba@ctibiopharma.com |
| ID | Term |
|---|---|
| D055728 | Primary Myelofibrosis |
| D011087 | Polycythemia Vera |
| D013920 | Thrombocythemia, Essential |
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D013163 | Splenomegaly |
| D000740 | Anemia |
| D009196 | Myeloproliferative Disorders |
| D013921 | Thrombocytopenia |
| ID | Term |
|---|---|
| D006425 | Hemic and Lymphatic Diseases |
| D019046 | Bone Marrow Neoplasms |
| D019337 | Hematologic Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D001778 | Blood Coagulation Disorders |
| D013922 | Thrombocytosis |
| D001791 | Blood Platelet Disorders |
| D006474 | Hemorrhagic Disorders |
| D006984 | Hypertrophy |
| D020763 | Pathological Conditions, Anatomical |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D000095542 | Cytopenia |
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| ID | Term |
|---|---|
| C561234 | 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 |
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| >=65 years |
|
| Male |
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| Netherlands |
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| Belgium |
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| Hungary |
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| Czechia |
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| United States |
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| Italy |
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| United Kingdom |
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| Australia |
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| France |
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| Germany |
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| Russia |
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