Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   44064   clinical trials with a EudraCT protocol, of which   7320   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2012-004239-21
    Sponsor's Protocol Code Number:PAC325
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-11-30
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedGermany - BfArM
    A.2EudraCT number2012-004239-21
    A.3Full title of the trial
    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
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study comparing current standard therapies with pacritinib taken by mouth for the treatment of myelofibrosis (either diagnosed alone or after polycythemia vera or essential thrombocytopenia)
    A.3.2Name or abbreviated title of the trial where available
    PERSIST-1
    A.4.1Sponsor's protocol code numberPAC325
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCTI BioPharma Corp.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportCTI BioPharma Corp.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCTI Life Sciences Ltd.
    B.5.2Functional name of contact pointVice President & EU General Manager
    B.5.3 Address:
    B.5.3.1Street Address6-9 The Square, Stockley Park
    B.5.3.2Town/ cityUxbridge
    B.5.3.3Post codeUB11 1FW
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+44149 459 6722
    B.5.5Fax number+44208 610 6060
    B.5.6E-maildpatel@cti-lifesciences.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/10/768
    D.3 Description of the IMP
    D.3.1Product namepacritinib
    D.3.2Product code SB1518
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNpacritinib
    D.3.9.1CAS number 937272-79-2
    D.3.9.2Current sponsor codepacritinib
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis
    E.1.1.1Medical condition in easily understood language
    myelofibrosis
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level PT
    E.1.2Classification code 10028537
    E.1.2Term Myelofibrosis
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To compare the efficacy of pacritinib with that of best available therapy (BAT) in patients with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thromocythemia myelofibrosis (PET-MF). The efficacy measure for this analysis is the proportion 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) scan.
    E.2.2Secondary objectives of the trial
    The key secondary objective is the proportion of patients with ≥ 50% reduction in total symptom score from baseline to Week 24 on the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF TSS 2.0).

    Other secondary objectives are to compare pacritinib with BAT with respect to:
    - Proportion of patients with baseline platelet count < 100,000/μL achieving ≥ 35% reduction in spleen volume from baseline to Week 24 as measured by MRI or CT scan
    - Proportion of patients with baseline platelet count < 100,000/μL achieving ≥ 50% reduction in TSS from baseline to Week 24
    - Proportion of patients with baseline platelet count < 50,000/μL achieving ≥ 35% reduction in spleen volume from baseline to Week 24 as measured by MRI or CT scan
    - Proportion of patients with baseline platelet count < 50,000/μL achieving ≥ 50% reduction in TSS from baseline to Week 24
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1). Intermediate -1 or -2 or high-risk (Passamonti et al 2010) PMF, PPV-MF, or PET-MF (Tefferi and Vardiman 2008; Barosi et al 2008)
    2). Palpable splenomegaly ≥ 5 cm below lower costal margin (LCM) in midclavicular line by physical examination
    3). Total Symptom Score (TSS) ≥ 13 on the MPN-SAF TSS 2.0, not including the inactivity question
    4). Eligible to receive a medically appropriate BAT therapy, taking into account all contraindications and precautions of available BAT therapy options
    5). In the judgement of the investigator, patient is not a candidate for treatment with ruxolitinib due to medical reasons
    6). Age ≥ 18 years old
    7). Eastern Cooperative Oncology Group (ECOG) performance status 0-3
    8). Peripheral blast count < 10%
    9). Absolute neutrophil count > 500/μL
    10). Patients who are platelet or red blood cell transfusion-dependent are eligible
    11). Adequate liver and renal function, defined by liver transaminases (AST/SGOT and ALT/SGPT) ≤ 3 × ULN (AST/ALT ≤ 5 × ULN if transaminase elevation is related to MF), total bilirubin ≤ 1.5 x ULN (≤ 3 x ULN if due to Gilbert disease or low-grade hemolysis), and creatinine ≤ 2.5 mg/dL
    12). At least 6 months from prior splenic irradiation
    13). At least 12 months from prior 32P therapy
    14). At least 1 week since prior treatment (most recent dose) with a potent CYP3A4 inhibitor
    15). At least 4 weeks since any experimental treatment for PMF, PPV-MF, or PET-MF
    16). At least 2 weeks since any treatment for PMF, PPV-MF, or PET-MF
    17). If fertile, willing to use effective birth control methods during the study. Women of childbearing potential must use highly effective methods (defined as those resulting in a failure rate of <1% per year when used consistently and correctly) for the duration of study treatment and for 12 months after last dose of study drug. The contraceptive methods that are considered highly effective are intrauterine devices and hormonal contraceptives (contraceptive pills, implants, transdermal patches, hormonal vaginal devices, or injections with prolonged release). Males must use a condom for the duration of the study and for 90 days after the last dose of study treatment. When abstinence is used as a method of birth control, only true abstinence is acceptable, when this is in line with the preferred and usual lifestyle of the patient. Periodic abstinence (calendar, ovulation, symptothermal, and post-ovulation methods) and withdrawal are not acceptable methods of contraception.
    18). Willing to undergo and able to tolerate frequent MRI or CT assessments on study
    19). Able to understand and willing to complete symptom assessments using a patient reported outcomes instrument
    20). Able to understand and willing to sign the Informed Consent Form
    E.4Principal exclusion criteria
    1). Any gastrointestinal (GI) or metabolic condition that could interfere with absorption of oral medication
    2). Life expectancy < 6 months
    3). Prior treatment with a JAK2 inhibitor
    4). Completed allogeneic stem cell transplantation (ASCT) or eligible for and willing to complete ASCT
    5). History of splenectomy or planning to undergo splenectomy
    6). Uncontrolled intercurrent illness, including but not limited to ongoing active infection or psychiatric illness or social situation that the treating physician judges would limit compliance with study requirements
    7). Other malignancy within last 3 years other than curatively treated basal cell or squamous cell skin cancer, carcinoma in situ of the cervix, organ-confined or treated nonmetastatic prostate cancer with negative prostate-specific antigen, in situ breast carcinoma after complete surgical resection, or superficial transitional cell bladder carcinoma
    8). Inflammatory or chronic functional bowel disorder such as Crohn disease, inflammatory bowel disease, chronic diarrhea, or constipation
    9). Clinically symptomatic and uncontrolled cardiovascular disease
    10). History of any of the following, within 6 months prior to randomization: myocardial infarction, severe/unstable angina, or symptomatic congestive heart failure
    11). New York Heart Association Class II, III, or IV congestive heart failure
    12). Patients with National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) (version 4) grade 2 cardiac arrhythmias may be considered for inclusion, with the approval of the medical monitor, if the arrhythmias are stable, asymptomatic and unlikely to affect patient safety. Patients will be excluded if they have ongoing cardiac dysrhythmias of CTCAE grade ≥3, corrected QT interval (QTc) prolongation > 450 ms or other factors that increase the risk for QT prolongation (eg, heart failure, hypokalemia [defined as serum potassium < 3.0 mEq/L that is persistant and refractory to correction], or family history of long QT interval syndrome)
    13). Erythropoietic agent within 28 days prior to randomization
    14). Thrombopoietic agent within 14 days prior to randomization
    15). Known seropositivity for human immunodeficiency virus (HIV)
    16). Known active hepatitis A, B, or C virus infection
    17). Women who are pregnant or lactating
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint for measurement of efficacy is the proportion 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) scan.
    E.5.1.1Timepoint(s) of evaluation of this end point
    MRI or CT assessments: Screening and Wk 24 (exploratory evaluation only at weeks 12, 36, 48 and every 12 weeks)
    E.5.2Secondary end point(s)
    The secondary efficacy endpoint is the proportion of patients with ≥ 50% reduction in total symptom score (TSS) from baseline to Week 24 as measured by the Myleloproliferative Neoplasm Symptom Assessment Form (MPN-SAF TSS 2.0).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Patients will complete the MPN-SAF TSS 2.0 daily for 7 to 10 consecutive days prior to start of study treatment and then daily from Day 1 through Week 48, or until patient discontinues study treatment, whichever occurs first.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Exploratory
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over Yes
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Open study as Investigator and patients unblinded, but central data review and Sponsor is blinded.
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    best available therapy
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned9
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA57
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Belgium
    Czech Republic
    France
    Germany
    Hungary
    Italy
    Netherlands
    New Zealand
    Russian Federation
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    The end of trial will be on completion of survival follow-up. This will continue until 3 years past Week 24 or 3 years past early termination of study treatment, whichever occurs first.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 178
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 144
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state25
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 202
    F.4.2.2In the whole clinical trial 322
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Most patients will receive normal standard of care. However, for those subjects still receiving clinical benefit from pacritinib at the end of the trial and where it is not yet commercially available, the Sponsor will continue to provide access to pactritinib within the limits set by regulatory authorities (method of provision to be determined).
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2013-05-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-05-23
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-11-30
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA