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    The EU Clinical Trials Register currently displays   44339   clinical trials with a EudraCT protocol, of which   7369   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).

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    Summary
    EudraCT Number:2013-004000-19
    Sponsor's Protocol Code Number:PAC326
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-02-21
    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 ConcernedUK - MHRA
    A.2EudraCT number2013-004000-19
    A.3Full title of the trial
    A Randomized Controlled Phase 3 Study of Oral Pacritinib versus Best Available Therapy in Patients with Thrombocytopenia and 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) in patients with a low platelet count
    A.3.2Name or abbreviated title of the trial where available
    PERSIST-2
    A.4.1Sponsor's protocol code numberPAC326
    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 pointRegulatory Director
    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+44800088 5356
    B.5.5Fax number+44208100 5030
    B.5.6E-mailemengou@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 with Thrombocytopenia
    E.1.1.1Medical condition in easily understood language
    Myelofibrosis with a low platelet count
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.1
    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
    The primary objective is to compare the efficacy of two dose-schedule arm(s) of pacritinib (pooled once daily [QD] and twice-daily [BID] dosing arms) with that of best available therapy (BAT) in patients with thrombocytopenia and primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF).
    E.2.2Secondary objectives of the trial
    The secondary objectives are:
    1. To compare the efficacy of QD pacritinib with that of BAT, as assessed by the proportion of patients achieving a ≥ 35% reduction in spleen volume from baseline to Week 24 by MRI or CT and the proportion of patients achieving a ≥ 50% reduction in the TSS from baseline to Week 24 on the MPN-SAF TSS 2.0.
    2. To compare the efficacy of BID pacritinib with that of BAT, as assessed by the proportion of patients achieving a ≥ 35% reduction in spleen volume from baseline to Week 24 by MRI or CT and the proportion of patients achieving a ≥ 50% reduction in the TSS from baseline to Week 24 on the MPN-SAF TSS 2.0.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Intermediate-1 or -2 or high-risk PMF, PPV-MF, or PET-MF
    2. Thrombocytopenia (platelet count ≤100,000/µL) at any time after signing informed consent
    3. Informed consent may be signed up to 35 days prior to randomization
    4. Palpable splenomegaly ≥5 cm below the LCM in midclavicular line by physical examination
    5. Total Symptom Score (TSS) ≥13 on the MPN-SAF-TSS 2.0, not including the inactivity question
    6. Age ≥18 years old
    7. ECOG performance status 0 to 3
    8. Peripheral blast count <10%
    9. Absolute neutrophil count (ANC) >500/μL
    10. Patients who are platelet or RBC 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), direct bilirubin ≤4 x ULN, 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 cytochrome P450 3A4 (CYP3A4) inhibitor
    15. At least 2 weeks since receiving any treatment for PMF, PPV-MF, or PET-MF
    16. If fertile, males and females must agree to use effective birth control methods during the study.
    17. Willing to undergo and able to tolerate frequent MRI or CT assessments during the study
    18. Able to understand and willing to complete symptom assessments using a patient-reported outcomes instrument
    19. 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 less than 6 months
    3. Prior treatment with more than 2 JAK2 inhibitors or pacritinib
    4. There is no maximum cumulative prior JAK2 inhibitor treatment (approved or investigational)
    5. Completed allogeneic stem cell transplantation (ASCT) or are eligible for and willing to complete ASCT
    6. History of splenectomy or planning to undergo splenectomy
    7. Uncontrolled intercurrent illness, including but not limited to ongoing active infection, psychiatric illness, or social situation that, in the judgment of the treating physician, would limit compliance with study requirements
    8. Active bleeding requiring hospitalization during the screening period
    9. Other malignancy within the 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
    10. Inflammatory or chronic functional bowel disorder, such as Crohn’s disease, inflammatory bowel disease, chronic diarrhea, or constipation
    11. Clinically symptomatic and uncontrolled cardiovascular disease
    12. History of any of the following within 6 months prior to randomization: myocardial infarction, severe/unstable angina, or symptomatic congestive heart failure
    13. New York Heart Association Class III or IV congestive heart failure
    14. Patients with National Cancer Institute (NCI) Common Terminology for Adverse Events (CTCAE) 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 persistent and refractory to correction], or family history of long QT interval syndrome).
    15. Erythropoietic agent within 28 days prior to randomization
    16. Thrombopoietic agent within 14 days prior to randomization
    17. Known seropositivity for human immunodeficiency virus (HIV)
    18. Known active hepatitis A, B, or C virus infection
    19. Women who are pregnant or lactating
    E.5 End points
    E.5.1Primary end point(s)
    1. 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;
    2. the proportion of patients achieving a ≥ 50% reduction in total symptom score (TSS) from baseline to Week 24 as measured by the Myeloproliferative Neoplasm Symptom Assessment Form 2.0 (MPN-SAF TSS 2.0).
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. MRI or CT assessments at: Screening and Wk 24 (additional exploratory evaluation at weeks 12, 36, 48 and every 12 weeks).
    2. TSS: assessed daily through week 48 of the study or until the patient discontinues study treatment, whichever occurs first.
    E.5.2Secondary end point(s)
    Not Applicable
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not Applicable
    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 No
    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 are 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 trial3
    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 concerned11
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA69
    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
    Canada
    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 the first of the following timepoints:
    - 3 years past Week 24
    - 3 years past last treatment with initially assigned study drug
    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 months6
    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: 170
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 130
    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 state35
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 150
    F.4.2.2In the whole clinical trial 300
    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 pacritinib 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 Decision2014-04-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-05-30
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-04-26
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