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    Summary
    EudraCT Number:2009-017095-24
    Sponsor's Protocol Code Number:AOP13007
    National Competent Authority:Lithuania - SMCA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2010-06-18
    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 ConcernedLithuania - SMCA
    A.2EudraCT number2009-017095-24
    A.3Full title of the trial
    A phase III, randomized, multicenter, subject- and sponsor-blinded, placebo controlled study to compare the efficacy and safety of “Anagrelide retard” versus placebo in “at risk” subjects with Essential Thrombocythaemia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical trial for comparison of "anagrelide retard" versus placebo in subjects with Essential Thrombocythaemia.
    A.3.2Name or abbreviated title of the trial where available
    ARETA
    A.4.1Sponsor's protocol code numberAOP13007
    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 SponsorAOP Orphan Pharmaceuticals AG
    B.1.3.4CountryAustria
    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 supportAOP Orphan Pharmaceuticals AG
    B.4.2CountryAustria
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAOP Orphan Pharmaceuticals AG
    B.5.2Functional name of contact pointMedical Director
    B.5.3 Address:
    B.5.3.1Street AddressWilhelminenstrasse 91/B4
    B.5.3.2Town/ cityVienna
    B.5.3.3Post code1160
    B.5.3.4CountryAustria
    B.5.4Telephone number431503 72 44 46
    B.5.5Fax number431503 72 44 41
    B.5.6E-mailoleh.zahriychuk@aoporphan.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameAnagrelide retard 2mg tablets
    D.3.2Product code not applicable
    D.3.4Pharmaceutical form Modified-release tablet
    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 INNAnagrelide hydrochloride monohydrate
    D.3.9.1CAS number 58579-51-4
    D.3.9.2Current sponsor codeNA
    D.3.9.3Other descriptive nameNA
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number1.9 to 2.1
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    male and female "at risk" patients with Essential Thrombocythaemia
    E.1.1.1Medical condition in easily understood language
    male and female "at risk" patients with Essential Thrombocythaemia
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level PT
    E.1.2Classification code 10015493
    E.1.2Term Essential thrombocythaemia
    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 determine whether "Anagrelide retard" compared to placebo will reduce ET-related complications in subjects with potential risk for ET-related Events
    E.2.2Secondary objectives of the trial
    to assess the effect of "Anagrelide retard" compared to placebo on the following:
    - reduction of platelet count
    - response rates
    - change to "high risk status"
    - cardiovascular safety
    - Quality of Life
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Willing and able to give written informed consent prior to any study specific procedures and able to comply with this protocol
    • Male or female patients aged between 18 and 60 years,
    • Confirmed diagnosis of ET according to WHO-criteria (2008) including assessment of JAK-2 status.
    • Presence of predisposing risk factors for ET related events confirmed by clinical or laboratory results:

    - Platelet count < 1.000 G/L
    additionally at least ONE of the following criteria has to be fulfilled:
    - Subjects aged between 40 and 60 years
    or
    - Subjects with ET and disease duration > 3 years (Diagnosis of ET has to be at least 3 years ago and confirmed at time of screening)
    or
    - Subjects with ONE of the following risk factors for thrombotic complications:
    a) JAK- 2 positivity, b) Protein C and/or Protein S deficiency, c) Antithrombin III deficiency, d) Factor V Leiden or Prothrombin mutation, e) Cardiovascular risk factors: Essential hypertension; Smoking (>5 cigarettes/d), Obesity (BMI>30), cholesterol (HDL/LDL ratio < 4); Hormone replacement therapy; hormonal contraception
    E.4Principal exclusion criteria
    • Diagnosis of any other myeloproliferative disorder
    • High-risk status (age > 60 years, platelet count ≥ 1.000 G/L, increase of platelet count > 300 G/L within 3 month, history of thrombotic/haemorhagic or ischemic complications).
    • Any known cause for a secondary thrombocytosis
    • Previous or current treatment of ET with cytoreductive therapy
    • Diagnosis of any malignancy, apart from ET, within the last 3 years
    • Known or suspected intolerance to the investigational products
    • Known or suspected congestive heart failure
    • WBC ≥ 15 G/L
    • Severe renal impairment (creatinine clearance <30 ml/min)
    • Severe liver impairment (ALT or AST >5 times normal)
    • Clinically significant abnormal laboratory values (excluding markers of essential thrombocythaemia) in investigator's opinion, including electrolytes disbalance
    • Poorly controlled diabetes mellitus
    • Infection with hepatitis B, hepatitis C or HIV
    • Subjects with a history of drug/alcohol abuse (within the previous 2 years)
    • Participation in another investigational study within 6 months prior to enrolment or for a longer duration if specified in local regulations
    • Women of childbearing potential with inadequate contraception
    • Pregnant or lactating women (pregnancy test to be assessed within 7 days prior to study treatment start)
    • Any significant psychiatric disorder that, in the opinion of the investigator, might prohibit the understanding and giving of informed consent or that might prevent the patient from completing the trial.
    E.5 End points
    E.5.1Primary end point(s)
    Time to the 1st clinically significant ET related event
    E.5.1.1Timepoint(s) of evaluation of this end point
    The main part of the study is planned as a 2-stage procedure according
    to Bauer and Köhne. After recruitment of 140 subjects an interim
    analysis with re-assessment of sample size is performed.
    E.5.2Secondary end point(s)
    Efficacy
    - reduction of platelet counts
    - occurrence of change to “high risk” status
    (i.e. platelets > 1.000 G/L or occurrence of an ET related event)
    - number of subjects achieving a complete response
    Safety
    - Adverse Events
    - cardiovascular safety (assessed by ECG, ECHO, NT-proBNP or
    BNP)
    Quality of Life: SF-36
    E.5.2.1Timepoint(s) of evaluation of this end point
    The main part of the study is planned as a 2-stage procedure according
    to Bauer and Köhne. After recruitment of 140 subjects an interim
    analysis with re-assessment of sample size is performed.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis Yes
    E.6.2Prophylaxis Yes
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic Yes
    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 No
    E.8.1.3Single blind Yes
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA40
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Austria
    Bulgaria
    Lithuania
    Poland
    Romania
    Russian Federation
    Slovakia
    Slovenia
    Turkey
    Ukraine
    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
    see protocol
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months0
    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: 240
    F.1.3Elderly (>=65 years) No
    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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2010-06-18. Yes
    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 state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 180
    F.4.2.2In the whole clinical trial 240
    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)
    "Patients, who benefit/ achieve well controlled ET during treatment in this study, can be further treated with Thromboreductin®
    at the investigator’s discretion after study completion/ end of treatment (EoT) as long as the patient benefits from the treatment."
    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 Decision2010-10-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2010-07-09
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-01-12
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