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    Summary
    EudraCT Number:2008-006020-53
    Sponsor's Protocol Code Number:MIPO3500108
    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:2009-04-28
    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 number2008-006020-53
    A.3Full title of the trial
    A Prospective Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of Mipomersen in Patients With Severe Hypercholesterolemia on a Maximally Tolerated Lipid-Lowering Regimen and Who are Not on Apheresis
    A.4.1Sponsor's protocol code numberMIPO3500108
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorGenzyme Europe B.V.
    B.1.3.4CountryNetherlands
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 nameMipomersen
    D.3.2Product code ISIS 301012
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeISIS 301012
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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 placeboSolution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Patients With Severe Hypercholesterolemia on a Maximally Tolerated Lipid-Lowering Regimen and Who are Not on Apheresis
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level LLT
    E.1.2Classification code 10020603
    E.1.2Term Hypercholesterolaemia
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To compare the safety and efficacy of 26 weekly, subcutaneous injections of mipomersen (200 mg) against placebo in treating severely hypercholesterolemic patients who are on a maximally tolerated lipid-lowering regimen and who are not on apheresis
    E.2.2Secondary objectives of the trial
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age ≥ 18 years
    2. Fasting TG < 350 mg/dL (4.0 mmol/L) at Screening
    3. Fasting LDL-C ≥ 200 mg/dL (5.1 mmol/L) at Screening and the presence
    of at least 1 of the following criteria:
    a) Myocardial infarction (MI)
    b) Percutaneous coronary intervention (PCI) or coronary artery bypass graft
    (CABG)
    c) CAD documented by angiography or any other accepted imaging
    technique
    d) Positive exercise test (≥ 1 mm ST-depression at maximal exercise or test
    terminated because of angina) or a perfusion defect, e.g., thallium or single
    photon emission computed tomography (SPECT)
    e) Other clinical atherosclerotic diseases: PAD, symptomatic carotid artery
    disease, AAA
    f) Or, if a) through e) are not met, fasting LDL-C ≥ 300 mg/dL (7.8 mmol/L)
    4. On a stable, maximally tolerated lipid-lowering regimen and expected to
    remain on it through Week 28 (must satisfy all criteria):
    a) A statin at a maximally tolerated dose per Investigator judgment, for at
    least 8 weeks prior to Screening
    b) A stable low-fat diet (e.g., NCEP-ATP III therapeutic lowering cholesterol
    [TLC] or equivalent) beginning at least 12 weeks prior to the first dose of
    study drug
    c) A medication from an additional class of hypolipidemic agents, per
    Investigator’s judgment (e.g., bile acid sequestrants, niacin/nicotinic acid,
    fibrates) for at least 8 weeks prior to Screening
    5. Body mass index (BMI) ≤ 40 kg/m2 with weight stable (± 4 kg) for
    > 6 weeks prior to Screening per patient report.
    6. Satisfy 1 of the following (see Section 9.2.6.8):
    a) Females: Non-pregnant and non-lactating; surgically sterile,
    postmenopausal, or patient or partner compliant with an acceptable and
    highly effective contraceptive regimen for 4 weeks prior to Screening,
    during the treatment phase, and 6 months after the last study drug dose
    b) Males: Surgically sterile or patient or partner is utilizing an acceptable and
    highly effective contraceptive method during the treatment phase and 6
    months after the last study drug dose
    7. Given informed consent
    E.4Principal exclusion criteria
    1. Any of the following diagnoses, conditions, or prior treatments:
    a) MI, PCI, CABG, cerebrovascular accident (CVA), unstable angina or
    acute coronary syndrome within 24 weeks of Screening
    b) Presence of a clinically significant arrhythmia deemed to be uncontrolled
    at any time < 12 months from screening or if medication for an arrhythmia
    has been started or dose has changed < 12 months from screening.
    Patients with implantable pacemakers or automatic implantable
    cardioverter defibrillators (AICDs) may be considered if deemed to be
    stable for the previous 12 months by the Investigator
    c) Type 1 diabetes mellitus
    d) New York Heart Association (NYHA) functional classification III or IV
    heart failure
    e) Hypertension, systolic blood pressure (BP) ≥ 160 mmHg, or diastolic BP
    ≥ 95 mmHg at Screening (despite antihypertensive medication/therapy)
    f) Active infection requiring systemic antimicrobial therapy unless treatment
    is expected to be completed prior to Day 1
    g) Positive test for human immunodeficiency virus (HIV) or hepatitis B or C
    at Screening
    h) Any uncontrolled condition that may predispose the patient to secondary
    hyperlipidemia such as uncontrolled hypothyroidism.
    i) Malignancy within 5 years, except for basal or squamous cell carcinoma of
    the skin that has been adequately treated
    j) Clinically significant hepatic or renal disease or Gilbert’s syndrome
    k) Apheresis within 3 months prior to Screening or expected to start
    apheresis during the treatment phase
    2. The following laboratory values at Screening:
    a) Serum creatine phosphokinase (CPK) ≥ 3 x upper limit of normal (ULN)
    b) Alanine aminotransferase (ALT) levels > 1.5 x ULN
    c) Serum creatinine > 0.1 mg/dL (> 8.8 µmol/L) above ULN for women, or
    > 0.2 mg/dL (> 17.7 µmol/L) above ULN for men
    d) Proteinuria (> 1+ on dipstick, confirmed on retest, with further
    confirmation by quantitative total urine protein > 1.0 g/24 hr)
    e) Total Bilirubin > 1.0 x ULN
    f) Glycosylated hemoglobin A (HbA1C) > 8.0%
    3. Use of the following medications within 12 weeks of Screening:
    a) Medications that may affect lipids except those allowed per the protocol,
    including but not limited to Cholestin™ (also known as red yeast rice, or
    monascus purpureus extract)
    b) Chronic systemic corticosteroids or anabolic agents except for replacement
    therapy
    c) Oral contraceptives or contraceptive patch
    4. Use of the following medications unless a stable dose regimen was used for at
    least 12 weeks prior to Screening and the dose and regimen are expected to
    remain stable until Week 28:
    a) Oral anticoagulants (e.g., warfarin)
    b) Hormone replacement therapy
    c) Diabetes medications including but not limited to sulfonylureas,
    metformin and glitazones, with the exception of changes of ± 10 units of
    insulin.
    d) Antiviral therapy for herpes simplex virus (HSV)
    e) Anti-obesity medications
    5. Treatment with another investigational drug, biological agent, or device within
    4 weeks of Screening or 5 half-lives of study agent, whichever is longer
    6. Recent history of, or current, drug or alcohol abuse, or unwillingness to limit
    alcohol consumption for the entire duration of the study, including follow-up
    7. Any disorders that would limit study participation or unwillingness to comply
    with study procedures, including follow-up, as specified by this protocol, or
    unwillingness to cooperate fully with the Investigator
    8. Have any other medical conditions that, in the opinion of the Investigator,
    would make the patient unsuitable for enrollment, or could interfere with the
    patient participating in or completing the study
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is the percent change in LDL-C from Baseline to
    Week 28/Early Termination (ET represents the observation closest to 2 weeks after last dose among patients who terminate study medication dosing early).
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    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 No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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.8.3 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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA11
    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
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    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 years2
    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.1.3Elderly (>=65 years) Yes
    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 65
    F.4.2.2In the whole clinical trial 75
    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 Decision2009-02-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2009-04-17
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2010-10-14
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