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    Summary
    EudraCT Number:2014-002421-35
    Sponsor's Protocol Code Number:ISIS304801-CS6
    National Competent Authority:Hungary - National Institute of Pharmacy
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-08-27
    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 ConcernedHungary - National Institute of Pharmacy
    A.2EudraCT number2014-002421-35
    A.3Full title of the trial
    A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of ISIS 304801 Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A controlled clinical trial of ISIS 304801 in patients with Familial Chylomicronemia Syndrome (FCS)
    A.4.1Sponsor's protocol code numberISIS304801-CS6
    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 SponsorIsis Pharmaceuticals, Inc.
    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 supportIsis Pharmaceuticals
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIsis Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointTeresa Brandt
    B.5.3 Address:
    B.5.3.1Street Address2855 Gazelle Ct.
    B.5.3.2Town/ cityCarlsbad, CA
    B.5.3.3Post code92010
    B.5.3.4CountryUnited States
    B.5.4Telephone number011760603-2738
    B.5.5Fax number011760603-3891
    B.5.6E-mailtbrandt@isisph.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 numberEMA/OD/180/13
    D.3 Description of the IMP
    D.3.1Product nameApoC-III Antisense Oligonucleotide
    D.3.2Product code ISIS 304801
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNISIS 304801
    D.3.9.1CAS number 915430-78-3
    D.3.9.2Current sponsor codeISIS 304801
    D.3.9.3Other descriptive nameISIS 304801
    D.3.9.4EV Substance CodeSUB130595
    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) 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 Yes
    D.3.11.13.1Other medicinal product type2'-MOE Antisense Oligonucleotide
    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
    Familial Chylomicronemia Syndrome (FCS)
    E.1.1.1Medical condition in easily understood language
    Familial Chylomicronemia Syndrome (FCS)
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level LLT
    E.1.2Classification code 10017339
    E.1.2Term Fredrickson Type I lipidaemia
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level LLT
    E.1.2Classification code 10060593
    E.1.2Term Fredrickson Type I lipidemia
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of ISIS 304801 (300 mg once weekly) as compared to placebo on the percent change in fasting TG from baseline.
    E.2.2Secondary objectives of the trial
    To evaluate the efficacy of ISIS 304801 (300 mg once weekly) as compared to placebo on the following:
    • Postprandial TG change from baseline
    • Absolute change from baseline in fasting TG
    • Proportion of patients who achieve fasting TG <750 mg/dL
    • Proportion of patients who achieve ≥40% fasting TG reduction from baseline
    • Patient reported abdominal pain
    o Frequency
    o Severity
    • Composite of episodes of acute pancreatitis and patient reported abdominal pain
    • Change from baseline in hepatosplenomegaly as assessed by MRI
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • History of chylomicronemia
    • A diagnosis of Familial Chylomicronemia Syndrome (Type 1 Hyperlipoproteinemia)
    • Fasting triglycerides ≥ 750 mg/dL (8.4 mmol/L) at Screening
    E.4Principal exclusion criteria
    • Diabetes mellitus if newly diagnosed or if HbA1c ≥ 9.0%
    • Other types of severe hypertriglyceridemia
    • Active pancreatitis within 4 weeks of screening
    • Acute Coronary Syndrome or major surgery within 3 months of screening
    • History of heart failure
    • Treatment with Glybera gene therapy within 2 years of screening
    • Previous treatment with APOCIII Rx
    • Have any other conditions in the opinion of the investigator which could interfere with the patient participating in or completing the study
    E.5 End points
    E.5.1Primary end point(s)
    The % change in fasting TG from baseline as measured at the primary analysis time point.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The primary analysis time point is at the end of Month 3 where the value is defined as the average of Week 12 and Week 13 fasting assessments.
    E.5.2Secondary end point(s)
    • Postprandial TG (AUC) change from baseline
    • Absolute change from baseline in fasting TG as measured at the primary analysis time point.
    • Treatment response rate, where a patient with fasting plasma TG <750 mg/dL at the primary analysis time point is defined as a responder. If a patient terminates treatment before the primary analysis time point due to AE or lack of efficacy or other types of treatment failure, then the patient is considered as non-responder. If a patient terminates treatment before the primary analysis time point due to other reason or has missing fasting plasma TG at the primary analysis time point, then the patient will not be included in the analysis.
    • Treatment response rate, where a patient who achieves fasting TG ≥40% reduction from baseline at the primary analysis time point is defined as a responder. If a patient terminates treatment before the primary analysis time point due to AE or lack of efficacy or other types of treatment failure, then the patient is considered as non-responder. If a patient terminates treatment before the primary analysis time point due to other reason or has missing fasting plasma TG at the primary analysis time point, then the patient will not be included in the analysis.
    • Frequency and severity of patient reported abdominal pain during the treatment period
    • Composite of episodes of acute pancreatitis and patient reported abdominal pain during the treatment period
    • Change from baseline in hepatosplenomegaly as assessed by MRI at Week 26
    E.5.2.1Timepoint(s) of evaluation of this end point
    Month 3 (defined as the average of Week 12 and Week 13 fasting assessments), Month 6 (defined as the average of Week 25 and week 26 fasting assessments) and Month 12 (defined as the average of Week 51 and Week 52 fasting assessments)
    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 Yes
    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 No
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA18
    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
    Brazil
    Canada
    France
    Germany
    Hungary
    Israel
    Italy
    Netherlands
    Spain
    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
    LVLS
    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 months1
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months1
    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: 50
    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 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 state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 26
    F.4.2.2In the whole clinical trial 50
    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)
    Standard of Care or patients may choose to participate in the proposed Open Label Extension trial.
    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 Decision2014-10-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-10-16
    P. End of Trial
    P.End of Trial StatusCompleted
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