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    Summary
    EudraCT Number:2015-003755-21
    Sponsor's Protocol Code Number:ISIS304801-CS7
    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:2016-02-26
    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 number2015-003755-21
    A.3Full title of the trial
    An Open-Label Study of Volanesorsen 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
    An open-label clinical trial of volanesorsen in patients with Familial Chylomicronemia Syndrome (FCS)
    A.4.1Sponsor's protocol code numberISIS304801-CS7
    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 SponsorAkcea Therapeutics
    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 supportIonis Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAkcea Therapeutics
    B.5.2Functional name of contact point
    B.5.3 Address:
    B.5.3.1Street Address22 Boston Wharf Road, 09th floor
    B.5.3.2Town/ cityBoston
    B.5.3.3Post codeMA 02210
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1617 207 0298
    B.5.5Fax number+1760602-1854
    B.5.6E-mailjmacpherson@akceatx.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 nameVolanesorsen
    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 INNVolanesorsen
    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
    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 20.0
    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 20.0
    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 safety and efficacy of dosing and of extended dosing with volanesorsen (volanesorsen sodium 300 mg) in patients with FCS
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Able and willing to participate in a 65-week study
    2. Group 1 and 2: Satisfactory completion of ISIS 304801-CS6 or ISIS 304801-
    CS16 (index studies) with an acceptable safety profile, per
    Sponsor and Investigator judgment
    3. Group 3: Patients who did not participate in the CS6 or CS16 index
    studies and meet additional inclusion criteria of Familial
    Chylomicronemia Syndrome (FCS) may enroll in the study.
    3a. History of chylomicronemia
    3b. A diagnosis of Familial Chylomicronemia Syndrome (Type 1 Hyperlipoproteinemia)
    3c. Fasting triglycerides ≥ 750 mg/dL (8.4 mmol/L) at Screening
    E.4Principal exclusion criteria
    For Group 1 and Group 2:
    1. Have any new condition or worsening of existing condition which 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
    2. Unwilling to comply with lifestyle requirements for the duration of the study
    For Group 3:
    a. Diabetes mellitus if newly diagnosed or if HbA1c ≥ 9.0%
    b. Active pancreatitis within 4 weeks of screening
    c. Acute Coronary Syndrome within 6 months of screening
    d. Major surgery within 3 months of screening
    e. Treatment with Glybera therapy within 2 years of screening
    f. 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)
    Efficacy Endpoints:
    • Percent change and absolute change from baseline in fasting TG
    • Frequency and severity of patient reported abdominal pain during the treatment period
    • Percent change and change from baseline in other fasting lipid measurements including total cholesterol, non-HDL-C, apoB, HDL-C,
    apolipoprotein A-1 (apoA-1), VLDL-C, and LDL-C
    • Percent change from baseline in fasting total apolipoprotein C-III
    • Quality of Life questionnaires (EQ-5D, SF-36)
    • Adjudicated acute pancreatitis event rate
    • Other symptoms: eruptive xanthoma, lipemia retinalis
    Safety Endpoints:
    • Adverse events including adjudicated events of pancreatitis and MACE
    • Vital signs and weight
    • Physical examinations
    • Clinical laboratory tests (serum chemistry, hematology, coagulation, urinalysis)
    • Echocardiography
    • Electrocardiograms (ECGs)
    • Use of concomitant medications
    • MRIs
    E.5.1.1Timepoint(s) of evaluation of this end point
    Fasting lipid measurements: Month 3 (average of W12 (D78) and W13 (D85) fasting assessments), Month 6 (average of W25 (D169) and W26 (D176) fasting assessments), and Month 12 (average of W51 (D351) and W52 (D358) fasting assessments) - For Extended Treatment Period: Month 17 (average of W70 (D484) and W74 (D512) fasting assessments), Month 21 (average of W86 (D596) and W90 (D624) fasting assessments).
    Abdominal pain: Weekly from Qualification to W65
    Quality of Life questionnaires: W1, 13, 26, 52, 65
    AEs including acute pancreatitis, eruptive xanthoma: at each visit
    Lipemia retinalis: Qualification and W52
    Clinical laboratory tests: every week
    Echocardiography: W26 and 52
    Electrocardiograms: W13, 26, 38, 52, 65 - If Extended Treatment Period: W76 and 104
    MRI: W52
    E.5.2Secondary end point(s)
    None
    E.5.2.1Timepoint(s) of evaluation of this end point
    None
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other No
    E.8.3 The trial involves single site in the Member State concerned Information not present in EudraCT
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA15
    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
    Israel
    Italy
    Netherlands
    South Africa
    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 years1
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months4
    E.8.9.2In all countries concerned by the trial days9
    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: 70
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 35
    F.4.2.2In the whole clinical trial 70
    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
    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 Decision2016-04-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-06-06
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-01-15
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