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    Summary
    EudraCT Number:2015-003755-21
    Sponsor's Protocol Code Number:ISIS304801-CS7
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2016-03-18
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2015-003755-21
    A.3Full title of the trial
    An Open-Label Extension Study of Volanesorsen Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS)
    Estudio de extensión abierto de volanesorsén administrado por vía subcutánea a pacientes con síndrome de hiperquilomicronemia familiar (SHQF)
    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)
    Estudio de extensión abierto de volanesorsén en pacientes con síndrome de hiperquilomicronemia familiar (SHQF)
    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 SponsorIonis 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 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 organisationMedpace Spain S.L.
    B.5.2Functional name of contact pointMónica Bermejo
    B.5.3 Address:
    B.5.3.1Street AddressJorge Juan 54, 2izq
    B.5.3.2Town/ cityMadrid
    B.5.3.3Post code28001
    B.5.3.4CountrySpain
    B.5.4Telephone number34 91 853 4105
    B.5.5Fax number34 900 981 853
    B.5.6E-mailm.bermejo@medpace.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)
    síndrome de hiperquilomicronemia familiar (SHQF)
    E.1.1.1Medical condition in easily understood language
    Familial Chylomicronemia Syndrome (FCS)
    síndrome de hiperquilomicronemia familiar (SHQF)
    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 18.1
    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 18.1
    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 safety and efficacy of extended dosing with volanesorsen (volanesorsen sodium 300 mg) in patients with FCS
    Evaluar la seguridad y la eficacia de la ampliación del tratamiento con
    volanesorsén (300 mg de volanesorsén sódico) en pacientes con SHQF.
    E.2.2Secondary objectives of the trial
    Not applicable
    No aplicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Satisfactory completion of ISIS 304801-CS6 or ISIS 304801-CS16 (index studies) with an acceptable safety profile
    2. History of chylomicronemia
    3. A diagnosis of Familial Chylomicronemia Syndrome (Type 1 Hyperlipoproteinemia)
    4. Fasting triglycerides ? 750 mg/dL (8.4 mmol/L) at Screening for the index studies
    1. Cumplimiento terapéutico satisfactorio de ISIS 304801-CS6 o 304801-
    CS16 (estudios de referencia) con un perfil de seguridad aceptable
    2. antecedentes de hiperquilomicronemia
    3. Diagnóstico de síndrome de hiperquilomicronemia familiar
    (hiperlipoproteinemia de tipo 1)
    4. TG en ayunas ? 750 mg/dl (8,4 mmol/l) en el screening para el
    estudio ISIS 304801-CS16.
    E.4Principal exclusion criteria
    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.
    1. Presencia de cualquier proceso patológico nuevo o empeoramiento
    de un proceso ya existente que, en opinión del investigador, haría
    que el paciente no fuera apto para su inclusión en el estudio, o
    podría interferir en la participación del paciente en el estudio o
    impedir que lo finalizase.
    2. Pacientes que no estén dispuestos a seguir las exigencias sobre el estilo de
    vida mientras dure el estudio
    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
    Criterios de valoración de la eficacia:
    - El cambio porcentual y el cambio absoluto respecto al periodo basal de las concentraciones de TG en ayunas
    - La frecuencia y la intensidad del dolor abdominal referido por los pacientes durante el periodo de tratamiento.
    - El cambio porcentual y el cambio respecto al periodo basal de las determinaciones de lípidos en ayunas, entre otras, colesterol total, colesterol transportado por lipoproteínas no de alta densidad (colesterol no-C-HDL), apolipoproteína B [apoB], colesterol transportado por lipoproteínas de alta densidad (C-HDL),
    apolipoproteína A-1 [apoA-1], colesterol transportado por lipoproteínas de muy baja densidad (C-VLDL) y C-LDL.
    - El cambio porcentual respecto al periodo basal de las concentraciones de apolipoproteína C-III (apoC-III) en ayunas.
    - Cuestionarios de la calidad de vida (EQ-5D, SF-36).
    - Índice de acontecimientos de pancreatitis aguda validados.
    - Otros síntomas: xantoma eruptivo, lipemia retiniana.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Fasting lipid measurements: Month 3 (defined as the average of Week 12 (Day 78) and Week 13 (Day 85) fasting assessments), Month 6 (defined as the average of Week 25 (Day 169) and Week 26 (Day 176) fasting assessments), and Month 12 (defined as the average of Week 51 (Day 351) and Week 52 (Day 358) fasting assessments).
    Abdominal pain: Weekly from Qualification to week 65
    Quality of Life questionnaires: Weeks 1, 13, 26, 52, 65
    Adverse events including acute pancreatitis, eruptive xanthoma: Qualification, weeks 1, 4, 8, 13, 26, 38, 52, 65
    Lipemia retinalis: Qualification and Week 52
    Clinical laboratory tests: Screening, weeks 1, 2, 4, 8, 12, 13, 19, 25, 26, 32, 38, 44, 51, 52, 58, 65
    Echocardiography: Weeks 26 and 52
    Electrocardiograms: Weeks 13, 26, 38, 52, 65
    MRI: Week 52
    Medidas de lípidos en ayunas: mes 3, mes 6 y mes 12
    Dolor abdominal: seminal desde la visita de selección hasta la semana 65
    Cuestionarios de calidad de vida: semanas 1, 13, 26, 52, 65
    Acontecimientos adversos que incluyen pancreatitis aguda, xantoma eruptive: voisita de selección, semanas 1, 4, 8, 13, 26, 38, 52, 65
    Lipemia retinalis: visita de selección y semana 52
    test de laboratorio: visita de screening, semanas 1, 2, 4, 8, 12, 13, 19, 25, 26, 32, 38, 44, 51, 52, 58, 65
    Ecocardiografía: semanas 26 y 52
    electrocardiograma: semanas 13, 26, 38, 52, 65
    MRI: semana 52
    E.5.2Secondary end point(s)
    None
    ninguno
    E.5.2.1Timepoint(s) of evaluation of this end point
    None
    Ninguno
    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 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 concerned5
    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
    última visita del ultimo paciente
    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 months2
    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 months2
    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: 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 state7
    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
    Práctica clínica habitual
    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 Decision2016-05-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-05-04
    P. End of Trial
    P.End of Trial StatusCompleted
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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