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    Summary
    EudraCT Number:2021-003280-95
    Sponsor's Protocol Code Number:ISIS678354-CS13
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-03-10
    Trial 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 number2021-003280-95
    A.3Full title of the trial
    An Open-Label Extension Study of AKCEA-APOCIII-LRX Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS)
    Estudio abierto de prolongación de AKCEA-APOCIII-LRx administrado por vía subcutánea a pacientes con síndrome de quilomicronemia familiar (SQF)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Open-Label Extension Study of AKCEA-APOCIII-LRX Administered to Patients with Familial Chylomicronemia Syndrome (FCS)
    Estudio abierto de prolongación de AKCEA-APOCIII-LRx administrado a pacientes con síndrome de quilomicronemia familiar (SQF)
    A.4.1Sponsor's protocol code numberISIS678354-CS13
    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
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIonis Pharmaceuticals
    B.5.2Functional name of contact pointStacy Woeppel
    B.5.3 Address:
    B.5.3.1Street Address2855 Gazelle Court
    B.5.3.2Town/ cityCarlsbad
    B.5.3.3Post code92010
    B.5.3.4CountryUnited States
    B.5.4Telephone number1 760 268 4986
    B.5.6E-mailclinicaltrials@ionisph.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 nameISIS 678354
    D.3.2Product code ISIS 678354
    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 INNSUB208551
    D.3.9.3Other descriptive nameISIS 678354 sodium salt
    D.3.9.4EV Substance CodeSUB208551
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 typeAntisense 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 quilomicronemia familiar (SQF)
    E.1.1.1Medical condition in easily understood language
    Patients with hypertriglyceridemia
    Pacientes con hipertrigliceridemia
    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 PT
    E.1.2Classification code 10059183
    E.1.2Term Familial hypertriglyceridaemia
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10020607
    E.1.2Term Hyperchylomicronemia
    E.1.2System Organ Class 100000004861
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective is to evaluate the effect of ISIS 678354 on the percent change in fasting triglycerides (TG) from Baseline
    El objetivo principal es evaluar el efecto de ISIS 678354 sobre la variación porcentual de los triglicéridos en ayunas (TG en el momento basal).
    E.2.2Secondary objectives of the trial
    To evaluate effect of ISIS 678354 as compared to baseline on:
    • Percent change in fasting TG upon extended treatment (durability of the effect)
    • Proportion of patients who achieve ≥ 40% reduction in fasting TG from Baseline
    • Percent change in fasting apolipoprotein B (apoB) and apolipoprotein 48 (apoB48) from Baseline
    • Proportion of patients who achieve fasting TG ≤ 750 mg/dL
    • Adjudicated acute pancreatitis event rate during the Treatment Period (Week 1 through Week 53 in patients with ≥ 2 events of adjudicated acute pancreatitis in 5 years prior to treatment with ISIS 678354.
    • Adjudicated acute pancreatitis event rate during the Treatment Period (Week 1 through Week 53)
    • Proportion of patients who achieve ≥ 70% reduction in fasting TG from Baseline
    • Proportion of patients who achieve fasting TG ≤ 500 mg/dL
    Evaluar el efecto de ISIS 678354 sobre:
    • Variación porcentual de los TG en ayunas entre el momento basal y el período de tratamiento prolongado (duración del efecto).
    • Proporción de pacientes que logran una reducción ≥ 40 % de los TG en ayunas con respecto al momento basal.
    • Variación porcentual de la apoB y la apoB48 en ayunas con respecto al momento basal.
    • Proporción de pacientes que logran un valor de TG en ayunas ≤ 750 mg/dl.
    • Tasa de episodios de pancreatitis aguda confirmados durante el período de tratamiento en pacientes con ≥ 2 episodios de pancreatitis aguda confirmados en los 5 años previos a la inclusión.
    • Tasa de episodios de pancreatitis aguda confirmados durante el período de tratamiento
    • Proporción de pacientes que logran una reducción ≥ 70 % de los TG en ayunas con respecto al momento basal.
    • Proporción de pacientes que logran un valor de TG en ayunas ≤ 500 mg/dl.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    2. Satisfactory completion of treatment with ISIS 678354 in the index study (last dose as scheduled at Week 49) with an acceptable safety profile, per Investigator judgement
    3. Willing to follow a diet comprising ≤ 20 g fat per day during the study
    2. Finalización satisfactoria del tratamiento con ISIS 678354 en el estudio de referencia (última dosis programada en la semana 49) con un perfil de seguridad aceptable, según el criterio del investigador.
    3. Compromiso de seguir una dieta de ≤ 20 g de grasa por día durante la duración del estudio.
    E.4Principal exclusion criteria
    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, including need for treatment with medications disallowed in the index study (ISIS 678354-CS3).
    Presencia de cualquier trastorno nuevo o empeoramiento de un trastorno existente que, en opinión del investigador, haga que el paciente no sea apto para el reclutamiento o que pueda interferir en la participación o la finalización del estudio, incluida la necesidad de tratamiento con medicamentos prohibidos en el estudio de referencia (ISIS 678354-CS3).
    E.5 End points
    E.5.1Primary end point(s)
    The primary objective is to evaluate safety and tolerability of treatment with ISIS 678354
    El objetivo principal es evaluar la seguridad y la tolerabilidad del tratamiento con ISIS 678354
    E.5.1.1Timepoint(s) of evaluation of this end point
    6 months
    6 meses
    E.5.2Secondary end point(s)
    • Percent change in fasting TG from Baseline at 6 months (average of Weeks 23, 25 and 27)
    • Percent change in fasting TG from Baseline at 12 months (average of Week 51 and 53)
    • Proportion of patients who achieve ≥ 40% reduction in fasting TG from Baseline at 6 months
    • Proportion of patients who achieve ≥ 40% reduction in fasting TG from Baseline at 12 months
    • Percent change in fasting apoB48 from Baseline at 6 months
    • Percent change in fasting apoB48 from Baseline at 12 months
    • Proportion of patients who achieve fasting TG ≤ 750 mg/dL at 6 months
    • Proportion of patients who achieve fasting TG ≤ 750 mg/dL at 12 months
    • Adjudicated acute pancreatitis event rate during the Treatment Period (Week 1 through Week 53), in patients with ≥ 2 events of adjudicated acute pancreatitis in 5 years prior to treatment with ISIS 678354
    • Adjudicated acute pancreatitis event rate during the Treatment Period (Week 1 through Week 53)
    • Proportion of patients who achieve ≥ 70% reduction in fasting TG from Baseline at 6 months
    • Proportion of patients who achieve ≥ 70% reduction in fasting TG from Baseline at 12 months
    • Proportion of patients who achieve fasting TG ≤ 500 mg/dL at 6 months
    • Proportion of patients who achieve fasting TG ≤ 500 mg/dL at 12 months
    • Variación porcentual de los TG en ayunas entre el momento basal y los 6 meses (media de las semanas 23, 25 y 27)
    • Variación porcentual de los TG en ayunas entre el momento basal y los 12 meses (media de las semanas 51 y 53)
    • Proporción de pacientes que logran una reducción ≥ 40 % de los TG en ayunas entre el momento basal y los 6 meses.
    • Proporción de pacientes que logran una reducción ≥ 40 % de los TG en ayunas entre el momento basal y los 12 meses.
    • Variación porcentual de la apoB48 en ayunas entre el momento basal y los 6 meses.
    • Variación porcentual de la apoB48 en ayunas entre el momento basal y los 12 meses.
    • Proporción de pacientes que logran un valor de TG en ayunas ≤ 750 mg/dl a los 6 meses.
    • Proporción de pacientes que logran un valor de TG en ayunas ≤ 750 mg/dl a los 12 meses.
    •Tasa de episodios de pancreatitis aguda confirmados durante el período de tratamiento (de la semana 1 a la semana 53) en pacientes con ≥ 2 episodios depancreatitis aguda confirmados en los 5 años previos al tratamiento con ISIS 678354.
    • Tasa de episodios de pancreatitis aguda confirmados durante el período de tratamiento (de la semana 1 a la semana 53).
    • Proporción de pacientes que logran una reducción ≥ 70 % de los TG en ayunas entre el momento basal y los 6 meses.
    • Proporción de pacientes que logran una reducción ≥ 70 % de los TG en ayunas entre el momento basal y los 12 meses.
    • Proporción de pacientes que logran un valor de TG en ayunas ≤ 500 mg/dl a los 6 meses.
    • Proporción de pacientes que logran un valor de TG en ayunas ≤ 500 mg/dl a los 12 meses.
    E.5.2.1Timepoint(s) of evaluation of this end point
    6 months and 12 months
    6 meses y 12 meses
    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 concerned13
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA14
    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
    Canada
    Israel
    United States
    France
    Germany
    Hungary
    Italy
    Netherlands
    Norway
    Portugal
    Slovakia
    Spain
    Sweden
    United Kingdom
    E.8.7Trial has a data monitoring committee No
    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
    UPUV
    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 months4
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months4
    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: 55
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 state13
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 60
    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)
    None
    Ninguno
    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 Decision2022-06-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-04-27
    P. End of Trial
    P.End of Trial StatusOngoing
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