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    Summary
    EudraCT Number:2011-001480-42
    Sponsor's Protocol Code Number:MIPO3801011
    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:2011-11-23
    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 number2011-001480-42
    A.3Full title of the trial
    A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients with Familial Hypercholesterolemia and Inadequately Controlled Low-Density-Lipoprotein Cholesterol
    Estudio de fase 3, randomizado, doble ciego, controlado con placebo y de grupos paralelos para evaluar la seguridad y la eficacia de dos pautas posológicas diferentes de Mipomersen en pacientes con hipercolesterolemia familiar y colesterol asociado a lipoproteínas de baja densidad insuficientemente controlado
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol
    Estudio de seguridad y eficacia de 2 regímenes diferentes de Mipomersen en pacientes con Hipercolesterolemia y control inadecuado de lipoproteinas de baja densidad.
    A.3.2Name or abbreviated title of the trial where available
    FOCUS FH
    A.4.1Sponsor's protocol code numberMIPO3801011
    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 SponsorGenzyme Corporation and its Affiliates
    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 supportGenzyme Corporation and its Affiliates
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGenzyme Europe B.V.
    B.5.2Functional name of contact pointMedical Information Genzyme Europe
    B.5.3 Address:
    B.5.3.1Street AddressGooimer 10
    B.5.3.2Town/ cityNaarden
    B.5.3.3Post code1411 DD
    B.5.3.4CountryNetherlands
    B.5.6E-maileumedinfo@genzyme.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 nameMipomersen
    D.3.2Product code ISIS 301012
    D.3.4Pharmaceutical form Solution for injection in pre-filled syringe
    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 INNMipomersen
    D.3.9.1CAS number 629167-92-6
    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) 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 No
    D.IMP: 2
    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 in pre-filled syringe
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    Subcutaneous use
    Parenteral use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNMipomersen
    D.3.9.1CAS number 629167-92-6
    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 number140
    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 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 in pre-filled syringe
    D.8.4Route of administration of the placeboSubcutaneous use
    D.8 Placebo: 2
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection in pre-filled syringe
    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
    Heterozygous familial hypercholesterolemia
    Hipercolesterolemia Familiar heterocigótica
    E.1.1.1Medical condition in easily understood language
    Heterozygous familial hypercholesterolemia
    Hipercolesterolemia Familiar heterocigótica
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level LLT
    E.1.2Classification code 10057079
    E.1.2Term Heterozygous familial hypercholesterolemia
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Determine whether mipomersen (ISIS 301012) significantly reduces atherogenic lipid levels in patients with severe heterozygous familial hypercholesterolemia (severe HeFH), defined as low-density lipoprotein cholesterol (LDL-C) levels ?200 mg/dL plus the presence of coronary heart disease (CHD)/risk equivalents or LDL-C levels ?300 mg/dL regardless of the presence of CHD/risk equivalents (referred to as Cohort 1) compared to placebo. Two different mipomersen dosing regimens will be studied: subcutaneous (SC) mipomersen 200 mg once weekly versus placebo, and SC mipomersen 70 mg thrice weekly versus placebo.
    El objetivo primario de este estudio es determinar si mipomersen (ISIS 301012) reduce de manera significativa las concentraciones de lípidos aterogénicos en los pacientes con hipercolesterolemia familiar heterocigótica grave (HFHe grave), definida como concentraciones de colesterol asociado a lipoproteínas de baja densidad (C-LDL) ?200 mg/dl más la presencia de arteriopatía coronaria (CHD)/equivalentes de riesgo o concentraciones de C-LDL ?300 mg/dl independientemente de la presencia de arteriopatía coronaria (CDC)/equivalentes de riesgo (a los que se hace referencia como Cohorte 1) en comparación con placebo. Se estudiarán dos pautas posológicas diferentes de mipomersen: 200 mg de mipomersen subcutáneo (SC) una vez a la semana en comparación con placebo y 70 mg de mipomersen SC tres veces a la semana en comparación con placebo.
    E.2.2Secondary objectives of the trial
    - Determine whether there are qualitative differences between the safety profiles of the 2 dosing regimens and placebo in Cohort 1, patients with HeFH with LDL-C levels ?160 mg/dL and <200 mg/dL plus the presence of CHD/risk equivalents (referred to as Cohort 2), and the overall study population (comprising patients in both Cohort 1 and Cohort 2)
    - Determine whether there are qualitative differences between the tolerability of the 2 dosing regimens and placebo in Cohort 1, Cohort 2, and the overall study population
    -Further characterize the pharmacokinetics (PK) of the 2 dosing regimens in Cohort 1, Cohort 2, and the overall study population
    - Determine whether the 2 mipomersen dosing regimens significantly reduce atherogenic lipid levels in Cohort 2 compared to placebo
    -Determinar si existen diferencias cualitativas entre los perfiles de seguridad de las dos pautas posológicas y el placebo en la cohorte 1, pacientes con HFHe con concentraciones de C-LDL ?160 mg/dl y <200 mg/dl más la presencia de arteriopatía coronaria /de equivalentes de riesgo (a los que se hace referencia como la cohorte 2) y la población general del estudio (que abarca pacientes de la cohorte 1 y la cohorte 2)
    -Determinar si existen diferencias cualitativas entre la tolerabilidad de las dos pautas posológicas y el placebo en la cohorte 1, la cohorte 2 y la población general del estudio
    -Además determinar la farmacocinética (FC) de las dos pautas posológicas en la cohorte 1, la cohorte 2 y la población general del estudio
    -Determinar si las dos pautas posológicas de mipomersen reducen de manera significativa las concentraciones de lípidos aterogénicos en la cohorte 2 en comparación con el placebo.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Diagnosis of severe hypercholesterolemia (LDL-C ?300 mg/dL (7.77 mmol/L) or LDL-C ? 200 mg/dL (5.18 mmol/L) and documented coronary heart disease (CHD) or CHD risk equivalent or diagnosis of Heterozygous Familial Hypercholesterolemia and LDL-C ?160 mg/dL (4.14 mmol/L) and <200 mg/dL (5.18 mmol/L))
    On stable, maximally tolerated, statin therapy for at least 12 weeks or if statin intolerant, on at least 1 medication from another class of hypolipidemic agents (i.e., bile acid sequestrants, niacin/nicotinic acid, cholesterol absorption inhibitors, fibrates).
    On stable, low fat diet for 12 weeks
    Body mass index (BMI) ?40 kg/m2 and stable weight for 6 weeks
    Diagnostico de Hipercolesterolemia servera (LDL-C ?300 mg/dL (7.77 mmol/L) o LDL-C ? 200 mg/dL (5.18 mmol/L) y en presencia de arteriopatía coronaria o equivalentes de riesgo o diagnósitico de Hipercolesterolemia Heterocigotica Familiar y LDL-C ?160 mg/dL (4.14 mmol/L) y <200 mg/dL (5.18 mmol/L))
    El paciente está en tratamiento estable con máxima dosis de estatinas durante al menos doce semanas antes del screening o en caso de intolerancia a las estatinas en tratamiento con al menos un hipolipemiante (es decir, secuestradores de ácidos biliares, niacina/ácido nicotínico, inhibidores de la absorción de colesterol, fibratos).
    El paciente está en una dieta estable baja en grasas durante al menos 12 semanas antes de screening y presenta un índice de masa corporal ?40 kg/m2 con peso estable (± 4 kg) durante >6 semanas antes del Screening.
    E.4Principal exclusion criteria
    Significant health problems in the recent past including heart attack, stroke, coronary syndrome, unstable angina, heart failure, significant arrhythmia, hypertension, blood disorders, liver disease, cancer, digestive disorders, Type I diabetes, or uncontrolled Type II diabetes
    Apheresis within 3 months prior to Screening or expected to start apheresis during the treatment phase
    El paciente ha tenido problemas cardiacos en el pasado, sufrido un IM, una intervención coronaria transluminal percutánea, CABG, un accidente cerebrovascular, angina de pecho inestable, un síndrome coronario agudo, arritmia clínicamente significativa, hipertensión, hepatopatía clínicamente significativa, problemas hepáticos, cancer, desordenes digestivos, diabetes mellitus (tipo 1) insulinodependiente, o diabetes de tipo 2 hemoglobina A glucosilada (HbA1c; glucohemoglobina) >8% en el screening.
    El paciente se ha sometido a aféresis en los tres meses previos al screening o tiene previsto comenzar la aféresis durante la fase de tratamiento
    E.5 End points
    E.5.1Primary end point(s)
    Percent change from Baseline in low-density lipoprotein cholesterol in Cohort 1
    Cambio porcentual desde el basal en C-LDL en la cohorte 1
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline to Week 60
    Basal a la semana 60
    E.5.2Secondary end point(s)
    Percent change from Baseline in Apolipoprotein
    Percent change from Baseline in Lipoprotein a
    Percent change from Baseline in low-density lipoprotein cholesterol in Cohort 2
    Number of Participants with Adverse Events
    Number of Participants with injection site reactions
    Blood plasma concentration of Mipomersen
    Cambio porcentual desde el basal en Apolipoproteina
    Cambio porcentual desde el basal en Lipoproteina a
    Cambio porcentual desde el basal en C-LDL en la cohorte 2
    Número de participantes con acontecimientos adversos
    Número de participantes con reacciones en puntos de inyección
    Concentración plasmática de Mipomersen
    E.5.2.1Timepoint(s) of evaluation of this end point
    Percent change from Baseline in Apolipoprotein : Baseline to Week 60
    Percent change from Baseline in Lipoprotein a : Baseline to Week 60
    Percent change from Baseline in low-density lipoprotein cholesterol in Cohort 2 : Baseline to Week 60
    Number of Participants with Adverse Events : 60 weeks
    Number of Participants with injection site reactions : 60 weeks
    Blood plasma concentration of Mipomersen : Pre-dose and at 2, 4, 6, 24, 48 and 72 hours post-dose at Weeks 1, 30, 36 and 60.
    Cambio porcentual desde el basal en Apolipoproteina: Basal a semana 60
    Cambio porcentual desde el basal en Lipoproteina a: basal a semana 60
    Cambio porcentual desde el basal en C-LDL en la cohorte 2: basal a semana 60
    Número de participantes con acontecimientos adversos: basal a semana 60
    Número de participantes con reacciones en puntos de inyección: basal a semana 60
    Concentración plasmática de Mipomersen: pre-dosis y a las 2, 4, 6, 24, 48 y 72 horas post-dosis en las semanas 1, 30, 36 y 60
    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 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 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.8.2.4Number of treatment arms in the trial4
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA61
    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
    Argentina
    Australia
    Brazil
    Canada
    Croatia
    Hong Kong
    India
    Israel
    Malaysia
    New Zealand
    Norway
    Russian Federation
    South Africa
    Taiwan
    Turkey
    Ukraine
    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
    Last visit of the last subject
    ultima visita del último 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 months8
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    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: 460
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 260
    F.4.2.2In the whole clinical trial 900
    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)
    Not applicable
    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 Decision2012-04-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-04-12
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-03-15
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