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    Summary
    EudraCT Number:2017-000351-95
    Sponsor's Protocol Code Number:R727-CL-1628
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-06-23
    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 ConcernedFrance - ANSM
    A.2EudraCT number2017-000351-95
    A.3Full title of the trial
    A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY TO EVALUATE THE EFFICACY AND SAFETY OF ALIROCUMAB IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA
    ÉTUDE RANDOMISÉE, EN DOUBLE AVEUGLE, CONTRÔLÉE PAR PLACEBO, EN GROUPES PARALLÈLES VISANT À ÉVALUER L’EFFICACITÉ ET L’INNOCUITÉ DE L’ALIROCUMAB CHEZ DES PATIENTS ATTEINTS D’HYPERCHOLESTÉROLÉMIE FAMILIALE HOMOZYGOTE
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to the safety and efficacy of alirocumab in patients with Hereditary abnormal (high) cholesterol level
    Étude sur la sécurité d’emploi et l’efficacité de l’alirocumab chez des patients présentant un taux de cholestérol anormal (élevé) héréditaire
    A.3.2Name or abbreviated title of the trial where available
    ODYSSEY HoFH
    ODYSSEY HoFH
    A.4.1Sponsor's protocol code numberR727-CL-1628
    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 SponsorRegeneron 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 supportRegeneron Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRegeneron Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address777 Old Saw Mill River Road
    B.5.3.2Town/ cityTarrytown
    B.5.3.3Post code10591
    B.5.3.4CountryUnited States
    B.5.6E-mailclinicaltrials@regeneron.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 Yes
    D.2.1.1.1Trade name Praluent
    D.2.1.1.2Name of the Marketing Authorisation holdersanofi-aventis groupe
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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.3.4Pharmaceutical form Solution for injection in pre-filled pen
    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 INNALIROCUMAB
    D.3.9.1CAS number 1245916-14-6
    D.3.9.3Other descriptive nameALIROCUMAB
    D.3.9.4EV Substance CodeSUB74847
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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 pen
    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
    HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA
    Hypercholestérolémie familiale homozygote
    E.1.1.1Medical condition in easily understood language
    Homozygous familial hypercholesterolemia is an inherited genetic condition which results in the presence of high levels of cholesterol in the blood
    L’hypercholestérolémie familiale homozygote est une maladie génétique héréditaire qui se traduit par des taux élevés de cholestérol dans le sang
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10057080
    E.1.2Term Homozygous familial hypercholesterolemia
    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
    The primary objective of the study is to demonstrate the reduction of LDL-C with alirocumab 150 mg subcutaneous (SC) every 2 weeks (Q2W) in comparison to placebo after 12 weeks of treatment.
    L’objectif principal de l’étude est de démontrer la baisse du cholestérol à lipoprotéines de faible densité (LDL-C) avec 150 mg d’alirocumab par voie sous-cutanée (SC) toutes les 2 semaines (1x/2 sem.) par rapport à un placebo au bout de 12 semaines de traitement.
    E.2.2Secondary objectives of the trial
    The secondary objectives of the study are:
    • To evaluate the effect of alirocumab 150 mg Q2W on other lipid parameters (i.e., apolipoprotein [Apo] A-1 and B, non-high-density lipoprotein cholesterol
    [non-HDL-C], total cholesterol [TC], proportion of patients with 15%, 30%, and 50% LDL-C reductions, Lp(a), HDL-C, triglycerides [TG]) in patients with HoFH
    • To evaluate the safety and tolerability of alirocumab 150 mg SC Q2W in patients with HoFH
    • To assess the pharmacokinetics of alirocumab 150 mg SC Q2W in patients with HoFH
    • To assess the potential development of anti-drug (alirocumab) antibodies
    Les objectifs secondaires de l’étude sont :
    • Évaluer l’effet de 150 mg d’alirocumab 1x/2 sem. sur les autres paramètres lipidiques (c.-à-d. les apolipoprotéines [Apo] A-1 et B, le cholestérol à lipoprotéines non à haute densité [non-HDL-C], le cholestérol total [TC], la proportion de patients associés à des baisses de 15 %, 30 % et 50 % du LDL-C, les Lp(a), le HDL-C, les triglycérides [TG]) chez des patients présentant une hypercholestérolémie familiale homozygote (HFHo)
    • Évaluer la sécurité d’emploi et la tolérance de 150 mg d’alirocumab par voie SC 1x/2 sem. chez des patients présentant une HFHo
    • Évaluer la pharmacocinétique de 150 mg d’alirocumab par voie SC 1x/2 sem. chez des patients présentant une HFHo
    • Évaluer le développement potentiel d’anticorps anti-médicament (alirocumab)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Males and females ≥12 years of age at the time of the screening visit.
    2. Adolescents ≥12 years of age and <18 years of age should be >50 kg at the time of the screening visit.
    3. Diagnosis of HoFH homozygous familial hypercholesterolemia (HoFH)
    4. Receiving a stable dose of a statin at the screening visit
    1. Patients des deux sexes âgés de ≥ 12 ans au moment de la visite de sélection.
    2. Les adolescents âgés de ≥ 12 ans et de < 18 ans doivent peser > 50 kg au moment de la visite de sélection.
    3. Diagnostic d’hypercholestérolémie familiale homozygote (HFHo).
    4. Patients recevant une dose stable d’une statine au moment de la visite de sélection.
    E.4Principal exclusion criteria
    1. Documented evidence of a null mutation in both LDLR alleles
    2. Use of a PCSK9 inhibitor within 10 weeks from screening visit.
    3. Background medical LMT that has not been stable for at least 4 weeks (6 weeks for
    fibrates, 24 weeks for mipomersen, 12 weeks for maximum tolerated dose of lomitapide)
    before the screening visit.
    4. LDL apheresis schedule/apheresis settings that have not been stable for at least 8 weeks before the screening visit or an apheresis schedule/settings that is not anticipated to be stable over the next 24 weeks.
    5. Use of nutraceuticals or over-the-counter (OTC) therapies known to affect lipids, at a dose/amount that has not been stable for at least 4 weeks prior to the screening visit or between the screening and randomization visits.
    1. Preuve documentée d’une mutation nulle dans les deux allèles du gène du récepteur des lipoprotéines de basse densité (LDLR).
    2. Utilisation d’un inhibiteur de la PCSK9 dans les 10 semaines précédant la visite de sélection.
    3. Traitement médical de fond modificateur des lipides qui n’est pas stable depuis au moins 4 semaines (6 semaines pour les fibrates, 24 semaines pour le mipomersen, 12 semaines pour la dose maximum tolérée de lomitapide) avant la visite de sélection.
    4. Plan d’aphérèse des lipoprotéines de basse densité (LDL)/paramètres d’aphérèse qui ne sont pas stables depuis au moins 8 semaines avant la visite de sélection ou plan/paramètres d’aphérèse qui ne devraient pas être stables au cours des 24 prochaines semaines.
    5. Utilisation de nutraceutiques ou de traitements en vente libre connus pour influer sur les lipides, à une dose/quantité qui n’est pas stable depuis au moins 4 semaines avant la visite de sélection ou entre la sélection et les visites de randomisation.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is the percent change in LDL-C from baseline to week 12 in the ITT population for alirocumab 150 mg Q2W as compared with placebo in patients with HoFH.
    The percent change in LDL-C from baseline to week 12 is defined as: 100 x (LDL-C value at week 12 -LDL-C value at baseline) LDL-C value at baseline


    For LDL-C analysis, both calculated and measured LDL-C values will be taken into account. In case both calculated and measured LDL-C values are available for the same sampling time point, the measured LDL-C will be considered. The baseline LDL-C value will be the last LDL-C value obtained before the first dose of double-blind-study drug. For randomized but not-treated patients, baseline will be defined as the last value before randomization. The LDL-C at week 12 will be the LDL-C value obtained within the week 12 analysis window, regardless of adherence to treatment (ITT estimand).
    All calculated and measured LDL-C values (scheduled or unscheduled, fasting or not fasting) may be used for the primary efficacy endpoint, if appropriate, according to the above definition. The analysis window used to allocate a time point to a measurement will be defined in the statistical analysis plan (SAP).
    Le critère d’évaluation principal de l’efficacité est la variation en procentuelle du LDL-C entre l’entrée dans l’étude et la Semaine 12 dans la population en intention de traiter (ITT) avec l’alirocumab 150 mg deux fois par semaine par rapport à un placebo chez des patients atteints de HFHo.
    La variation en procentuelle du LDL-C entre l’entrée dans l’étude et la Semaine 12 est définie de la manière suivante : 100 x (valeur LDL-C à la Semaine 12 - valeur LDL-C à l’entrée dans l’étude) Valeur LDL-C à l’entrée dans l’étude

    Pour l’analyse du LDL-C, les valeurs LDL-C calculées et mesurées seront toutes deux prises en compte. Si les valeurs LDL-C calculées et mesurées sont toutes deux disponibles pour le même moment d’échantillonnage, c’est la valeur LDL-C mesurée qui sera prise en compte. La valeur LDL-C à l’entrée dans l’étude sera la dernière valeur LDL-C obtenue avant la première dose du médicament de l’étude en double aveugle. Pour les patients randomisés mais non traités, la valeur à l’entrée dans l’étude sera définie comme la dernière valeur avant la randomisation. Le LDL-C à la Semaine 12 correspondra à la valeur LDL-C obtenue dans la fenêtre d’analyse de la Semaine 12, quelle que soit l’observance vis-à-vis du traitement (ITT estimée).
    Toutes les valeurs LDL-C calculées et mesurées (programmées ou non, à jeun ou non) pourront être utilisées pour le critère d’évaluation principal de l’efficacité, le cas échéant, conformément à la définition ci-dessus. La fenêtre d’analyse utilisée pour attribuer un moment à une mesure sera définie dans le plan d’analyse statistique (PAS).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Screening, Baseline, Week 12
    Sélection, Entrée dans l’étude, Semaine 12
    E.5.2Secondary end point(s)
    4.2.2.1. Key Secondary Efficacy Endpoints
    • The percent change in Apo B from baseline to week 12 (ITT estimand).
    • The percent change in non-HDL-C from baseline to week 12 (ITT estimand).
    • The percent change in total cholesterol from baseline to week 12 (ITT estimand).
    • Proportion of patients with ≥15% reduction in LDL-C at week 12 (ITT estimand).
    • Proportion of patients with ≥30% reduction in LDL-C at week 12 (ITT estimand).
    • The percent change in Lp(a) from baseline to week 12 (ITT estimand).
    • Proportion of patients with ≥50% reduction in LDL-C at week 12 (ITT estimand).
    • The percent change in HDL-C from baseline to week 12 (ITT estimand).
    • The percent change in fasting TG from baseline to week 12 (ITT estimand).
    • The percent change in Apo A-1 from baseline to week 12 (ITT estimand).
    4.2.2.1 Critères principaux d'efficacité secondaires
    • La variation procentuelle des Apo B entre l’entrée dans l’étude et la Semaine 12 (estimande de la population en intention de traiter [ITT]).
    • La variation procentuelle du non-HDL-C de l’entrée dans l’étude à la Semaine 12 (estimande de l’ITT).
    • La variation procentuelle du TC de l’entrée dans l’étude à la Semaine 12 (estimande de l’ITT).
    • La proportion de patients associés à ≥ 15 % de baisse du LDL-C en Semaine 12 (estimande de l’ITT).
    • La proportion de patients associés à ≥ 30 % de baisse du LDL-C en Semaine 12 (estimande de l’ITT).
    • La variation procentuelle des Lp(a) de l’entrée dans l’étude à la Semaine 12 (estimande de l’ITT).
    • La proportion de patients associés à ≥ 50 % de baisse du LDL-C en Semaine 12 (estimande de l’ITT).
    • La variation procentuelle de l’HDL-C de l’entrée dans l’étude à la Semaine 12 (estimande de l’ITT).
    • La variation procentuelle des TG à jeun de l’entrée dans l’étude à la Semaine 12 (estimande de l’ITT).
    • La variation procentuelle des Apo A-1 de l’entrée dans l’étude à la Semaine 12 (estimande de l’ITT).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Screening, Baseline, Week 12
    Sélection, Entrée dans l’étude, Semaine 12
    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 Yes
    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 trial2
    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 EEA20
    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
    Austria
    Belgium
    Bulgaria
    Canada
    Croatia
    Czech Republic
    Denmark
    Germany
    Greece
    Israel
    Italy
    Netherlands
    Poland
    Portugal
    Serbia
    Slovenia
    South Africa
    Spain
    Sweden
    Tunisia
    Turkey
    Ukraine
    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
    Dernière visite du dernier patient
    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 months0
    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 months0
    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 Yes
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 44
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 54
    F.4.2.2In the whole clinical trial 54
    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)
    There are no plans for treatment or care after the subject has ended
    the participation in the trial. Care will be determined and provided by
    subject's physician.
    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 Decision2017-07-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-07-06
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-02-13
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
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