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    Summary
    EudraCT Number:2017-003170-13
    Sponsor's Protocol Code Number:R1500-CL-1719
    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:2018-10-26
    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 ConcernedFrance - ANSM
    A.2EudraCT number2017-003170-13
    A.3Full title of the trial
    An open-label study to evaluate the long-term safety and efficacy of evinacumab in patients with homozygous familial hypercholesterolemia
    Étude en ouvert visant à évaluer la sécurité d’emploi et l’efficacité à long terme de l’évinacumab 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
    Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
    Evaluer la sécurité d’emploi et l’efficacité à long terme de l’évinacumab chez des patients atteints d’hypercholestérolémie familiale homozygote
    A.4.1Sponsor's protocol code numberR1500-CL-1719
    A.7Trial is part of a Paediatric Investigation Plan Yes
    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, NY
    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 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 nameEvinacumab (REGN1500)
    D.3.2Product code REGN1500
    D.3.4Pharmaceutical form Powder and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNEvinacumab
    D.3.9.1CAS number 1446419-85-7
    D.3.9.2Current sponsor codeREGN1500
    D.3.9.3Other descriptive nameEVINACUMAB
    D.3.9.4EV Substance CodeSUB181899
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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.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 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Homozygous familial hypercholesterolemia
    E.1.1.1Medical condition in easily understood language
    Hypercholesterolemia, also called dyslipidemia, is the presence of high levels of cholesterol in the blood
    Hypercholesterolémie, également appelé dyslipidémie, est la présence de taux élevé de cholestérol dans le sang
    E.1.1.2Therapeutic area Body processes [G] - Metabolic Phenomena [G03]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10020604
    E.1.2Term Hypercholesterolemia
    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 of the study is to evaluate the long-term safety and tolerability of evinacumab in patients with HoFH.
    L’objectif principal de l’étude est d’évaluer la sécurité d’emploi et la tolérance à long terme de l’évinacumab chez des patients atteints d’hypercholestérolémie familiale homozygote
    E.2.2Secondary objectives of the trial
    The secondary objectives of the study are:
    - To evaluate the effect of evinacumab on lipid parameters (ie, LDL-C, Apo B, non-HDL-C, total cholesterol [TC], and TG) in patients with HoFH
    - To evaluate the potential development of anti-evinacumab antibodies
    Les objectifs secondaires de l’étude sont :

    • Évaluer l’effet d’évinacumab sur les paramètres lipidiques (c.-à-d. le cholestérol des lipoprotéines de faible densité [LDL-C], l’apolipoprotéine B [Apo B], le cholestérol des lipoprotéines de densité non élevée [non-HDL-C], le cholestérol total [CT] et les triglycérides [TG]) chez des patients atteints d’HFHo.

    • Évaluer le développement potentiel d’anticorps anti-évinacumab.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Genomics Sub-Study, Protocol R1500-CL-1719 Amendment 2A and 2B, Protocol Section 8.2.8.3.
    The purpose of the genomic analyses is to identify genomic associations with clinical or biomarker response, other clinical outcome measures and possible AEs. In addition, associations between genomic variants and prognosis or progression of hypercholesterolemia as well as related diseases may also be studied.
    E.3Principal inclusion criteria
    1. Male and female patients ≥12 years of age with HoFH. Patients aged ≥12 years old will be enrolled only in countries where permitted by the Regulatory Agency and Institutional Review Board (IRB) or Ethics Committee (EC).
    2. Diagnosis of functional HoFH by at least 1 of the following genetic or clinical criteria:
    a. Documented functional mutation or mutations in both LDLR alleles
    Note: patients who have null receptor mutations on both LDLR alleles, ie, double null, are eligible
    b. Presence of homozygous or compound heterozygous mutations in Apo B or PCSK9
    Note: patients who are double heterozygous, ie, mutations on different genes (eg, LDLR/PCSK9) and patients with homozygous LDLRAP1 mutations are eligible
    c. Untreated TC >500 mg/dL (12.93 mmol/L) and TG <300 mg/dL (3.39 mmol/L) AND both parents with documented TC >250 mg/dL (6.47 mmol) OR cutaneous or tendinous xanthoma before the age of 10 years
    3. For patients who have participated in a previous evinacumab or alirocumab study: completion of the study in which they participated.
    4. Willing and able to comply with clinic visits and study-related procedures.
    5. Provide signed informed consent.
    1. Patients et patientes âgés de ≥ 12 ans atteints d’HFH. Les patients âgés de ≥ 12 ans ne pourront s’inscrire que dans les pays autorisés par l’agence réglementaire et le comité de protection des personnes (CPP) ou le comité d’éthique (CE).
    2. Diagnostic de la HFH fonctionnelle conformément à au moins l’un des critères génétiques ou cliniques suivants :
    a. Mutation ou mutations fonctionnelles documentées dans les deux allèles du RLDL
    Remarque : les patients présentant des mutations de récepteurs nuls sur les deux allèles LDLR (c.-à-d. double nuls) sont éligibles.
    b. Présence de mutations hétérozygotes composées ou homozygotes dans Apo B ou PCSK9
    Remarque : les patients qui sont doubles hétérozygotes, c’est-à-dire présentent des mutations de différents gènes (par ex. LDLR/PCSK9) et les patients présentant des mutations homozygotes LDLRAP1 sont éligibles.
    c. CT non traité > 500 mg/dl (12,93 mmol/l) et TG < 300 mg/dl (3,39 mmol/l) ET les deux parents avec un CT documenté > 250 mg/dl (6,47 mmol/l) OU xanthome cutané ou tendineux avant l’âge de 10 ans
    3. Pour les patients ayant participé à une étude antérieure sur l’évinacumab ou l’alirocumab : l’étude à laquelle ils ont participé doit être terminée.
    4. Être disposé(e) et capable de se conformer aux visites à la clinique et aux procédures liées aux études.
    5. Fournir un consentement éclairé signé.
    E.4Principal exclusion criteria
    Exclusion Criteria for Evinacumab-Naïve Patients
    1. Concomitant medications and procedures that have not been stable prior to the baseline visit
    2. Any new condition or worsening of an 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.
    3. History of a MI, unstable angina leading to hospitalization, coronary artery bypass graft surgery, percutaneous coronary intervention, uncontrolled cardiac arrhythmia, carotid surgery or stenting, stroke, transient ischemic attack, valve replacement surgery, carotid revascularization, endovascular procedure or surgical intervention for peripheral vascular disease within 3 months prior to the baseline visit
    4. Presence of any clinically significant uncontrolled endocrine disease known to influence serum lipids or lipoproteins
    5. Newly diagnosed (within 3 months prior to screening visit diabetes mellitus or poorly controlled (HbA1c >9%) diabetes
    6. Use of systemic corticosteroids, unless used as replacement therapy for pituitary/adrenal disease with a stable regimen for at least 6 weeks prior to screening visit
    7. Use of estrogen or testosterone therapy unless the regimen has been stable 6 weeks prior to the screening visit and no plans to change the regimen during the study
    8. Systolic blood pressure >160 mmHg or diastolic blood pressure >100 mmHg at the screening visit
    9. History of cancer within the past 5 years, except for adequately treated basal cell skin cancer, squamous cell skin cancer, or in situ cervical cancer
    10. History of New York Heart Association (NYHA) Class IV heart failure within 12 months before screening
    11. Laboratory findings during the screening period (applies to patients undergoing Screening):
    • Alanine aminotransferase or aspartate aminotransferase >3 x upper limit of normal (ULN)(1 repeat lab is allowed)
    • CPK >3 x ULN (1 repeat lab is allowed)
    • Positive serum beta-human chorionic gonadotropin or urine pregnancy test in WOCBP
    • TSH >1.5 x ULN of the central laboratory (1 repeat lab is allowed) for patients not on thyroid replacement therapy
    • Positive test for hepatitis B surface antigen and/or hepatitis C antibody (associated with a positive HCV RNA polymerase chain reaction)
    • eGFR <30 mL/min/1.73 m2 (calculated by central lab)
    • Postmenopausal status will be confirmed by measurement of follicle-stimulating hormone (FSH)
    12. Member of the clinical site study team and/or his/her immediate family.
    13. Pregnant of breastfeeding women
    14. Women of child bearing potential (see protocol for more information)
    15. Sexually active men unwilling to use forms of medically acceptable methods of birth control (see protocol for more information)
    16. LDL-C level <40 mg/dL at screening visit
    17. Use of any active investigational drugs (except alirocumab) within 1 month or 5 half lives prior to the screening visit, whichever is longer.
    18. Age <12 years at the screening visit
    19. Tanner stage <2 at the screening visit

    Exclusion Criteria for Patients from a Previous Evinacumab Study
    1. Significant protocol deviation in the previous study based on the investigator’s judgment, such as non-compliance by the patient.
    2. Concomitant medications and procedures that have not been stable prior to the baseline visit.
    3. Adverse event leading to permanent discontinuation from previous study.
    4. Any new condition or worsening of an 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.
    5. Member of the clinical site study team and/or his/her immediate family.
    6. Pregnant of breastfeeding women
    7. Women of child bearing potential (see protocol for more information)
    8. Sexually active men unwilling to use forms of medically acceptable methods of birth control (see protocol for more information)
    9. Age <12 years at the screening visit
    10. Tanner stage <2 at the screening visit
    Critères d’exclusion pour les patients naïfs d’évinacumab
    1. Procédures et traitements concomitants qui n’étaient pas stables avant la visite initiale
    2. Toute pathologie nouvelle ou aggravation d’une pathologie existante qui, selon l’opinion de l’investigateur rendrait le patient inapte à l’inclusion ou pourrait empêcher le patient de participer ou de terminer l’étude.
    3. Antécédent d’un infarctus du myocarde, angine instable menant à l’hospitalisation, pontage aorto-coronarien, intervention coronaire percutanée, arythmie cardiaque non contrôlée, chirurgie de la carotide ou implantation d’endoprothèse vasculaire, accident vasculaire cérébral, accident ischémique transitoire, chirurgie de remplacement valvulaire, revascularisation de la carotide, intervention endovasculaire ou intervention chirurgicale pour une maladie vasculaire périphérique dans les 3 mois précédant la visite initiale
    4. Présence d’une maladie endocrine non contrôlée cliniquement significative connue pour influer sur les lipides sériques ou les lipoprotéines
    5. Diabète sucré nouvellement diagnostiqué (dans les 3 mois précédant la visite de sélection) ou diabète mal maîtrisé (HbA1c > 9 %)
    6. Utilisation de corticoïdes systémiques, sauf si utilisés comme traitement substitutif pour une maladie hypophysaire/surrénale avec un schéma posologique stable pendant au moins 6 semaines avant la visite de sélection
    7. Utilisation d’un traitement à base d’œstrogènes ou de testostérone à moins que le schéma posologique soit stable 6 semaines avant la visite de sélection et qu’aucun projet de modification du schéma posologique ne soit envisagé pendant l’étude
    8. Tension artérielle systolique > 160 mmHg ou tension artérielle diastolique > 100 mmHg lors de la visite de sélection
    9. Antécédent de cancer au cours des 5 dernières années, sauf le carcinome basocellulaire, le carcinome épidermoïde cutané ou le carcinome cervical in situ si ils sont traités convenablement
    10. Antécédent d’une insuffisance cardiaque de classe IV selon l’association de cardiologie de New York (NYHA) dans les 12 mois précédant la sélection
    11. Résultats de laboratoire au cours de la période de sélection (s’applique aux patients subissant la sélection) :
    • Alanine aminotransférase ou aspartate aminotransférase > à 3 fois la limite supérieure de la normale (LSN) (1 répétition du laboratoire est autorisée)
    • CPK > à 3 fois la LSN (1 répétition du laboratoire autorisée)
    • Test de grossesse à la gonadotrophine chorionique sérique humaine bêta-positive ou test de grossesse urinaire positif chez les femmes en âge d’avoir des enfants
    • TSH > à 1,5 fois la LSN du laboratoire central (1 répétition du laboratoire est autorisée) pour les patients qui ne suivent pas de traitement de remplacement de la thyroïde
    • Test positif pour l’antigène de surface de l’hépatite B et/ou un anticorps hépatite C (associé à une réaction en chaîne par polymérase positive de l’ARN du VHC)
    • GFRe < 30 ml/min/1,73 m2 (calculé par le laboratoire central)
    • Le statut de ménopausique sera confirmé par la mesure de l’hormone folliculostimulante (FSH)
    12. Membre de l’équipe d’étude du centre clinique et/ou de sa famille proche.
    13. Femmes enceintes ou qui allaitent
    14. Femmes en âge d’avoir des enfants (voir le protocole pour plus d’informations)
    15. Hommes sexuellement actifs non disposés à utiliser des méthodes de contraception médicalement acceptables (voir le protocole pour plus d’informations)
    16. Taux de LDL-C < 40 mg/dl lors de la visite de sélection
    17. Utilisation de tout médicament expérimental actif (à l’exception de l’alirocumab) dans le mois ou les cinq demi-vies précédant la visite de sélection, selon la période la plus longue.
    18. Âge < 12 ans à la visite de sélection
    19. Classification de Tanner < 2 à la visite de sélection

    Critères d’exclusion pour les patients issus d’une étude précédente de l’évinacumab
    1. Déviation significative au protocole dans l’étude précédente conformément au jugement de l’investigateur, telle qu’une non-conformité provenant du patient
    2. Interventions et traitements concomitants qui n’étaient pas stables avant la visite initiale.
    3. Événement indésirable conduisant à une interruption définitive de l’étude précédente.
    4. Toute pathologie nouvelle ou aggravation d’une pathologie existante qui, selon l’opinion de l’investigateur rendrait le patient inapte à l’inclusion ou pourrait empêcher le patient de participer ou de terminer l’étude.
    5. Membre de l’équipe d’étude du centre clinique et/ou de sa famille proche.
    6. Femmes enceintes ou qui allaitent
    7. Femmes en âge d’avoir des enfants (voir le protocole pour plus d’informations)
    8. Hommes sexuellement actifs non disposés à utiliser des méthodes de contraception médicalement acceptables (voir le protocole pour plus d’informations)
    9. Âge < 12 ans à la visite de sélection
    10. Classification de Tanner < 2 à la visite de sélection
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the incidence and severity of treatment-emergent adverse events (TEAEs) and other safety variables during the open-label treatment period.
    Le critère d’évaluation principal est l’incidence et la gravité des événements indésirables émergents du traitement (EIET), et d’autres variables de sécurité au cours de la période de traitement en ouvert.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Overall safety will be assessed by monitoring/evaluation of TEAEs, physical examinations, electrocardiograms (ECG), and clinical safety laboratory tests at pre-specified time points. The potential emergence of anti-evinacumab antibodies will also be evaluated.
    La sécurité d’emploi globale sera évaluée par la surveillance/évaluation des EIET, des examens physiques, des électrocardiogrammes (ECG) et des analyses biologiques de la sécurité d’emploi clinique à certaines échéances prédéfinies. L’apparition potentielle d’anticorps anti-évinacumab sera également évaluée.
    E.5.2Secondary end point(s)
    The secondary efficacy endpoints are:
    - The percent and absolute change in LDL-C over time
    - The percent and absolute change in Apo B over time
    - The percent and absolute change in non-HDL-C over time
    - The percent and absolute change in TC over time
    - The percent and absolute change in TGs over time
    Les critères d’efficacité secondaires sont :
    - le pourcentage et la variation absolue du C-LDL au fil du temps
    - le pourcentage et la variation absolue de l’Apo B au fil du temps
    - le pourcentage et la variation absolue du C-HDL au fil du temps
    - le pourcentage et la variation absolue du CT au fil du temps
    - le pourcentage et la variation absolue des TG au fil du temps
    E.5.2.1Timepoint(s) of evaluation of this end point
    Efficacy will be assessed by clinical laboratory evaluation of lipid levels at pre-specified time points throughout the study.
    L’efficacité sera estimée par évaluation biologique clinique des taux lipidiques
    à certaines échéances prédéfinies tout au long de l’étude.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis Yes
    E.6.2Prophylaxis No
    E.6.3Therapy No
    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 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 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
    Australia
    Austria
    Canada
    Czech Republic
    France
    Germany
    Greece
    Italy
    Japan
    Netherlands
    Norway
    South Africa
    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
    LSLV
    Dernière visite du dernier patient
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    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 years4
    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.1.1Number of subjects for this age range: 6
    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: 6
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 94
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 42
    F.4.2.2In the whole clinical trial 100
    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
    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 Decision2017-01-25
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-04-13
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