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    Summary
    EudraCT Number:2016-003307-62
    Sponsor's Protocol Code Number:R1500-HTG-1522
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-12-17
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2016-003307-62
    A.3Full title of the trial
    A Phase 2, Randomized, Placebo-Controlled Study of Safety and Efficacy, Following Repeat-Dose Administration of Evinacumab (anti-ANGPTL3) in Patients with Severe Hypertriglyceridemia (sHTG) at Risk for Acute Pancreatitis
    Studio di fase 2, randomizzato, controllato con placebo sulla sicurezza e sull¿efficacia di evinacumab (anticorpo anti-ANGPTL3) dopo la somministrazione a dosi ripetute in pazienti affetti da ipertrigliceridemia grave (SHTG) a rischio di pancreatite acuta
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety and Efficacy Following Repeat-Dose of Evinacumab (Anti-ANGPTL3) in Patients With Severe Hypertriglyceridemia (sHTG) at Risk for Acute Pancreatitis
    Sicurezza ed efficacia dopo la somministrazione di dosi ripetute di evinacumab (anticorpo anti- ANGPTL3) in pazienti affetti da ipertrigliceridemia grave (SHTG) a rischio di pancreatite acuta
    A.3.2Name or abbreviated title of the trial where available
    -
    -
    A.4.1Sponsor's protocol code numberR1500-HTG-1522
    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, NY
    B.5.3.3Post code10591
    B.5.3.4CountryUnited States
    B.5.4Telephone number000000000
    B.5.5Fax number000000000
    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
    D.3.2Product code REGN1500
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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) Yes
    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 placeboConcentrate for solution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Severe Hypertriglyceridemia (sHTG)
    Ipertrigliceridemia grave (SHTG)
    E.1.1.1Medical condition in easily understood language
    Severe Hypertriglyceridemia (sHTG)
    Ipertrigliceridemia grave (SHTG)
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10020870
    E.1.2Term Hypertriglyceridemia
    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 determine the change in Triglycerides (TG) levels following 12 weeks of repeated Intravenous (IV) doses of evinacumab.
    L'obiettivo primario è determinare la variazione nei livelli di trigliceridi (TG) dopo 12 settimane di dosi endovenose (EV) ripetute di evinacumab.
    E.2.2Secondary objectives of the trial
    - To determine the percent change from baseline in TG levels following 2 to 24 weeks of repeated IV doses of evinacumab.
    - To assess changes in patient reported abdominal and gastrointestinal (GI) symptoms, dietary habits, and symptom/dietary impact measures.
    - To assess the degree of pancreatic injury/inflammation through 18F-2- Fluoro-2-Deoxy-D glucose positron emission tomography (18F-FDG-PET) imaging and diffusion weighted-magnetic resonance imaging (DW-MRI).
    - To evaluate the total evinacumab and total ANGPTL3 concentrations, and anti-drug antibody (ADA) during the evinacumab treatment and follow-up periods
    - To evaluate the safety and tolerability of evinacumab.
    -Determinare la variazione percentuale rispetto al basale nei livelli di TG dopo 2 24 settimane di dosi EV ripetute di evinacumab.
    -Valutare le variazioni nei sintomi addominali e gastrointestinali (GI), nelle abitudini alimentari e nelle misure dell' impatto di sintomi/alimentazione riferiti dal paziente.
    -Valutare il grado di lesione/infiammazione del pancreas mediante tomografia ad emissione di positroni con 18F-2-fluoro-2-desossi-D glucosio (18F-FDG-PET) e risonanza magnetica pesata in diffusione (DW-RM).
    - Valutare le concentrazioni totali di evinacumab e ANGPTL3 e gli anticorpi anti-farmaco (ADA) durante i periodi di trattamento con evinacumab e di follow-up.
    -Valutare la sicurezza e la tollerabilità di evinacumab.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1-Previous documentation in the patient's medical records of a fasting serum TG measurement = o > 1000 mg/dL (11.3 mmol/L) on more than 1 occasion, and all fasting TG values = o >500 mg/dL (5.6 mmol/L) at
    screening
    2._History of a hospitalization and diagnosis of acute pancreatitis in the past 10 years
    3_A stable lipid-modifying diet and/or medication (eg, statins, niacin, omega-3 fatty acids) for at least 4 weeks (6 weeks for fibrates, 8 weeks for PCSK9 inhibitors) prior to screening
    4_Body mass index (BMI) of 18-40 kg/m2

    Note: other protocol defined inclusion criteria may apply
    1_Precedente documentazione nella cartella clinica del paziente di una misurazione dei valori di TG sierici a digiuno = o >1000 mg/dl (11,3 mmol/l) in più di 1 occasione e tutti i valori di TG a digiuno = o >500 mg/dl (5,6 mmol/l) allo screening
    2._Anamnesi di ricovero e diagnosi di pancreatite acuta negli ultimi 10 anni.
    3_Una dieta e/o un farmaco (ad es. statine, niacina, acidi grassi omega-3) modificanti il profilo lipidico stabili per almeno 4 settimane (6 settimane per i fibrati, 8 settimane per gli inibitori di PCSK9) prima dello screening
    4_ Indice IMC di 18-40 kg/m2
    Nota: si possono applicare altri criteri di inclusione definiti nel protocollo
    E.4Principal exclusion criteria
    1_A hospital or clinic discharge diagnosis of acute pancreatitis within 12 weeks of screening
    2. Lipid apheresis or plasma exchange treatment within the last 4 weeks or plans to undergo apheresis or plasma exchange during the time frame of the study
    3_History of class 3/4 heart failure at any time in the past, or hospitalization for heart failure, diagnosis of a myocardial infarction, stroke, Transient ischemic attack (TIA), unstable angina, Coronary artery bypass surgery (CABG), Percutaneous coronary intervention (PCI), carotid surgery/ stenting within 3 months before the screening visit
    4_History of bleeding disorders, esophageal varices, heparin induced thrombocytopenia, or contraindications to receiving heparin (eg, allergic
    reaction to heparin)
    5_Previous treatment with Glybera® in the past 5 years or treatment with lomitapide or mipomersen in the past 6 months
    6. Pregnant or breast feeding women

    Note: Other protocol defined Inclusion/Exclusion criteria may apply
    1_Diagnosi alle dimissioni dalla clinica o dall’ospedale di pancreatite acuta entro 12 settimane dallo screening
    2_Trattamento di aferesi lipidica o ricambio plasmatico entro le ultime 4 settimane o trattamento pianificato di aferesi o ricambio plasmatico durante l’arco temporale dello studio
    3_Anamnesi di insufficienza cardiaca di classe 3/4 in qualsiasi momento in passato, o ricovero per insufficienza cardiaca, diagnosi di infarto miocardico, ictus, TIA, angina instabile, bypass aorto-coronarico (CABG), angioplastica (PCI), intervento/stenting della carotide nei 3 mesi precedenti la visita di screening
    4_Anamnesi di disturbi emorragici, varici esofagee, trombocitopenia indotta da eparina o controindicazioni a ricevere eparina (ad es. reazione allergica all’eparina)
    5_Precedente trattamento con Glybera® negli ultimi 5 anni o trattamento con lomitapide o mipomersen negli ultimi 6 mesi.
    6_Gravidanza o allattamento.
    Nota bene: si possono applicare altri criteri di inclusione/esclusione definiti dal protocollo.
    E.5 End points
    E.5.1Primary end point(s)
    Percent lowering of TG levels from baseline following 12 weeks of repeated IV doses of evinacumab.
    Riduzione percentuale rispetto al basale dei livelli di TG dopo 12 settimane di dosi EV ripetute di evinacumab.
    E.5.1.1Timepoint(s) of evaluation of this end point
    week 12
    settimana 12
    E.5.2Secondary end point(s)
    2. Percent TG lowering from baseline following 2 to 24 weeks of repeated IV doses of evinacumab
    3_Changes in patient reported abdominal and GI daily symptom questionnaire.
    4_Changes in patient reported daily dietary habits and impact questionnaire.
    5_Degree of pancreatic injury/inflammation through 18F-2-Fluoro-2-Deoxy-D glucose positron emission tomography (18F-FDG-PET) imaging at baseline as assessed by 18F-FDG standardized uptake values SUVmax and SUVmean.
    6_Change from baseline to degree of pancreatic injury/inflammation through 18F-FDG-PET imaging following 12 weeks of treatment with evinacumab as assessed by 18F-FDG standardized uptake values
    SUVmax and SUVmean.
    7_Degree of pancreatic injury/inflammation through Diffusion Weighted-Magnetic Resonance Imaging (DW-MRI) at baseline as assessed by ADC.
    8_Change from baseline to degree of pancreatic injury/inflammation through DW-MRI following 12 weeks of treatment with evinacumab as assessed by ADC.
    9_Change from baseline to degree of pancreatic injury/inflammation through DW-MRI following 24 weeks of treatment with evinacumab as assessed by ADC.
    10_Total evinacumab concentration in serum.
    11_Total ANGPTL3 concentrations.
    12_Incidence of anti-drug antibody (ADA)
    13_Incidence of Treatment-emergent adverse events (TEAEs)
    14_Incidence of serious adverse events (SAEs).
    15_Incidence of laboratory abnormalities.
    2_Riduzione percentuale rispetto al basale dei livelli di TG dopo 2-24 settimane di dosi EV ripetute di evinacumab.
    3_Variazioni nel questionario sui sintomi addominali e GI giornalieri riferiti dal paziente.
    4_Variazioni nel questionario sulle abitudini alimentari giornaliere e sull¿impatto riferiti dal paziente.
    5_Grado di lesione/infiammazione del pancreas valutato mediante tomografia ad emissione di positroni con 18F-2-fluoro-2-desossi-D glucosio (18F-FDG-PET) al basale, come misurato in base ai valori di captazione standardizzati (SUV) del 18F FDG SUVmax e SUVmedio.
    6_Variazione rispetto al basale nel grado di lesione/infiammazione del pancreas valutato mediante 18F-FDG-PET dopo 12 settimane di trattamento con evinacumab, come misurata in base ai valori di captazione standardizzati del 18F-FDG SUVmax e SUVmedio.
    7_Grado di lesione/infiammazione del pancreas valutato mediante risonanza magnetica pesata in diffusione (DW-RM) al basale, come misurato in base al coefficiente di diffusione apparente (ADC).
    8_Variazione rispetto al basale nel grado di lesione/infiammazione del pancreas valutato mediante DW-RM dopo 12 settimane di trattamento con evinacumab, come misurata in base all¿ADC.
    9_Variazione rispetto al basale nel grado di lesione/infiammazione del pancreas valutato mediante DW-RM dopo 24 settimane di trattamento con evinacumab, come misurata in base all¿ADC.
    10_Concentrazione totale di evinacumab nel siero.
    11_Concentrazioni totali di ANGPTL3.
    12_Incidenza di anticorpi anti-farmaco (ADA).
    13_Incidenza di eventi avversi emergenti dal trattamento (TEAE).
    14_Incidenza di eventi avversi seri (SAE).
    15_Incidenza di anomalie di laboratorio.
    E.5.2.1Timepoint(s) of evaluation of this end point
    These will be assessed at various timepoints detailed in the protocol:
    2. Up to 24 Weeks
    3. Up to 48 Weeks.
    4. Up to 48 Weeks.
    5. Baseline.
    6. Week 12.
    7. Baseline.
    8. Week 12.
    9. Week 24.
    10. Up to 48 Weeks.
    11. Up to 48 Weeks.
    12. Up to 48 Weeks.
    13. Up to 48 Weeks.
    14. Up to 48 Weeks.
    15. Up to 48 Weeks.
    Le valutazioni avverranno in corrispondenza di time point diversi specificati nel protocollo:
    2. Fino a 24 settimane
    3. Fino a 48 settimane
    4. Fino a 48 settimane
    5. Basale
    6. Settimana 12
    7. Basale
    8. Settimana 12
    9. Settimana 24
    10. Fino a 48 settimane
    11. Fino a 48 settimane
    12. Fino a 48 settimane
    13. Fino a 48 settimane
    14. Fino a 48 settimane
    15. fino a 48 settimane
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA7
    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 Information not present in EudraCT
    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
    Italy
    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
    LVLS
    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 months1
    E.8.9.1In the Member State concerned days27
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months1
    E.8.9.2In all countries concerned by the trial days27
    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: 35
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 15
    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 18
    F.4.2.2In the whole clinical trial 57
    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
    Nessuno
    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 Decision2018-05-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-04-12
    P. End of Trial
    P.End of Trial StatusCompleted
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