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    The EU Clinical Trials Register currently displays   38003   clinical trials with a EudraCT protocol, of which   6235   are clinical trials conducted with subjects less than 18 years old.
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    Summary
    EudraCT Number:2016-003307-62
    Sponsor's Protocol Code Number:R1500-HTG-1522
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2018-02-23
    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 ConcernedUK - MHRA
    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
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2 study to evaluate safety and efficacy of Evinacumab in patients with severe hypertriglyceridemia at risk for acute pancreatitis.
    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.5.6E-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 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.3.9.2Current 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) 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)
    E.1.1.1Medical condition in easily understood language
    Severe Hypertriglyceridemia (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 of the study is to determine the change in TG levels following 12 weeks of repeated IV doses of evinacumab in the subset of patients with a documented history of sHTG (TG ≥1000 mg/dL), a TG level of at least 500 mg/dL (5.6 mmol/L) at screening, a history of acute pancreatitis, and without loss of function (LOF) mutations in genes in the lipoprotein lipase (LPL) pathway; and to assess whether a reduction in TG of at least 40% from the baseline placebo period has been achieved.
    E.2.2Secondary objectives of the trial
    To
    - Determine the % change from baseline in TG levels following 2 to 24 weeks of repeated IV doses of evinacumab overall and in subgroups with homozygous or compound heterozygous LOF mutations, heterozygous LOF mutations, and without LOF mutations in genes in the LPL pathway
    - Assess changes in patient reported abdominal and GI symptoms, dietary habits, and symptom/dietary impact measures
    - Aassess the degree of pancreatic injury/inflammation through 18F-FDG-PET imaging at baseline and change from baseline following 12 weeks of treatment with evinacumab as assessed by 18F-FDG standardized uptake values SUVmax and SUVmean
    - Assess the degree of pancreatic injury/inflammation through DW-MRI at baseline and the change from baseline following 12 and 24 weeks of treatment with evinacumab as assessed by ADC
    - Evaluate the total evinacumab and total ANGPTL3 concentrations, and ADA during the evinacumab treatment and follow-up periods
    - Evaluate the safety and tolerability of evinacumab
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Males and females ≥18 to 75 years of age at screening
    2. Previous documentation in the patient’s medical records of a fasting serum TG measurement ≥ 1000 mg/dL (11.3 mmol/L) on more than 1 occasion, and all fasting TG values >500 mg/dL (5.6 mmol/L) at screening
    3. History of a hospitalization and diagnosis of acute pancreatitis in the past 10 years.
    4. On a stable lipid-modifying diet with or without medications (eg, statins, niacin, omega-3 fatty acids). Lipid-modifying diet and doses of medications should be stable for at least 4 weeks (6 weeks for fibrates, 8 weeks for PCSK9 inhibitors) prior to screening
    5. BMI index of 18-40 kg/m2
    6. Provide signed informed consent
    7. Willing and able to comply with clinic visits and study-related procedures
    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, TIA, unstable angina, CABG, 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. New clinically significant findings on 12-lead electrocardiogram (ECG) that would place the patient at risk or interfere with participation in the study
    6. Women of childbearing potential (WOCBP)* who are unwilling to practice a highly effective birth control method prior to the initial dose, during the study, and for 24 weeks after the last dose of study drug.
    *Postmenopausal women must be amenorrheic for at least 12 months in order not to be considered of childbearing potential. Postmenopausal status will be confirmed by measurement of follicle-stimulating hormone (FSH). Pregnancy testing and contraception are not required for women with documented hysterectomy and/or oophorectomy.
    7. Men who are sexually active with WOCBP and are unwilling to consistently use condoms during the study drug treatment period and for 24 weeks after the last administration of study drug, regardless of vasectomy status. Sperm donation is prohibited during the study and for up to 24 weeks after the last administration of study drug.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the percent lowering of TG levels from baseline following 12 weeks of repeated IV doses of evinacumab in the subset of patients with a documented history of sHTG (TG ≥1000 mg/dL [11.3 mmol/L]), a TG level of at least 500 mg/dL (5.6 mmol/L) at screening, a history of acute pancreatitis, and without LOF mutations in genes in the LPL pathway. (For patients randomized to evinacumab, baseline is defined as the period prior to the double-blind period; for patients randomized to placebo, baseline is defined as the period prior to the single-blind period).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Following 12 weeks of repeated IV doses of evinacumab
    E.5.2Secondary end point(s)
    - Percent TG lowering from baseline following 2 to 24 weeks of repeated IV doses of evinacumab overall and in subgroups with homozygous or compound heterozygous LOF mutations, heterozygous LOF mutations, and without LOF mutations in genes in the LPL pathway
    - Changes in patient reported abdominal and GI symptoms, dietary habits, and symptom/dietary impact measures
    - Degree of pancreatic injury/inflammation through 18F-FDG-PET imaging at baseline (placebo run-in) and change from baseline following 12 weeks of treatment with evinacumab as assessed by 18F-FDG standardized uptake values SUVmax and SUVmean
    - Degree of pancreatic injury/inflammation through DW-MRI and T2-MRI at baseline (placebo run-in period) and the change from baseline following 12 and 24 weeks of treatment with evinacumab as assessed by ADC
    - The total evinacumab concentrations, total ANGPTL3 concentrations, and ADA during the evinacumab treatment and follow-up periods
    - Incidence and severity of treatment-emergent adverse event (TEAEs), SAEs, laboratory abnormalities, and other safety variables in patients treated with evinacumab
    E.5.2.1Timepoint(s) of evaluation of this end point
    These will be assessed at various timepoints detailed in the protocol
    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 Yes
    E.8.1.7.1Other trial design description
    Double blind treatment period (Day 1 to 85) followed by single blind treatment period (Day 86 to 169
    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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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
    Italy
    Poland
    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
    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 days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months8
    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: 50
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 8
    F.4.2.2In the whole clinical trial 50
    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 Decision2018-04-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-07-20
    P. End of Trial
    P.End of Trial StatusOngoing
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