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    The EU Clinical Trials Register currently displays   42564   clinical trials with a EudraCT protocol, of which   7007   are clinical trials conducted with subjects less than 18 years old.
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    Summary
    EudraCT Number:2011-004308-39
    Sponsor's Protocol Code Number:R727-CL-1018
    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:2012-02-10
    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 number2011-004308-39
    A.3Full title of the trial
    A Phase 2 Pilot Study with a Randomized Double-Blind Treatment Phase to Evaluate the Pharmacodynamics and Safety of REGN727 in Patients with Autosomal Dominant Hypercholesterolemia and Gain-of-Function Mutations in 1 or Both Alleles of the PCSK9 Gene
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to determine if REGN727 will have an effect of circulating lipids in patients with gain-of-function mutations in their PCSK9 gene
    A.4.1Sponsor's protocol code numberR727-CL-1018
    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 organisationsanofi-aventis France
    B.5.2Functional name of contact pointDirection des opérations Cliniques
    B.5.3 Address:
    B.5.3.1Street Address9 Bd Romain Rolland
    B.5.3.2Town/ cityParis cedex 14
    B.5.3.3Post code75159
    B.5.3.4CountryFrance
    B.5.4Telephone number0 800 222 555
    B.5.6E-mailPublic-Registry-MA-France@sanofi.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.3.2Product code REGN727/SAR236553
    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.3.9.1CAS number 1245916-14-6
    D.3.9.2Current sponsor codeREGN727/SAR236553
    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 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
    Hypercholesterolemia
    E.1.1.1Medical condition in easily understood language
    High blood cholesterol level
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the effect of REGN727 on serum Low-density lipoprotein cholesterol in patients with autosomal domianant hypersholesterolemia and gain-of-function mutations in the PCSK9 gene.
    E.2.2Secondary objectives of the trial
    - The safety and tolerability of subcutaneously administered REGN727
    - The effect of REGN727 on other serum lipids and apolipoproteins
    - The PK profile of REGN727
    - The immunogenicity of REGN727
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Man or woman between the ages of 18 and 70 years, inclusive
    - A history of molecularly confirmed PCSK9 GOFm
    - Plasma LDLc levels ≥70 mg/dL (x 0.0259 mmol/L) and considered to be not at goal by the investigator
    - Body mass index ≥18.0 and ≤40.0 kg/m2 at the screening visit (visit 1 [day -28 to 15])
    - Systolic Blood Pressure ≤150 mm Hg and diastolic BP ≤95 mm Hg
    - Willing to refrain from the consumption of no more than 2 standard alcoholic drinks in any 24-hour period for the duration of the study. A standard alcoholic drink is the equivalent of 12 ounces beer, 5 ounces of wine, or 1.5 ounces of hard liquor
    - Willing to refrain from the consumption of alcohol for 24 hours before each study visit
    - Willing to maintain their usual stable diet and exercise regimen throughout the study
    - Willing and able to comply with clinic visits and study-related procedures
    - Provide signed informed consent
    E.4Principal exclusion criteria
    - Serum triglycerides >350 mg/dL (x 0.01129 mmol/L) measured after an 8 to 12 hour fast
    - History of heart failure (New York Heart Association Class II-IV) within the 12 months before the screening visit
    - History of myocardial infarction, acute coronary syndrome, unstable angina pectoris, stroke, peripheral vascular disease, transient ischemic attack, or cardiac revascularication within the 6 months before the screening visit
    - History of uncontrolled, clinically significant cardiac dysrhythmias or clinically significant recent changes in electrocardiogram 6 months before the screening visit
    - History of undergoing LDL apheresis within 3 months before the screening visit
    - Uncontrolled diabetes mellitus with hemoglobin A1C (HbA1c) >8.5% at the screening visit
    - Thyroid stimulating hormone (TSH) >1.5 x upper limit of normal at the screening visit
    - Alanine aminotransferase or aspartate aminotransferase >2 x upper limit of normal at the screening visit
    - Creatine phosphokinase >3 x upper limit of normal at the screening visit
    - Known sensitivity to monoclonal antibody therapeutics
    - Participation in a clinical research study evaluating an investigational drug within 30 days, or at least 5 half-lives of the investigational drug, before the screening visit, whichever is longer
    - Known to be positive for human immunodeficiency virus, hepatitis B virus, or hepatitus C virus
    - History of malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases
    - Pregnant or breast-feeding women
    - Sexually active man or woman of childbearing potential who is unwilling to practice adequate contraception during the study
    - Any medical or psychiatric condition which, in the opinion of the investigator, would place the patient at risk, interfere with patient’s participation in the study or interfere with the interpretation of the study results

    E.5 End points
    E.5.1Primary end point(s)
    percent change in direct (ultracentrifuged) serum LDL C from day 1 (baseline) to day 15 for group A compared to group B.
    E.5.1.1Timepoint(s) of evaluation of this end point
    14 days
    E.5.2Secondary end point(s)
    1. The incidence and severity of TEAEs in patients treated with REGN727 reported between the first dose and the end-of-study visit
    2. Percent change in ApoB100 from day 1 (baseline) to day 15 for group A compared to group B
    3. Percent change in non-HDL-C from day 1 (baseline) to day 15 for group A compared to group B
    4. Percent change in total cholesterol from day 1 (baseline) to day 15 for group A compared to group B
    5. Percent change in ApoB100/ApoA1 ratio from day 1 (baseline) to day 15 for group A compared to group B
    6. Trough concentrations of total REGN727 following repeat SC dosing with REGN727 throughout the study
    7. Immunogenicity of repeat SC dosing with REGN727 throughout the study
    E.5.2.1Timepoint(s) of evaluation of this end point
    Items 2, 3, 4, 5 in section E.5.2: 14 days
    Items 1, 6, 7 in section E.5.2: 55 days
    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 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) 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 No
    E.8.5.1Number of sites anticipated in the EEA3
    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
    France
    United States
    E.8.7Trial has a data monitoring committee No
    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 years
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months6
    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: 11
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 6
    F.4.2.2In the whole clinical trial 12
    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 Decision2012-02-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-02-23
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-07-28
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