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    Summary
    EudraCT Number:2015-003772-74
    Sponsor's Protocol Code Number:MDCO-PCS-15-01
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-10-23
    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 ConcernedGermany - BfArM
    A.2EudraCT number2015-003772-74
    A.3Full title of the trial
    A placebo-controlled, double-blind, randomized trial to compare the effect of different doses of ALN-PCSSC given as single or multiple subcutaneous injections in subjects with high cardiovascular risk and elevated LDL-C.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to evaluate the effect of ALN-PCSSC treatment on low-density lipoprotein cholesterol levels
    A.4.1Sponsor's protocol code numberMDCO-PCS-15-01
    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 SponsorThe Medicines Company
    B.1.3.4CountrySwitzerland
    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 supportThe Medicines Company
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationThe Medicines Company
    B.5.2Functional name of contact pointGlobal Health Science Center
    B.5.3 Address:
    B.5.3.1Street Address8 sylvan Way
    B.5.3.2Town/ cityParsippany
    B.5.3.3Post codeNJ 07054
    B.5.3.4CountryUnited States
    B.5.4Telephone number+19732906000
    B.5.6E-mailmedical.information@themedco.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 nameALN-PCSSC
    D.3.2Product code ALN-PCSSC
    D.3.4Pharmaceutical form Solution for injection
    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 INNALN-60212
    D.3.9.2Current sponsor codeALN-60212
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    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
    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
    Hypercholesterolemia, specifically, elevated low density lipoprotein cholesterol (LDL-C), is one of the major risk factors for the development of coronary heart disease (CHD)
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    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 No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the effect of ALN-PCSSC treatment on LDL-C levels at Day 180.
    E.2.2Secondary objectives of the trial
    To evaluate the effect of ALN-PCSSC on the following:
    •LDL-C levels at other time points
    •PCSK9 levels over time
    •other lipids, lipoproteins, apolipoproteins
    •the proportion of subjects achieving different global lipid guidelines
    •individual responsiveness to different doses
    •duration of lipid-lowering effect of different doses
    •the safety and tolerability profile of ALN-PCSSC
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female subjects 18 years of age or older, and of any race.
    2.History of ASCVD or ASCVD-risk equivalents (symptomatic atherosclerosis, Type 2 diabetes, familial hypercholesterolemia, including subjects whose 10-year risk of a cardiovascular [CV] event assessed by Framingham Risk Score or equivalent has a target LDL-C of < 100mg/dL) and receiving maximum-tolerated lipid-lowering therapy
    3. Serum LDL-C ≥1.8 mmol/L (≥70 mg/dL) for ASCVD subjects or ≥2.6 mmol/L (≥100 mg/dL) for ASCVD-risk equivalent subjects at screening
    4. Fasting triglyceride <4.52 mmol/L (<400 mg/dL) at screening
    5. Calculated glomerular filtration rate 30 mL/min or higher by estimated glomerular filtration rate (eGFR) using standardized local clinical methodology.
    6. Subjects on statins should be receiving the maximally tolerated dose
    7. Subjects on lipid-lowering therapies (such as statins and/or ezetimibe) should be on a stable dose for ≥30 days before screening with no planned medication or dose change during study participation.
    8. Willing and able to give written and informed consent before initiation of any study-related procedures and willing to comply with all required study procedures.
    E.4Principal exclusion criteria
    1. Any uncontrolled or serious disease, or any medical or surgical condition, that may either interfere with participation in the clinical study, and/or put the subject at significant risk (according to Investigator’s [or delegate] judgment) if he/she participates in the clinical study
    2. An underlying known disease, or surgical, physical, or medical condition that, in the opinion of the Investigator (or delegate) might interfere with interpretation of the clinical study results
    3. New York Heart Failure Association (NYHA) class II, III or IV heart failure or last known left ventricular ejection fraction <30%.
    4. Cardiac arrhythmia within 3 months prior to randomization that is not controlled by medication or via ablation.
    5. Any history of hemorrhagic stroke.
    6. Major adverse cardiac event (MACE) within 6 months prior to randomization.
    7. Uncontrolled severe hypertension: systolic blood pressure >180 mmHg or diastolic blood pressure >110 mmHg prior to randomization despite anti-hypertensive therapy.
    8. Poorly controlled Type II diabetes, ie, HbA1c >10.0% prior to randomization.
    9. Active liver disease defined as any known current infectious, neoplastic, or metabolic pathology of the liver or unexplained alanine aminotransferase (ALT), aspartate aminotransferase (AST), elevation > 2x the upper limit of normal (ULN), or total bilirubin elevation > 1.5x ULN at screening confirmed by a repeat measurement at least one week apart.
    10. Serious comorbid disease in which the life expectancy of the subject is shorter than the duration of the trial (eg, acute systemic infection, cancer, or other serious illnesses). This includes all cancers with the exception of treated basal-cell carcinoma occurring >5 years before screening.
    11. Females who are pregnant or nursing, or who are of childbearing potential and unwilling to use at least two methods of contraception (oral contraceptives, barrier methods, approved contraceptive implant, long- term injectable contraception, intrauterine device or tubal litigation). Women who are >2 years postmenopausal defined as ≥1 year since last menstrual period AND if less than 55 years old with a negative pregnancy test within 24 hours of randomization or surgically sterile are exempt from this exclusion.
    12. Males who are unwilling to use an acceptable method of birth control during the entire study period (ie, condom with spermicide).
    13. Known history of alcohol and/or drug abuse.
    14. Treatment with other investigational medicinal products or devices within 30 days or five half lives, whichever is longer.
    15. Use of other investigational medicinal products or devices during the course of the study.
    16. Any condition that according to the investigator could interfere with the conduct of the study, such as but not limited to:
    a. Inappropriate for this study, including subjects who are unable to communicate or to cooperate with the investigator.
    b. Unable to understand the protocol requirements, instructions and study-related restrictions, the nature, scope, and possible consequences of the study (including subjects whose cooperation is doubtful due to drug abuse or alcohol dependency).
    c. Unlikely to comply with the protocol requirements, instructions and study-related restrictions (eg, uncooperative attitude, inability to return for follow-up visits, and improbability of completing the study).
    d. Have any medical or surgical condition, which in the opinion of the investigator would put the subject at increased risk from participating in the study.
    e. Involved with, or a relative of, someone directly involved in the conduct of the study.
    f. Any known cognitive impairment (eg, Alzheimer’s Disease)
    E.5 End points
    E.5.1Primary end point(s)
    Primary Endpoint: Percentage change in LDL-C from baseline to Day 180.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Included in previous section.
    E.5.2Secondary end point(s)
    Secondary Endpoints:
    • Percentage change in LDL-C from baseline to Days 14, 30, 60, 90, 120, 150, and 210
    • Proportion of subjects in each group with LDL-C greater than 80% of the baseline value at Day 180 and Day 210
    • Duration of time on treatment for subjects to return to 80% of baseline or greater LDL-C or PCSK9 protein (if not resolved by Day 210, subjects will continue to be followed in the OLE)
    • Individual responsiveness defined as the number of subjects reaching on treatment LDL-C levels of <30 mg/dL, <50 mg/dL, <70 mg/dL, and <100 mg/dL at Days 90, 120, and 180
    • Proportion of subjects in each group with greater or equal to 50% LDL-C reduction from baseline at Days 90, 120, and 180
    • Percentage change in PCSK9 levels from baseline to Days 14, 30, 60, 90, 120, 150, 180 and 210
    • Percentage change in other lipids, lipoproteins, apolipoproteins from baseline at each subsequent visit to Day 210
    • Proportion Proportion of subjects in each group with greater or equal to 50% LDL-C reduction from baseline at Days 90, 120, and 180
    E.5.2.1Timepoint(s) of evaluation of this end point
    Included in previous section.
    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 No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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 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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial5
    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 concerned15
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA40
    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
    Germany
    Netherlands
    United Kingdom
    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 years1
    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 years1
    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 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: 240
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 240
    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 state75
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 400
    F.4.2.2In the whole clinical trial 480
    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)
    Subjects completing the study to Day 210 will be given the opportunity to enroll in a separate long-term open-label extension (OLE) study. Any subject in whom LDL-C levels have not returned to >80% of baseline values, will continue to be followed as part of the OLE study.
    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 Decision2016-02-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-02-22
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-06-07
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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