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    Summary
    EudraCT Number:2019-004394-10
    Sponsor's Protocol Code Number:ACT16248
    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:2019-12-09
    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 ConcernedGermany - BfArM
    A.2EudraCT number2019-004394-10
    A.3Full title of the trial
    A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Efficacy, Pharmacodynamics, and Pharmacokinetics of Lademirsen (SAR339375) for Subcutaneous Injection Administered Every Week in Patients with Alport Syndrome
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of lademirsen (SAR339375) in patients with Alport Syndrome
    A.3.2Name or abbreviated title of the trial where available
    HERA
    A.4.1Sponsor's protocol code numberACT16248
    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 SponsorGenzyme Corporation
    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 supportGenyme Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSanofi-Aventis Deutschland GmbH
    B.5.2Functional name of contact point
    B.5.3.4CountryGermany
    B.5.6E-mailmedinfo.de@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 Yes
    D.2.5.1Orphan drug designation numberEU/3/15/1451
    D.3 Description of the IMP
    D.3.1Product nameLademirsen
    D.3.2Product code SAR339375
    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.3.9.1CAS number 1885122-05-3
    D.3.9.2Current sponsor codeSAR339375
    D.3.9.3Other descriptive nameRG-012
    D.3.9.4EV Substance CodeSUB184101
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number110
    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
    Congenital, hereditary and neonatal diseases
    E.1.1.1Medical condition in easily understood language
    Alport's Syndrome
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10001843
    E.1.2Term Alport's syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    -To assess the efficacy of lademirsen (SAR339375) in reducing the decline in renal function
    -To assess the safety and tolerability of lademirsen (SAR339375) in subjects with Alport syndrome
    E.2.2Secondary objectives of the trial
    -To assess plasma pharmacokinetic (PK) parameters of the parent compound and its metabolites
    -To assess the potential formation of anti-drug antibodies (ADAs) following administration of lademirsen (SAR339375)
    -To assess the pharmacodynamic effect of lademirsen (SAR339375) on miR-21 and on changes in renal injury and function biomarkers.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Male or female
    - Confirmed diagnosis of Alport syndrome
    1.Clinical diagnosis (hematuria, family history, hearing loss, ocular change), AND
    2.Genetic confirmation of Alport Syndrome in the subject or the family member, OR
    3.Kidney biopsy showing glomerular basement membrane abnormalities (eg, significant thinning, thickening, irregularity or lucencies) consistent with Alport Syndrome.
    - Age 18-55 years old
    - eGFR > 35 ml/min/1.73m^2 and <90 mL/min/1.73m^2 (based on CKD-EPI) at screening
    - Renal Function Criteria (patients must meet at least one of the following CRITERIA A, B or C):
    - A)Decline in eGFR of ≥4 mL/min/1.73 m^2/year (eGFR slope <= -4) based on a linear regression slope analysis of ≥4 eGFR measurements within 3 years prior to the study and with a minimum of 2-year time span (the last, of the screening measurement, and first eGFR measurements should be separated by at least 2 years). eGFR should be calculated by using either the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation.
    - B) proteinuria (UPCR) >2000 mg/g (UACR>1000 mg/g)
    - C) Age and sex adjusted eGFR (based on CKD-Epi; male 18-23 eGFR<90 mL/min/1.73m^2
    - ACE inhibitor and/or ARB, the dosing regimen should be stable for at least 30 days prior to screening
    - Sexually active female subjects of childbearing potential and sexually mature male subjects must agree to practice true abstinence in line with their preferred and usual lifestyle or to use two acceptable effective methods of contraception for the entire duration of the study and for at least 6 weeks after last dose.
    - Negative drug screen for opiates, cocaine, heroin, phencyclidine, amphetamines (including ecstasy), barbiturates, benzodiazepines, and cannabinoids. At the Investigator’s discretion, subjects prescribed benzodiazepines, cannabinoids, or opiates with positive results on a drug screen may be allowed.
    - Negative screening results for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, and human immunodeficiency virus (HIV) antibody
    - Normal biological tests
    - Able to understand all study procedures in the informed consent form (ICF) and willing to comply with all aspects of the protocol
    E.4Principal exclusion criteria
    - Causes of chronic kidney disease aside from Alport syndrome (including but not limited to other heritable disorders leading to chronic kidney disease, diabetic nephropathy, hypertensive nephropathy, lupus nephritis, IgA nephropathy)
    - ESRD as evidenced by ongoing dialysis therapy or history of renal transplantation
    - Any clinically significant illness within 30 days before screening or surgical or medical condition (other than Alport syndrome) that could interfere with the subject’s study compliance; confound the study results; impact subject safety; or significantly alter the absorption, distribution, metabolism, or excretion of drugs.
    - Weight > 110 kg
    - Any history of active malignancy within the last 1 year (history of localized basal cell or squamous cell carcinoma and cervical carcinoma in situ that has been excised/appropriately treated or a fully excised malignant lesion with a low probability of recurrence will not be considered exclusionary)
    - Prior treatment with Bardoxolone within 90 days prior to screening
    - History or presence of alcoholism or drug abuse within 2 years before screening or other concurrent social conditions that would potentially interfere with the subject’s study compliance, at the discretion of the Investigator
    - Participation in a recent investigational study and receipt of an investigational drug or investigational use of a licensed drug within 30 days or 5 half lives, whichever is longer, prior to screening
    - History or presence of hypersensitivity or idiosyncratic, allergic, or other clinically significant reaction to the study drug (including placebo), inactive ingredients, or related compounds (eg, other oligonucleotide products)
    - Any other condition or circumstance that, in the opinion of the Investigator, may make the subject unlikely to complete the study or comply with study procedures and requirements, or may pose a risk to the subject’s safety and well-being
    E.5 End points
    E.5.1Primary end point(s)
    1-Number of participants with treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
    2-Annualized change in estimated glomerular filtration rate eGFR from baseline to 48 weeks
    E.5.1.1Timepoint(s) of evaluation of this end point
    1-Baseline to maximum 106 weeks
    2-Baseline to 48 weeks
    E.5.2Secondary end point(s)
    1-Pharmacokinetics (PK) : Maximum concentration in plasma (Cmax):Plasma concentrations (Cmax) - Plasma concentrations (Cmax) of lademirsen (SAR339375), RG0005, and sum of lademirsen (SAR339375) and RG0005 (SUM)
    2-Pharmacokinetics (PK) : Trough plasma concentration (Ctrough):Plasma concentrations (Ctrough) - Plasma concentrations (Ctrough) of sum of lademirsen (SAR339375) and RG0005 (SUM)
    3-Number of participants with anti-drug antibodies (ADAs) incidents and titer
    4-Number of participant with adverse events associated to ADA:Association of ADAs with adverse events per participants will be collected
    5-Percent change in eGFR values from baseline to 24 weeks and 48 weeks
    6-Proportion of subjects who reach end staged renal disease (ESRD):Proportion of participants who reach ESRD as defined by an eGFR ≤15 mL/min/1.73 m^2 or initiation of hemodialysis or renal transplantation from baseline to 48 weeks
    7-Change in circulating miR-21 - Change of circulating miR-21 from baseline to 24 weeks, 48 weeks and 96 weeks
    8-Change in renal injury and function biomarkers from baseline - Change of biomarkers from baseline to 24 weeks, 48 weeks and 96 weeks
    E.5.2.1Timepoint(s) of evaluation of this end point
    1-2: 24 weeks and 48 weeks
    3-4: Baseline to maximum 106 weeks
    5: Baseline to 24 weeks and 48 weeks
    6: Baseline to 48 weeks
    7: Weeks 24, 48, and 96
    8: Baseline to Weeks 24, 48, and 96
    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 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 Yes
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA11
    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
    China
    France
    Germany
    Spain
    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 years3
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days6
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months7
    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: 45
    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 state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 25
    F.4.2.2In the whole clinical trial 45
    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 Decision2020-03-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-06-30
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-09-22
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