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    Summary
    EudraCT Number:2019-000337-39
    Sponsor's Protocol Code Number:4658-102-OLE
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2021-02-01
    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 number2019-000337-39
    A.3Full title of the trial
    An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients with Duchenne Muscular Dystrophy Who Have Completed Study 4658-102
    Studio in aperto sulla sicurezza, tollerabilità ed efficacia di eteplirsen in pazienti con distrofia muscolare di Duchenne che hanno completato lo studio 4658-102
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients who have completed study 4658-102
    Uno studio di ricerca di un nuovo farmaco sperimentale per il trattamento dei pazienti con Distrofia Muscolare di Duchenne che hanno completato lo studio 4658-102
    A.3.2Name or abbreviated title of the trial where available
    Not applicable
    Non applicabile
    A.4.1Sponsor's protocol code number4658-102-OLE
    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 SponsorSAREPTA THERAPEUTICS, 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 supportSarepta Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationVoisin Consulting
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address64 avenue Pierre Grenier
    B.5.3.2Town/ cityBoulogne-Billancourt
    B.5.3.3Post code92100
    B.5.3.4CountryFrance
    B.5.6E-mailclinicaltrialinformation@voisinconsulting.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/3/08/586
    D.3 Description of the IMP
    D.3.1Product nameEteplirsen
    D.3.2Product code [AVI-4658]
    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 INNETEPLIRSEN
    D.3.9.1CAS number 1173755-55-9
    D.3.9.2Current sponsor codeAVI-4658
    D.3.9.4EV Substance CodeSUB129287
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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 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) 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
    Distrofia muscolare di Duchenne sensibile al salto dell’esone 51
    E.1.1.1Medical condition in easily understood language
    Duchenne Muscular Dystrophy
    Distrofia muscolare di Duchenne
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10013801
    E.1.2Term Duchenne muscular dystrophy
    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 evaluate the ongoing safety and tolerability of additional treatment with eteplirsen administered once weekly by intravenous (IV) infusion in male Duchenne muscular dystrophy (DMD) patients who have successfully completed the 96-week eteplirsen study: Study 4658-102.
    Valutare la sicurezza e la tollerabilità continue del trattamento aggiuntivo con eteplirsen somministrato una volta alla settimana tramite infusione endovenosa (EV) in pazienti maschi affetti da distrofia muscolare di Duchenne (DMD) che hanno completato con successo lo studio su eteplirsen della durata di 96 settimane: studio 4658-102.
    E.2.2Secondary objectives of the trial
    To evaluate the efficacy of additional treatment with eteplirsen administered once weekly by IV infusion in male DMD patients who have successfully completed Study 4658-102
    Valutare l'efficacia del trattamento aggiuntivo con eteplirsen somministrato una volta alla settimana tramite infusione EV in pazienti maschi affetti da DMD che hanno completato con successo lo studio 4658-102
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patient has successfully completed 96 weeks of treatment in Study 4658-102.
    2. Parent(s) or legal guardian(s) who is/are able to understand and comply with the study requirements.
    3. Patient and/or their parent(s)/legal guardian(s) are willing and able to provide signed informed consent.
    1. Corretto completamento delle 96 settimane di trattamento dello studio 4658-102.
    2. Genitori o tutori legali in grado di comprendere e rispettare i requisiti dello studio.
    3. Disponibilità e capacità dei pazienti e/o dei genitori/tutori legali di fornire il consenso informato firmato.
    E.4Principal exclusion criteria
    1. Patient has a prior or ongoing medical condition that, in the Investigator’s opinion, could adversely affect the safety of the patient, or make it unlikely that the course of treatment or follow-up would be completed, or impair the assessment of study results.
    1. Patologie pregresse o in corso che potrebbero, a giudizio dello sperimentatore, influire negativamente sulla sicurezza del paziente, impedire il completamento del trattamento o del follow-up o compromettere la valutazione dei risultati dello studio.
    E.5 End points
    E.5.1Primary end point(s)
    - Incidence of AEs, SAEs, and discontinuation from treatment due to AEs
    - Incidence of deaths due to AEs
    - Incidence of adverse events of special interest, including infusion-related reactions, hypersensitivity, and renal events
    - Clinically significant laboratory testing, including hematology, coagulation, serum chemistry, and urinalysis
    - Clinically significant cardiac function assessments, including electrocardiogram (ECG)
    - Clinically significant vital signs
    - Clinically significant physical examinations
    • Frequenza e gravità degli AE, eventi avversi seri (SAE) e interruzione del trattamento dovuta ad AE
    • Decessi dovuti ad AE
    • Eventi avversi di speciale interesse, tra cui reazioni correlate all'infusione, ipersensibilità ed eventi renali
    • Analisi di laboratorio, tra cui esami ematologici, della coagulazione, del siero e analisi delle urine
    • Valutazioni della funzione cardiaca, tra cui elettrocardiogramma (ECG)
    • Segni vitali
    • Esame obiettivo
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline to end of study
    Baseline alla fine dello studio
    E.5.2Secondary end point(s)
    The efficacy of eteplirsen will be assessed by the change from Baseline to the end of Week 144, Week 192, and Week 284 in the NSAA score.
    L'efficacia di eteplirsen sarà stimata valutando la variazione dei punteggi NSAA dal basale alla fine della settimana 144, settimana 192 e settimana 284.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline to the end of Week 144, Week 192, and Week 284
    Baseline alla fine Settimane 144, Settimane 192, e Settimane 284
    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 No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    Tolerability
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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 EEA4
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    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 years6
    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 Yes
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 15
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 15
    F.4.2.2In the whole clinical trial 15
    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)
    Patients may transition to commercially available product if commercial eteplirsen is available at any time during the study
    I pazienti possono passare al prodotto disponibile in commercio se eteplirsen è immesso sul mercato in qualsiasi momento durante lo studio
    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 Decision2019-06-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-05-21
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2022-08-31
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