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    Summary
    EudraCT Number:2016-000951-29
    Sponsor's Protocol Code Number:4658-102
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-02-07
    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 number2016-000951-29
    A.3Full title of the trial
    An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
    Studio in aperto sulla sicurezza, tollerabilità e farmacocinetica di eteplirsen in pazienti giovani con distrofia muscolare di Duchenne sensibile al salto dell’esone 51
    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 young Duchenne Muscular Dystrophy patients
    Studio di ricerca su un nuovo medicinale
    in fase di sperimentazione per il trattamento di pazienti giovani con distrofia muscolare di Duchenne
    A.3.2Name or abbreviated title of the trial where available
    N/A
    N/A
    A.4.1Sponsor's protocol code number4658-102
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/279/2016
    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 Address3 rue des Longs Prés
    B.5.3.2Town/ cityBoulogne-Billancourt
    B.5.3.3Post code92100
    B.5.3.4CountryFrance
    B.5.4Telephone number0033141318300
    B.5.5Fax number0033141318309
    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 Yes
    D.2.1.1.1Trade name Exondys 51
    D.2.1.1.2Name of the Marketing Authorisation holderSarepta Therapeutics, Inc.
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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 IMP
    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.3.9.3Other descriptive nameETEPLIRSEN
    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 safety and tolerability of eteplirsen administered once weekly by intravenous (IV) infusion in male Duchenne muscular dystrophy (DMD) patients ages 6 months to 48 months, inclusive
    Valutare la sicurezza e la tollerabilità di eteplirsen somministrato una volta alla settimana mediante infusione endovenosa (e.v.) in pazienti maschi con distrofia muscolare di Duchenne (DMD) da 6 mesi a 48 mesi di età compresi
    E.2.2Secondary objectives of the trial
    To determine the pharmacokinetics (PK) of eteplirsen at the 2-, 10-, 20- and 30-mg/kg dose levels, administered once weekly by IV infusion in male DMD patients ages 6 months to 48 months, inclusive
    • Determinare la farmacocinetica (PK) di eteplirsen a livelli di dose di 2, 10, 20 e 30 mg/kg, somministrato una volta alla settimana mediante infusione e.v. in pazienti maschi con DMD da 6 mesi a 48 mesi di età compresi
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Be a male between 6 months to 48 months of age, inclusive.
    2. Have an established clinical diagnosis of DMD with a deletion mutation amenable to exon 51 skipping (e.g., deletions of exons 45-50, 47-50, 48-50, 49-50, 50, 52, 52-63).
    3. Have a parent(s) or legal guardian(s) who is able to understand and comply with the study requirements and is willing to provide written informed consent for the patient to participate in the study.
    1.Essere un maschio tra i 6 mesi e i 48 mesi d'età compresi.
    2. Avere una diagnosi clinica accertata di DMD con una mutazione per delezione sensibile al salto dell’esone 51 (ad es. delezioni degli esoni 45 50, 47 50, 48 50, 49 50, 50, 52, 52 63).
    3. Avere un genitore(i) o tutore(i) legale(i) in grado di comprendere e rispettare i requisiti dello studio e disposto(i) a fornire il consenso informato scritto per la partecipazione del paziente allo studio.
    E.4Principal exclusion criteria
    1. Has received any pharmacologic treatment that might have an effect on muscle strength or function within 12 weeks prior to dosing at Week 1 (e.g., growth hormone, anabolic steroids).
    2. Has received previous or current treatment with any experimental treatment. Prior drisapersen therapy is permitted if a patient has not received drisapersen for 6 months prior to the Week 1 dose.
    3. Has a clinically significant illness other than DMD, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, behavioural disease, or malignancy likely to impair the patient's ability to participate in this study.
    4. Has a clinically significant laboratory abnormality that is either not expected or is of a greater severity than what is expected in DMD patients.
    5. Has any other condition that, in the Investigator's opinion, could interfere with the patient's participation in the study.
    1. Ha ricevuto qualsiasi trattamento farmacologico che potrebbe avere un effetto sulla forza o sulla funzione muscolare nelle 12 settimane precedenti la somministrazione alla Settimana 1 (ad es. ormone della crescita, steroidi anabolizzanti).
    2. È stato sottoposto in precedenza o è attualmente sottoposto a un trattamento sperimentale. La precedente terapia con drisapersen è consentita se un paziente non ha ricevuto drisapersen per 6 mesi prima della dose della Settimana 1.
    3. È affetto da una malattia clinicamente significativa diversa dalla DMD, incluse malattia cardiaca, polmonare, epatica, renale, ematologica, immunologica, comportamentale, o tumore maligno che potrebbe compromettere la capacità del paziente di partecipare a questo studio.
    4. Presenta un'anomalia di laboratorio clinicamente significativa, non prevista o di severità maggiore di quella prevista nei pazienti con DMD.
    5. Presenta qualsiasi altra condizione clinica che, a parere dello Sperimentatore, potrebbe interferire con la partecipazione del paziente allo studio.
    E.5 End points
    E.5.1Primary end point(s)
    Safety and tolerability of eteplirsen as measured by:
    • Incidence of adverse events (AEs)
    • Abnormal changes from baseline or clinically significant worsening of clinical safety laboratory abnormalities (hematology, chemistry, coagulation, and urinalysis)
    • Abnormal changes from baseline or worsening of vital signs
    • Abnormal changes from baseline or worsening of physical examination findings
    • Abnormal changes from baseline or clinically significant worsening of ECGs and ECHOs
    L’endpoint primario è costituito dalla sicurezza e tollerabilità di eteplirsen in questa popolazione di studio, misurate da:
    • Incidenza di AE
    • Variazioni anomale rispetto al basale o peggioramento clinicamente significativo di anomalie cliniche di laboratorio per la sicurezza (parametri ematologici, parametri chimici, coagulazione e analisi delle urine)
    • Variazioni anomale rispetto al basale o peggioramento dei parametri vitali
    • Variazioni anomale rispetto al basale o peggioramento dei risultati dell'esame obiettivo
    • Variazioni anomale rispetto al basale o peggioramento clinicamente significativo di ECG ed ECHO
    E.5.1.1Timepoint(s) of evaluation of this end point
    Incidence of AEs: continuous
    Clinical safety laboratory sample: Screening, Weeks 2, 4, 8, 12, 24, 36, 48, 60, 72, 84, 96 and End-of-Study Follow-up Visit
    Vital signs: Screening, baseline, on weekly infusion days, Week 96 and End-of-Study Follow-up Visit
    Physical examination: Screening, baseline, Week 4, 8, 12, 16, 20-48, 60, 72, 84, 96 and End-of-Study Follow-up Visit
    ECG: Screening, Weeks 8, 12, 24, 48, 60, 72, 84, 96
    ECHO: Screening, Weeks 24, 48, 72, 96
    Incidenza di AE: continuo
    campione di laboratorio Sicurezza clinica: Screening, settimane 2, 4, 8, 12, 24, 36, 48, 60, 72, 84, 96 e visita di follow-up di fine studio
    Parametri vitali: Screening, baseline, nei giorni di infusione settimanali, settimane 96 e visita di follow-up di fine studio
    Esame obiettivo: Screening, baseline, Settimane 4, 8, 12, 16, 20-48, 60, 72, 84, 96 e visita di follow-up di fine studio
    ECG: Screening, Settimane 8, 12, 24, 48, 60, 72, 84, 96
    ECHO: Screening, Settimane 24, 48, 72, 96
    E.5.2Secondary end point(s)
    PK of eteplirsen by population PK methods, including assessment of the following PK parameters (if evaluable):
    • Maximum plasma concentration (Cmax)
    • Time of Cmax (Tmax)
    • AUC
    • Apparent volume of distribution at steady state (Vss)
    • Clearance (CL)
    • Elimination half-life (t½)
    • Amount of drug eliminated in urine (Ae%)
    Determinare la PK di eteplirsen mediante metodi di farmacocinetica di popolazione, inclusa la valutazione dei seguenti parametri farmacocinetici (se valutabili):
    • Concentrazione plasmatica massima (Cmax)
    • Tempo della Cmax (Tmax)
    • Area sotto la curva concentrazione/tempo (AUC)
    • Volume di distribuzione apparente allo steady state (Vss)
    • Clearance (CL)
    • Emivita di eliminazione (t½)
    • Quantità di farmaco eliminata nelle urine (Ae%)
    E.5.2.1Timepoint(s) of evaluation of this end point
    PK plasma and urine sampling: Weeks 2, 6, 8, 10, 24
    PK plasma e il campionamento delle urine: settimane 2, 6, 8, 10, 24
    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 No
    E.6.6Pharmacokinetic Yes
    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
    Tollerabilità
    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 Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    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.8.3 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 EEA5
    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
    The trial will be considered to have been completed upon the last visit of the last patient who had their treatment extended to Week 98.
    Lo studio sarà considerato completato al momento dell’ultima visita dell’ultimo paziente il cui trattamento sia stato esteso alla Settimana 98.
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    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.1.1Number of subjects for this age range: 1
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 6
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 6
    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 Information not present in EudraCT
    F.3.3.2Women of child-bearing potential using contraception Information not present in EudraCT
    F.3.3.3Pregnant women Information not present in EudraCT
    F.3.3.4Nursing women Information not present in EudraCT
    F.3.3.5Emergency situation Information not present in EudraCT
    F.3.3.6Subjects incapable of giving consent personally Information not present in EudraCT
    F.3.3.7Others Information not present in EudraCT
    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 12
    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)
    In the event of potential clinical benefit and the absence of any safety signals, the protocol would be amended beyond Week 96, to allow continued dosing until patients could transition into a separate eteplirsen study until the product is commercially available.
    In caso di potenziale beneficio clinico e di assenza di qualsiasi segnale di sicurezza, il protocollo sarà modificato oltre la Settimana 96, per consentire il continuo dosaggio fino a quando i pazienti non possano passare in uno studio sull’eteplirsen separato finché il prodotto è disponibile in commercio.
    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 Decision2017-06-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-04-20
    P. End of Trial
    P.End of Trial StatusCompleted
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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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