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    Summary
    EudraCT Number:2016-005001-39
    Sponsor's Protocol Code Number:4658-us-202
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2017-03-16
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2016-005001-39
    A.3Full title of the trial
    Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects with Duchenne Muscular Dystrophy who Participated in Study 4658-us-201
    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 patients with Duchenne Muscular Dystrophy.
    A.4.1Sponsor's protocol code number4658-us-202
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01540409
    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 organisationSarepta Therapeutics, Inc.
    B.5.2Functional name of contact pointMedical Director
    B.5.3 Address:
    B.5.3.1Street Address215 First Street
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post code02142
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1617274-4000
    B.5.6E-mailtrialinfo@sarepta.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 numberEMEA/OD/049/08
    D.3 Description of the IMP
    D.3.1Product nameEteplirsen Injection
    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 INNAVI-4658
    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 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
    E.1.1.1Medical condition in easily understood language
    Duchenne muscular dystrophy
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.1
    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 assess the ongoing efficacy, safety, and tolerability of treatment with eteplirsen injection in Duchenne muscular dystrophy (DMD) subjects who have successfully completed Study 4658-us-201.
    E.2.2Secondary objectives of the trial
    To evaluate the correlation between biomarkers for DMD and the clinical status of participating DMD subjects.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    The subject has successfully completed 28 weeks of treatment in Study 4658-us-201.
    E.4Principal exclusion criteria
    The subject has a prior or ongoing medical condition that, in the Investigator's opinion, (i) could adversely affect the safety of the subject, (ii) or make it unlikely that the course of treatment or follow-up would be completed, (iii) or impair the assessment of study results.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint will be the change from baseline in 6-Minute Walk Test (6MWT) through Week 240 (cumulative Study 4658-us-201 + 4658-us-202) by Treatment Week.

    The primary pharmacodynamic (biological) endpoint will be the change from baseline to Week 48 (cumulative Study 4658-us-201 + 4658-us-202) in the percentage of dystrophin positive fibers as measured by immunohistochemistry (IHC) using anti-dystrophin antibody MANDYS106.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 240 (cumulative Study 4658-us-201 + 4658-us-202)
    Week 48 (cumulative Study 4658-us-201 + 4658-us-202)
    E.5.2Secondary end point(s)
    Changes from baseline through combined Week 240 in:
    North Star Ambulatory Assessment (NSAA) Total Score, ability to independently rise from supine, rise time, and timed 10-meter walk/run
    Timed 4-Step Test
    9-Hole Peg Test
    Maximum voluntary isometric contraction test (MVICT) to measure elbow flexion and extension, knee flexion and extension, and hand grip strength
    Pulmonary function tests (PFTs): forced vital capacity (FVC), percent predicted FVC (FVC%p), maximum expiratory pressure (MEP), percent predicted MEP (MEP%p), maximum inspiratory pressure (MIP), percent predicted MIP (MIP%p), forced expiratory volume in 1 second (FEV1), percent predicted FEV1, and FEV1/FVC ratio

    Patient-Reported Outcomes Efficacy Endpoints
    Change from baseline through combined Week 240 in Pediatric Quality of Life Inventory™ (PedsQL):
    Child report (total score and domain scores)
    Parent report (total score and domain scores)
    Child neuromuscular report total score
    Parent neuromuscular report total score

    Other key pharmacodynamic (biological) endpoints are:
    Exon skipping (assessed by reverse transcription polymerase chain reaction [RT-PCR]) through combined Week 180
    Change from baseline in percentage of dystrophin positive fibers at Weeks 12 and 24 as measured in the muscle biopsy tissue using IHC
    Difference from untreated controls in percentage of dystrophin positive fibers at combined Week 180 as measured in the muscle biopsy tissue using IHC
    Changes from baseline through combined Week 48 in muscle biopsy levels of dystrophin intensity per fiber (determined by BIOQUANT® software)
    Difference from untreated controls at combined Week 180 in muscle biopsy levels of dystrophin intensity per fiber (determined by BIOQUANT)
    Difference from untreated controls in dystrophin percent of normal protein by Western blot, combined Week 180 only
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 240 (cumulative Study 4658-us-201 + 4658-us-202)
    Week 180 (cumulative Study 4658-us-201 + 4658-us-202)
    Weeks 12 and 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 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 No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    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.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months1
    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: 12
    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: 12
    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 Yes
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Males between the ages 7-13, inclusive
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    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)
    Patients completing the trial may go on to commercial therapy.
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
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