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    Summary
    EudraCT Number:2021-000078-27
    Sponsor's Protocol Code Number:SRP-9001-102
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2022-11-10
    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 number2021-000078-27
    A.3Full title of the trial
    A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial for Duchenne Muscular Dystrophy Using SRP-9001
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 (Delandistrogene Moxeparvovec) for Duchenne Muscular Dystrophy (DMD)
    A.4.1Sponsor's protocol code numberSRP-9001-102
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03769116
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/052/2022
    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 pointPatient Recruitment
    B.5.3 Address:
    B.5.3.1Street Address215 First Street
    B.5.3.2Town/ cityCambridge, MA
    B.5.3.3Post code02142
    B.5.3.4CountryUnited States
    B.5.4Telephone number1888727-3782
    B.5.6E-mailSareptAlly@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 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/3/20/2250
    D.3 Description of the IMP
    D.3.1Product nameDelandistrogene moxeparvovec
    D.3.2Product code SRP-9001
    D.3.4Pharmaceutical form 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 INNDelandistrogene moxeparvovec
    D.3.9.1CAS number 2305040-16-6
    D.3.9.2Current sponsor codeSRP-9001
    D.3.9.3Other descriptive namemicroDys-IV-001
    D.3.9.4EV Substance CodeSUB197789
    D.3.10 Strength
    D.3.10.1Concentration unit vector genomes (vg)/mL
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number13300000000000
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    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) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    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 Yes
    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 infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Duchenne Muscular Dystrophy
    E.1.1.1Medical condition in easily understood language
    Duchenne muscular dystrophy is a rare, serious, life-threatening, X-linked recessive, degenerative neuromuscular disease caused by mutations in the dystrophin gene.
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10052655
    E.1.2Term Duchenne muscular dystrophy gene carrier
    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
    The primary objectives of this study are the assessments of the safety and efficacy of intravenous administration of SRP-9001 in DMD participants.
    E.2.2Secondary objectives of the trial
    The secondary objectives include the effects of SRP-9001 on physical functional assessments and micro-dystrophin expression as measured by immunofluorescence (IF) and IF percent dystrophin positive fibers (PDPF) of biopsied muscle tissue.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Established clinical diagnosis of DMD and documented dystrophin gene mutation of DMD phenotype.
    • Indication of symptomatic muscular dystrophy by protocol-specified criteria.
    • Ability to cooperate with motor assessment testing.
    • Stable dose equivalent of oral corticosteroids for at least 12 weeks.
    • A frameshift mutation contained between exons 18 and 58 (inclusive).

    Other inclusion criteria apply.
    E.4Principal exclusion criteria
    • Impaired cardiovascular function on echocardiogram.
    • Prior or ongoing medical condition on physical examination, electrocardiogram, or laboratory findings that could adversely affect participant safety, compromise completion of follow-up, or impair assessment of study results.
    • Exposure to another investigational drug or exon skipping medication within 6 months of screening.
    • Exposure to an investigational or commercial gene therapy product.
    • Abnormal liver or renal function by protocol-specified criteria.

    Other exclusion criteria apply.
    E.5 End points
    E.5.1Primary end point(s)
    • Change From Baseline in Quantity of Micro-dystrophin Protein Expression as Measured by Western Blot
    • Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score
    E.5.1.1Timepoint(s) of evaluation of this end point
    • Western Blot: Baseline up to Week 12 (Part 1)
    • NSAA Total Score: Baseline up to Week 48 (Part 1)
    E.5.2Secondary end point(s)
    • Change From Baseline in Time to Rise From the Floor
    • Change From Baseline in Time to Ascend 4 Steps
    • Change From Baseline in Time of 10 Meter Timed Test
    • Change From Baseline in Time of 100 Meter Timed Test
    • Change From Baseline in Quantity of Micro-dystrophin Expression Measured by IF Fiber Intensity
    • Change From Baseline in Quantity of Micro-dystrophin Expression Measured by IF PDPF
    E.5.2.1Timepoint(s) of evaluation of this end point
    • Rise From the Floor: Baseline up to Week 48 (Part 1)
    • Ascend 4 Steps: Baseline up to Week 48 (Part 1)
    • 10 Meter Timed Test: Baseline up to Week 48 (Part 1)
    • 100 Meter Timed Test: Baseline up to Week 48 (Part 1)
    • IF Fiber Intensity: Baseline up to Week 12 (Part 1)
    • IF PDPF: Baseline up to Week 12 (Part 1)
    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 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 No
    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 Yes
    E.8.1.7.1Other trial design description
    Parallel up to the measurement of the primary outcomes at Week 48.
    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 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 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
    Last participant last visit
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years5
    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: 41
    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: 41
    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
    Pediatric participants: the youngest trial participant was 4 years old.
    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 41
    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
    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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