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    Summary
    EudraCT Number:2022-000691-19
    Sponsor's Protocol Code Number:BN43881
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-09-02
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2022-000691-19
    A.3Full title of the trial
    A two-part, open-label systemic gene delivery study to evaluate the safety and expression of RO7494222 (SRP-9001) in subjects under the age of four with Duchenne muscular dystrophy
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to assess the safety and expression of RO7494222 (SRP-9001) in participants under the age of 4 with Duchenne muscular dystrophy
    A.3.2Name or abbreviated title of the trial where available
    ENVOL
    A.4.1Sponsor's protocol code numberBN43881
    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 SponsorF. Hoffmann-La Roche Ltd
    B.1.3.4CountrySwitzerland
    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 supportF. Hoffmann-La Roche Ltd
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationF. Hoffmann-La Roche Ltd
    B.5.2Functional name of contact pointTrial Information Support Line-TISL
    B.5.3 Address:
    B.5.3.1Street AddressGrenzacherstrasse 124
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4070
    B.5.3.4CountrySwitzerland
    B.5.6E-mailglobal.rochegenentechtrials@roche.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 RO7494222 (SRP-9001)
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation Yes
    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 codeRO7494222 (SRP-9001)
    D.3.9.3Other descriptive nameDelandistrogene moxeparvovec
    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 number1.33 x 10^13
    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 Yes
    D.3.11.3.5.1CAT classification and reference numberClassification is Gene therapy medicinal product; reference number is EMA/CAT/113473/2019
    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
    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
    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.1.2 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 of delandistrogene moxeparvovec
    E.2.2Secondary objectives of the trial
    To evaluate SRP-9001-dystrophin expression from delandistrogene moxeparvovec at 12 weeks as measured by Western blot of biopsied muscle tissue
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    •Signed Informed Consent Form
    •Signed Assent Form when appropriate, as determined by patient's age and individual site and country standards
    •Male at birth
    •Meets the following age requirements at the time of study drug infusion:
    – For Cohort A: 3 years of age
    – For Cohort B: 2 years of age
    – For Cohort C: >6 months to <2 years of age
    – For Cohort D: ≤6 months of age
    Note: To ensure that cohort-specific age criteria are met at the time of dosing, at the time of signing the Informed Consent Form participants should be approximately 1 month younger than the maximum age to qualify for a cohort that is actively enrolling.
    •Has a definitive diagnosis of DMD prior to screening based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test. Genetic report must describe a frameshift deletion, frameshift duplication, premature stop ("nonsense"), canonical splice site mutation, or other pathogenic variant in the DMD gene fully contained between exons 18 to 79 (inclusive) that is expected to lead to absence of dystrophin protein.
    – Mutations between or including exons 1-17 are not eligible.
    – In-frame deletions, in-frame duplications, and variants of uncertain significance (“VUS”) are not eligible.
    •Able to cooperate with age-appropriate motor assessment testing in the opinion of the investigator.
    •Has (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with the study visit schedule and all protocol requirements.
    E.4Principal exclusion criteria
    •Has elevated anti rAAVrh74 antibody titers as determined by an investigational Elecsys anti-rAAVrh74 antibody assay obtained within 31 days of the infusion day
    •Receiving regular oral corticosteroids as a treatment for DMD or planning to receive oral corticosteroids as a treatment for DMD within 1 year of baseline.
    •Major surgery within 3 months prior to Day 1 or planned surgery during Part 1 of the study.
    •Any other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy or concomitant illness or requirement for chronic drug treatment that creates unnecessary risks to participate in the study in the opinion of the investigator.
    •Known hypersensitivity to delandistrogene moxeparvovec or any excipients of the formulation
    •Medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the participant’s ability to comply with the protocol-required testing or procedures, or compromise the participant’s well-being or safety, or clinical interpretability.
    •Left ventricular ejection fraction (LVEF) <50% on the screening echocardiogram (ECHO) or clinical signs and/or symptoms of cardiomyopathy
    •Known contact with acute or active hepatitis within 12 weeks or known contact with an infected person (e.g., suspected Epstein-Barr virus [EBV], Varicella zoster virus [VZV], parvovirus B19, human herpes virus 6, and cytomegalovirus [CMV]) within 6 weeks prior to Day 1
    •Symptomatic infection (e.g., upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to Day 1.
    •Positive COVID-19 test (antigen or PCR) on Day 1 prior to infusion
    •Cohort A and B: Positive serology testing for HIV 1 and/or 2, hepatitis C, or hepatitis B
    •Cohort C and D:
    – Born prematurely (before completion of gestation at 37 weeks) or relevant pregnancy complications in the opinion of the investigator
    – Participant’s mother: Serological evidence of current, chronic, or active HIV 1 and / or 2, hepatitis B, or hepatitis C infection
    – Participant’s mother if breastfeeding:
    o Clinical signs of acute CMV infection with confirmation by CMV PCR (urine)
    o Clinically significant abnormal liver function (GGT, AST, ALT, ALP, total bilirubin, GLDH) indicative of infectious hepatitis
    o Clinically significant illness or acute infection indicative of hepatotropic virus infection (e.g., CMV, EBV, VZV, etc) within 6 weeks prior to Day 1
    o Known contact with an infected person with acute or active hepatitis within 12 weeks prior to Day 1
    •Demonstrates cognitive delay or impairment that could confound motor development in the opinion of the investigator.
    •Treatment with any of the following therapies during the specified time periods:
    – Any time:
    o Gene therapy
    o Cell-based therapy (e.g., stem cell transplantation)
    o CRISPR/Cas9, or any other form of gene editing
    – Within 12 weeks of Day 1 and any time during the study:
    o Use of human growth factor or vamorolone
    – Within 6 months of Day 1 and any time during the study:
    o Any investigational medication
    o Any treatment designed to increase dystrophin expression (e.g., Translarna™, EXONDYS 51™, VILTEPSO™)
    •Has received a live virus vaccine or mRNA vaccine within 4 weeks or inactive vaccine within 2 weeks of the Day 1 visit, or expects to receive a vaccination that cannot be reasonably delayed to accommodate concomitant corticosteroid administration during the first 3 months after Day 1.
    •Has abnormal laboratory values considered clinically significant including but not limited to:
    – GGT >2 x upper limit of normal (ULN)
    – GLDH >ULN
    – Total bilirubin >ULN.
    o Elevations in total bilirubin confirmed to be due to Gilbert’s syndrome are not exclusionary.
    – White blood cell count >18,500 per µl
    – Platelets ≤ 150,000 per µl
    •In the opinion of the investigator, the participant is not likely to be compliant with the study protocol.
    •Family does not want to disclose participant’s study participation with general practitioner or primary care physician and other medical providers.
    •Poor peripheral venous access, which, in the opinion of the investigator, will lead to difficulty in venipuncture for the purposes of protocol-mandated procedures.
    E.5 End points
    E.5.1Primary end point(s)
    •Incidence of treatment-emergent adverse events
    •Incidence of serious adverse events
    •Incidence of adverse events of special interest
    •Clinically significant changes in vital signs and physical examination findings
    •Clinically significant changes in safety laboratory assessments, ECGs, and ECHOs
    E.5.1.1Timepoint(s) of evaluation of this end point
    Throughout the study
    E.5.2Secondary end point(s)
    Change in quantity of SRP-9001-dystrophin protein expression from baseline to Week 12 as measured by Western blot
    E.5.2.1Timepoint(s) of evaluation of this end point
    At baseline and at Week 12
    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 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 Yes
    E.6.13.1Other scope of the trial description
    Immunogenicity
    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) 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.8.5The 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 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
    United Kingdom
    Belgium
    France
    Germany
    Italy
    Spain
    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 years10
    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 years10
    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: 21
    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: 7
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 14
    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
    Subjects under the age of 4 will be enrolled
    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 15
    F.4.2.2In the whole clinical trial 21
    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)
    Participants will be given the option to continue in a long-term
    extension trial after the end of the study.
    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 Decision2024-03-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-01-05
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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