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    Summary
    EudraCT Number:2020-004686-39
    Sponsor's Protocol Code Number:EFC14462
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2021-05-04
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2020-004686-39
    A.3Full title of the trial
    An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of avalglucosidase alfa in Treatment naïve Pediatric Participants with Infantile-Onset Pompe Disease (IOPD)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa
    A.3.2Name or abbreviated title of the trial where available
    Baby-COMET
    A.4.1Sponsor's protocol code numberEFC14462
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1246-6645
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/174/2020
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorSanofi-Aventis Recherche & Développement
    B.1.3.4CountryFrance
    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 supportSanofi-Aventis Recherche & Développement
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSanofi B.V.
    B.5.2Functional name of contact pointTeam Manager
    B.5.3 Address:
    B.5.3.1Street AddressPaasheuvelweg 25
    B.5.3.2Town/ cityAmsterdam
    B.5.3.3Post code1105 BP
    B.5.3.4CountryNetherlands
    B.5.4Telephone number0031202454000
    B.5.5Fax number0031202453952
    B.5.6E-mailstartup.nl@sanofi.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 Nexviadyme
    D.2.1.1.2Name of the Marketing Authorisation holderSanofi B.V
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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-001945-PIP01
    D.3 Description of the IMP
    D.3.1Product nameAvalglucosidase Alfa
    D.3.2Product code GZ402666
    D.3.4Pharmaceutical form Powder for 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 INNavalglucosidase alfa
    D.3.9.2Current sponsor codeGZ402666
    D.3.9.3Other descriptive nameRECOMBINANT HUMAN Α-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN
    D.3.9.4EV Substance CodeSUB120294
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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) Yes
    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 Yes
    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
    Glycogen storage disease type II
    E.1.1.1Medical condition in easily understood language
    Glycogen storage disease type II
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10053185
    E.1.2Term Glycogen storage disease type II
    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 determine the effect of avalglucosidase alfa treatment on survival and invasive ventilator-free survival of IOPD participants less than or equal to 6 months of age after 52 weeks of treatment.
    E.2.2Secondary objectives of the trial
    - To determine the effect of avalglucosidase alfa treatment on survival and invasive ventilator-free survival at 12 and 18 months of age, as well as the change in left ventricular mass Z-score (LVM Z-score); Alberta Infant Motor Scale (AIMS) score; body length, body weight, and head circumference Z scores; and urinary Hex4 at Week 52 in IOPD participants less than or equal to 6 months of age

    - To determine safety, tolerability, and immunogenicity of avalglucosidase alfa

    - To determine the PK profile at Week 12 and Week 52
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Participants must have confirmed diagnosis of infantile-onset Pompe disease defined as: the presence of 2 lysosomal acid α-glucosidase (GAA) pathogenic variants and a documented GAA deficiency from blood, skin, or muscle tissue; or the presence of 1 GAA pathogenic variant and a documented GAA deficiency from blood, skin and muscle tissue in 2 separate samples (from either 2 different tissues or from the same tissue but at 2 different sampling dates).

    - Participants must have established cross-reactive immunological material (CRIM) status available prior to enrollment.

    - Participants must have cardiomyopathy at the time of diagnosis: ie, left ventricular mass index (LVMI) equivalent to mean age specific LVMI
    +1 standard deviation for participants diagnosed by newborn screening or sibling screening;
    +2 standard deviation for participants diagnosed by clinical evaluation.

    - Parents or legally authorized representative(s) must be capable of giving signed informed consent
    E.4Principal exclusion criteria
    Participants are excluded from the study if any of the following criteria apply:
    - Participants with symptoms of respiratory insufficiency, including any ventilation use (invasive or noninvasive) at the time of enrollment.
    - Participants with major congenital abnormality.
    - Participants with clinically significant organic disease (with the exception of symptoms relating to Pompe disease).
    - Participant received any Pompe disease specific treatment, eg ERT gene therapy.
    - Participant who has previously been treated in any clinical trial of avalglucosidase alfa.
    - Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of participants who are alive and free of invasive ventilation at Week 52
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 52
    E.5.2Secondary end point(s)
    1/ Proportion of participants who are alive and free of invasive ventilation at 12 and 18 months of age

    2/ Proportion of participants who are alive at Week 52

    3/ Proportion of participants who are alive at 12 and 18 months of age

    4/ Proportion of participants who are free of ventilator use (invasive and non-invasive separate and combined) at Week 52

    5/ Proportion of participants who are free of supplemental oxygen use at Week 52

    6/ Change from baseline to Week 52 in left ventricular mass (LVM)-Z score

    7/ Change from baseline to Week 52 in Alberta Infant Motor Scale (AIMS) score

    8/ Change from baseline to Week 52 in body length Z-scores

    9/ Change from baseline to Week 52 in body weight Z-scores

    10/ Change from baseline to Week 52 in head circumference Z-scores

    11/ Change from baseline to Week 52 in body length percentiles

    12/ Change from baseline to Week 52 in body weight percentiles

    13/ Change from baseline to Week 52 in head circumference percentiles

    14/ Change from baseline to Week 52 in urinary Hex4

    15/ Assessment of treatment-emergent adverse events (TEAE) including infusion-associated reactions (IAR)

    16/ Physical examination

    17/ Clinical laboratory evaluations

    18/ Vital signs measurements

    19/ 12-lead electrocardiogram (ECG)

    20/ Immunogenicity assessments

    21/ Plasma concentration of avalglucosidase alfa
    E.5.2.1Timepoint(s) of evaluation of this end point
    1/, 3/: at 12 and 18 months of age

    2/ and from 4/ to 14/ : Week 52

    15/: week 52, week 212

    From 16/ to 20/: Week 52, Week 208

    21/ : at Day 1, Week 12, and Week 52
    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 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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA9
    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
    Taiwan
    China
    United Kingdom
    United States
    Belgium
    France
    Germany
    Italy
    Netherlands
    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 years5
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months4
    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: 18
    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) Yes
    F.1.1.3.1Number of subjects for this age range: 2
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 16
    F.1.1.5Children (2-11years) No
    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 Yes
    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 No
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Infants
    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 10
    F.4.2.2In the whole clinical trial 18
    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)
    LPLV
    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 Decision2021-05-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-10-04
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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