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    Summary
    EudraCT Number:2020-004686-39
    Sponsor's Protocol Code Number:EFC14462
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-06-07
    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 ConcernedItaly - Italian Medicines Agency
    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 inTreatment-naïve Pediatric Participants Less than or Equal to 6 Months of Age with Infantile-Onset Pompe Disease (IOPD)
    Studio in aperto, multinazionale, multicentrico sull’efficacia, la sicurezza, la farmacocinetica e la farmacodinamica di avalglucosidasi alfa somministrato per infusione endovenosa in partecipanti pediatrici naïve al trattamento di età inferiore o uguale a 6 mesi con malattia di Pompe a esordio infantile (IOPD)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Clinical Study for IOPD Participants Less Than or Equal to 6 Months of age to Evaluate Efficacy and Safety of enzyme replacement therapy (ERT) WithAvalglucosidase Alfa(Baby-COMET)
    Studio clinico per i/le partecipanti con IOPD di età inferiore o uguale a 6 mesi per valutare l’efficacia e la sicurezza di ERT con avalglucosidasi alfa (Baby-COMET)
    A.3.2Name or abbreviated title of the trial where available
    Baby-COMET
    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 E DEVELOPPEMENT
    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 S.r.l
    B.5.2Functional name of contact pointContact Point
    B.5.3 Address:
    B.5.3.1Street AddressContact Point
    B.5.3.2Town/ cityViale Luigi Bodio 37/B
    B.5.3.3Post code20158
    B.5.3.4CountryItaly
    B.5.4Telephone number800226343
    B.5.5Fax number0239394168
    B.5.6E-mailinformazioni.medicoscientifiche@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 No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameAvalglucosidase Alfa
    D.3.2Product code [GZ402666]
    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 INNavalglucosidasi alfa
    D.3.9.2Current sponsor codeGZ402666
    D.3.9.3Other descriptive nameRECOMBINANT HUMAN ALFA-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 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 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
    Glicogenosi di tipo II
    E.1.1.1Medical condition in easily understood language
    Glycogen storage disease type II
    Glicogenosi di tipo 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.
    Determinare l’effetto del trattamento con avalglucosidasi alfa sulla sopravvivenza e sulla sopravvivenza libera da ventilazione invasiva dei/delle partecipanti con IOPD di età inferiore o pari a 6 mesi dopo 52 settimane di trattamento.
    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 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
    - To determine safety, tolerability, and immunogenicity of avalglucosidase alfa
    - To determine the PK profile at Week 12 and Week 52
    -Determinare l’effetto del trattamento con avalglucosidasi alfa sulla sopravvivenza e sulla sopravvivenza libera da ventilazione invasiva a 12 e 18 mesi, nonché la variazione del punteggio LVM-Z; punteggio AIMS; altezza, peso corporeo e Z-score della circonferenza cranica; Hex4 urinario alla Settimana 52
    -Determinare sicurezza, tollerabilità e immunogenicità di avalglucosidasi alfa
    - Determinare il profilo PK alla Settimana 12 e alla Settimana 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 a-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,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
    -I/Le partecipanti devono presentare una diagnosi confermata di malattia di Pompe a esordio infantile definita come: presenza di 2 varianti patogene di GAA e un deficit documentato di GAA nel sangue, pelle o tessuto muscolare; o la presenza di 1 variante patogena di GAA e un deficit documentato di GAA in 2 campioni distinti di sangue, pelle e tessuto muscolare (da 2 diversi tessuti o dallo stesso tessuto ma a 2 diverse date di campionamento;
    - I/Le partecipanti devono presentare del materiale immunologico cross-reattivo (CRIM) accertato prima dell’arruolamento;
    - I/Le partecipanti devono presentare cardiomiopatia al momento della diagnosi: ovvero, LVMI equivalente all’LVMI specifico per età media come pubblicato da Vogel
    • +1 deviazione standard per i/le partecipanti diagnosticati/e mediante screening neonatale o screening di fratelli/sorelle;
    • +2 deviazione standard per i/le partecipanti diagnosticati mediante valutazione clinica.
    - Genitori o il/i rappresentante/i legale/i deve/devono essere in grado di fornire il consenso informato firmato
    E.4Principal exclusion criteria
    Participants are excluded from the study if any of the following criteriaapply:
    - 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 enzyme-replacement therapy (ERT) with recombinant human acid a glucosidase (rhGAA) from any source.
    - 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.
    In presenza di uno dei seguenti criteri i partecipanti saranno esclusi dallo studio:
    Condizioni mediche
    - Partecipanti con sintomi di insufficienza respiratoria incluso qualsiasi utilizzo di ventilazione (invasiva o non invasiva) al momento dell’arruolamento.
    - Partecipanti con importanti anomalie congenite
    - Partecipanti con malattia organica clinicamente significativa (a eccezione dei sintomi relativi alla malattia di Pompe)
    - Partecipanti che abbiano ricevuto Terapia Enzimatica Sostitutiva con a-glucosidasi acida umana ricombinante( rhGAA) da qualsiasi fonte
    - Partecipanti precedentemente trattati in qualsiasi sperimentazione clinica su avalglucosidasi alfa
    - Partecipanti non idonei alla partecipazione, a giudizio dello sperimentatore, a prescindere dal motivo, comprese condizioni mediche o cliniche, o partecipanti potenzialmente a rischio di non aderenza alle procedure dello studio.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of participants who are alive and free of invasive ventilation at Week 52
    Percentuale di partecipanti in vita e liberi/e da ventilazione invasiva alla Settimana 52
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 52
    Settimana 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 and free of supplemental oxygen use at Week 52
    5/ Change from baseline to Week 52 in LVM-Z score
    6/ Change from baseline to Week 52 in AIMS score
    7/ Change from baseline to Week 52 in body length Z-scores
    8/ Change from baseline to Week 52 in body weight Z-scores
    9/ Change from baseline to Week 52 in head circumference Z-scores
    10/ Change from baseline to Week 52 in body lenght percentiles
    11/ Change from baseline to Week 52 in body weight percentiles
    12/ Change from baseline to Week 52 in head circumference percentiles
    13/ Change from baseline to Week 52 in urinary Hex4
    14/ Assessment of treatment-emergent adverse events (TEAE) including infusion-associated reactions (IAR)
    15/ Physical examination
    16/ Clinical laboratory evaluations
    17/ Vital signs measurements
    18/ 12-lead electrocardiogram (ECG)
    19/ Immunogenicity assessments
    20/ Plasma concentration of avalglucosidase alfa
    1/ Percentuale di partecipanti in vita e liberi/e da ventilazione invasiva a 12 e 18 mesi di età
    2/ Percentuale di partecipanti in vita alla Settimana 52
    3/ Percentuale di partecipanti in vita a 12 e 18 mesi di età
    4/ Percentuale di partecipanti liberi/a da utilizzo di ventilatore e liberi/e da utilizzo di ossigeno supplementare alla Settimana 52
    5/ Variazione rispetto al basale del punteggio LVM-Z alla Settimana 52
    6/ Variazione rispetto al basale del punteggio AIMS alla Settimana 52
    7/Variazione rispetto al basale alla Settimana 52 nei Z-score di crescita corporea
    8/Variazione rispetto al basale alla Settimana 52 nei Z-score di crescita corporea
    9/Variazione rispetto al basale alla Settimana 52 nei Z-score di circonferenza cranica
    10/Variazione rispetto al basale alla Settimana 52 nei percentili di lunghezza corporea
    11/ariazione rispetto al basale alla Settimana 52 nei percentili di peso corporeo
    12/ Variazione rispetto al basale alla Settimana 52 nei percentili di circonferenza cranica
    13/ Variazione rispetto al basale alla Settimana 52 dell’Hex4 urinario
    14/ Valutazione di TEAE, comprese le IAR
    15/ Esame fisico
    16/ Valutazioni cliniche di laboratorio
    17/ Misurazione dei parametri vitali
    18/ ECG a 12 derivazioni
    19/ Valutazioni di immunogenicità
    20/ Concentrazione plasmatica di avalglucosidase alfa
    E.5.2.1Timepoint(s) of evaluation of this end point
    From 1/ to 13/ : Week 52
    14/: week 52, week 212
    From 15/ to 19/: Week 52, Week 208
    20/ : at Day 1, Week 12, and Week 52
    Da 1/ a 13/: settimana 52
    14/: settimana 52 e settimana 212
    Da 15/a 19/: settimana 52 e settimana 208
    20/: al giorno 1, alla settimana 12 e alla settimana 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 No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    China
    Taiwan
    United States
    Belgium
    France
    Germany
    Italy
    Netherlands
    United Kingdom
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months1
    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.1.1.1In 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: 18
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Pediatric Participants Less than or Equal to 6 Months of Age
    partecipanti pediatrici di età inferiore o uguale a 6 mesi
    F.3.3.7Others No
    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 8
    F.4.2.2In the whole clinical trial 20
    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)
    Not applicable
    NA
    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 Decision2021-04-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-03-02
    P. End of Trial
    P.End of Trial StatusOngoing
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