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    Summary
    EudraCT Number:2016-000942-77
    Sponsor's Protocol Code Number:EFC14028
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Restarted
    Date on which this record was first entered in the EudraCT database:2018-02-06
    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 number2016-000942-77
    A.3Full title of the trial
    A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of NeoGAA (GZ402666) and Alglucosidase Alfa in Treatmentnaïve Patients with Late-onset Pompe Disease
    Studio randomizzato di fase 3, multicentrico, internazionale, in doppio cieco, per il confronto dell’efficacia e della sicurezza di infusioni ripetute ogni due settimane di neoGAA (GZ402666) e alglucosidasi alfa in pazienti naïve al trattamento con malattia di Pompe a esordio tardivo
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease
    Studio per il confronto dell’efficacia e
    della sicurezza della terapia di sostituzione enzimatica neoGAA e alglucosidasi alfa somministrati una volta ogni due settimane in
    pazienti con malattia di Pompe a esordio tardivo mai precedentemente trattati per la malattia di Pompe
    A.3.2Name or abbreviated title of the trial where available
    COMET
    COMET
    A.4.1Sponsor's protocol code numberEFC14028
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1178-4806
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorGENZYME CORPORATION
    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 supportGenzyme Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSANOFI S.p.A.
    B.5.2Functional name of contact pointCONTACT POINT
    B.5.3 Address:
    B.5.3.1Street AddressVIALE BODIO, 37/B
    B.5.3.2Town/ cityMILANO
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/14/1251
    D.3 Description of the IMP
    D.3.1Product nameneoGAA
    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 IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 1802558-87-7
    D.3.9.2Current sponsor codeGZ402666
    D.3.9.3Other descriptive nameα-glucosidasi acida umana ricombinante coniugata con multiple copie di bis-mannosio-6- fosfato-tetra-mannosio glicano sintetico (NEOGAA)
    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.IMP: 2
    D.1.2 and D.1.3IMP RoleComparator
    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 MYOZYME®
    D.2.1.1.2Name of the Marketing Authorisation holderGenzyme Europe 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 numberEU/3/00/018
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNALGLUCOSIDASE ALFA
    D.3.9.1CAS number 420784-05-0
    D.3.9.2Current sponsor code.
    D.3.9.4EV Substance CodeSUB21275
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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 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
    Pompe disease (acid alpha-glucosidase deficiency)
    Malattia di Pompe (deficit di alfa-glucosidosi acido)
    E.1.1.1Medical condition in easily understood language
    Treatment of enzyme deficiency in glycogen storage disease type II (Pompe disease)
    Trattamento del deficit enzimatico in glicogenosi di tipo II (malattia di Pompe)
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10036143
    E.1.2Term Pompe's disease
    E.1.2System Organ Class 100000004850
    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 neoGAA (GZ402666) treatment on respiratory muscle strength measured by percent predicted forced vital capacity (% FVC) in the upright position, as compared to alglucosidase alfa.
    Determinare l’effetto del trattamento con neoGAA sulla forza della muscolatura respiratoria,
    misurata come percentuale predetta della capacità vitale forzata (FVC) in posizione eretta, rispetto ad alglucosidasi alfa.
    E.2.2Secondary objectives of the trial
    To determine the safety and effect of neoGAA treatment on inspiratory muscle strength (maximum inspiratory pressure [MIP]), expiratory muscle strength (maximum expiratory pressure [MEP]), unctional endurance (6-minute walk test[6MWT]), lower extremity muscle strength (hand-held dynamometry [HHD]), motor function (Quick Motor Function Test [QMFT]), and health-related quality of life (SF-12).
    Determinare la sicurezza e l’effetto del trattamento con neoGAA sulla forza della muscolatura
    inspiratoria (pressione inspiratoria massima [MIP]),sulla forza della muscolatura espiratoria (pressione espiratoria massima [MEP]),
    sulla resistenza funzionale (test del cammino dei 6 minuti [6-minute walk test, 6MWT]), sulla forza della muscolatura delle estremità inferiori
    (dinamometria manuale [hand-held dynamometry, HHD]), sulla funzionalità motoria (Test rapido della funzione motoria [Quick Motor Function Test, QMFT]) e sulla qualità della vita correlata alla salute (modulo breve a 12 voci [Short Form-12, SF-12]).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -The patient has confirmed GAA enzyme deficiency from any tissue source and/or 2 confirmed GAA gene mutations.
    -The patient and/or their parent/legal guardian is willing and able to provide signed informed consent, and the patient, if <18 years of age, is willing to provide assent if deemed able to do so.
    -The patient (and patient’s legal guardian if patient is <18 years of age) must have the ability to comply with the clinical protocol.
    -The patient, if female and of childbearing potential, must have a negative pregnancy test (betahuman chorionic gonadotropin) at baseline.
    - Il paziente presenta un deficit dell’enzima GAA confermato a partire da qualsiasi fonte di tessuto e/o 2 mutazioni confermate del
    gene GAA.
    -Il paziente e / o i loro genitori / tutori legali sono disposti e in grado di fornire e firmare il consenso informato, e il paziente, se ha
    meno di 18 anni di età, è disposto a fornire l'assenso se ritenuto in grado di farlo.
    -Il paziente ( e tutore legale del paziente se il paziente è <18 anni di età) deve avere la capacità di rispettare il protocollo clinico.
    -Il paziente, se di sesso femminile e in età fertile, deve avere un test di gravidanza negativo (beta-gonadotropina corionica umana)
    al basale
    E.4Principal exclusion criteria
    -The patient is <3 years of age.
    -The patient has known Pompe specific cardiac hypertrophy.
    -The patient is wheelchair dependent.
    -The patient is not able to ambulate 40 meters (approximately 130 feet) without topping and without an assistive device.
    -The patient requires invasive-ventilation (non-invasive ventilation is allowed).
    -The patient is not able to successfully perform repeated forced vital capacity (FVC) measurements in upright position of ≥40% predicted and ≤85% predicted.
    -The patient has had previous treatment with alglucosidase alfa or any investigational therapy for Pompe disease.
    -The patient has prior or current use of immune tolerance induction therapy
    - Il paziente ha <3 anni di età
    - il paziente presenta un’ipertrofia cardiaca nota
    specifica della malattia di Pompe
    - il paziente è dipendente dalla sedia a rotelle
    - il paziente non è in grado di camminare per 40 metri (circa 130 piedi) senza fermarsi e senza un dispositivo di assistenza
    - il paziente richiede una ventilazione invasiva (la ventilazione non invasiva è consentita)
    - il paziente non è in grado di eseguire con successo misurazioni ripetute della FVC in posizione eretta ≥40% del predetto e ≤85%
    del predetto
    - il paziente ha ricevuto un precedente trattamento con alglucosidasi alfa o qualsiasi terapia sperimentale per la malattia di Pompe
    - Il paziente ha fatto uso in precedenza, o sta facendo uso, di una terapia di induzione della tolleranza immunologica
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline in percent predicted
    forced vital capacity (%FVC) in upright position
    Variazione della % predetta di FVC in posizione eretta
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline to 12 months
    dal basale a 12 mesi
    E.5.2Secondary end point(s)
    Change from baseline in maximal
    inspiratory pressure in upright position
    Change from baseline in maximal expiratory pressure in upright position
    Change from baseline in six-minute walk test scores
    Change from baseline in hand-held dynamometry measurement
    Change from baseline in Quick Motor Function Test scores
    Change from baseline in 12- Item Short-form health survey scores
    Variazione rispetto al basale della massima pressione inspiratoria in posizione eretta
    Variazione rispetto al basale della pressione espiratoria massima in posizione eretta
    Variazione rispetto al basale del punteggio del test del cammino dei 6 minuti
    Variazione rispetto al basale della misurazione con dinamometria manuale
    Variazione rispetto al basale del punteggio del test rapido della funzionalità motoria
    Variazione rispetto al basale del punteggio del modulo breve a 12 voci sulla qualità della salute
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline to 12 months
    dal basale a 12 mesi
    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 Yes
    E.6.12Pharmacoeconomic Yes
    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 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 No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA36
    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
    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 years4
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months3
    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: 1
    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: 1
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 1
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 84
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 4
    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 Yes
    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
    Patients <18 years of age
    I pazienti <18 anni di età
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 43
    F.4.2.2In the whole clinical trial 90
    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
    nessuno
    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 Decision2016-08-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-08-01
    P. End of Trial
    P.End of Trial StatusRestarted
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