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    Summary
    EudraCT Number:2016-000942-77
    Sponsor's Protocol Code Number:EFC14028
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2016-06-22
    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 ConcernedSpain - AEMPS
    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
    Estudio de fase III aleatorizado, multicéntrico, multinacional y doble ciego que compara la eficacia y la seguridad de infusiones bisemanales de neoGAA (GZ402666) y alglucosidasa alfa en pacientes con enfermedad de Pompe de inicio tardío sin tratamiento previo
    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
    Estudio para comparar la eficacia y la seguridad del tratamiento de sustitución enzimática de neoGAA (GZ402666) y alglucosidasa alfa administrado bisemanal en pacientes con enfermedad de Pompe de inicio tardío sin tratamiento previo
    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-aventis, s.a.
    B.5.2Functional name of contact pointUnidad de Estudios Clínicos
    B.5.3 Address:
    B.5.3.1Street Addressc/ Josep Pla nº2, 4ª planta
    B.5.3.2Town/ cityBarcelona
    B.5.3.3Post code08019
    B.5.3.4CountrySpain
    B.5.4Telephone number93 485 94 00
    B.5.6E-mailES-unidadestudiosclinicos@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 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 INNNeoGAA
    D.3.9.1CAS number 1802558-87-7
    D.3.9.2Current sponsor codeGZ402666
    D.3.9.3Other descriptive nameRecombinant human α-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (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 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.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.3.1Product nameMyozyme®
    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 INNALGLUCOSIDASE ALFA
    D.3.9.1CAS number 420784-05-0
    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 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
    Pompe disease (acid alpha-glucosidase deficiency)
    Enfermedad de Pompe (deficiencia en ácido alglucosidasa alfa)
    E.1.1.1Medical condition in easily understood language
    Treatment of enzyme deficiency in glycogen storage disease type II (Pompe disease)
    El tratamiento de la deficiencia de la enzima en el almacenamiento de glucógeno tipo II de la enfermedad (enfermedad de 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 19.0
    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.
    El objetivo principal del estudio es determinar el efecto del tratamiento con neoGAA sobre la fuerza muscular respiratoria determinada por el % del valor previsto de Capacidad Vital Forzada (CVF) en posición erguida, frente a alglucosidasa 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).
    Determinar la seguridad y el efecto del tratamiento con neoGAA sobre la fuerza muscular inspiratoria (presión inspiratoria máxima [PIM]), la fuerza muscular espiratoria (presión espiratoria máxima [PEM]), la resistencia funcional (prueba de marcha de 6 minutos [PM6M]), la fuerza muscular de las extremidades inferiores (dinamometría manual [DM]), la función motora (Prueba Rápida de la Función Motora [Quick Motor Function Test, QMFT]), y la calidad de vida relacionada con la salud (formulario abreviado-12 [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.
    -El paciente tiene deficiencia confirmada de la enzima GAA en cualquier tejido y/o 2 mutaciones confirmadas en el gen de GAA.
    -El paciente y / o su padre / tutor legal esté dispuesto y es capaz de dar su consentimiento informado por escrito, y el paciente, si <18 años de edad, esté dispuesto a dar el asentimiento si se considera en condiciones de hacerlo.
    -El paciente (y el tutor legal del paciente si el paciente es <18 años de edad) deben tener la capacidad de cumplir con el protocolo del ensayo clínico.
    -El paciente, si es mujer y en edad fértil, debe tener una prueba de embarazo negativa (betahuman gonadotropina coriónica) al inicio del estudio.
    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
    -El paciente tiene <3 años
    -El paciente tiene hipertrofia cardiaca específica de Pompe conocida
    -El paciente depende de una silla de ruedas
    -El paciente no es capaz de deambular 40 metros sin parar y sin un dispositivo de ayuda,
    -El paciente necesita ventilación invasiva (se permite la ventilación no invasiva),
    -El paciente no es capaz de realizar correctamente mediciones repetidas de la CVF en posición erguida de 40% del valor previsto y de 85% del
    valor previsto,
    -El paciente ha recibido tratamiento previo con alglucosidasa alfa, o cualquier tratamiento en investigación para la enfermedad de Pompe.
    -El paciente ha recibido previa o actualmente terapia de inducción de tolerancia inmune.
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline in percent predicted forced vital capacity (%FVC) in upright position
    Cambio desde el momento basal en el % del valor previsto de CVF en posición erguida
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline to 12 months
    Desde la basal hasta los 12 meses.
    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
    Cambio del valor inicial de la presión inspiratoria máxima en posición vertical

    Cambio del valor inicial de la presión espiratoria máxima en posición vertical

    El cambio en resultados desde la línea base de las pruebas de caminata de seis minutos

    Cambio del valor inicial en la medición del dinamómetro de mano

    El cambio en resultados desde la línea base de los exámenes de la función motora rápida

    Cambio en la puntuaciones desde el inicio en el cuestionario de 12 puntos de salud
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline to 12 months
    Desde la basal hasta los 12 meses.
    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 concerned4
    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 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
    Argentina
    Austria
    Belgium
    Brazil
    Canada
    Colombia
    Czech Republic
    Denmark
    France
    Germany
    Italy
    Japan
    Korea, Republic of
    Mexico
    Netherlands
    Russian Federation
    Spain
    Sweden
    Switzerland
    Taiwan
    Turkey
    United Kingdom
    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
    LVLS
    Última visita del último paciente
    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 days
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months3
    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: 2
    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 No
    F.3.3.2Women of child-bearing potential using contraception Yes
    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
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    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
    Ninguno
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2016-10-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-10-06
    P. End of Trial
    P.End of Trial StatusOngoing
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