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    Summary
    EudraCT Number:2015-001578-17
    Sponsor's Protocol Code Number:SHP-GCB-402
    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:2016-01-13
    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 number2015-001578-17
    A.3Full title of the trial
    An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of Treatment with Velaglucerase alfa on Bone-related Pathology in Treatment-naïve Patients with Type 1 Gaucher Disease
    Estudio de fase 4, multicéntrico, abierto y de un solo grupo del efecto del tratamiento con velaglucerasa alfa sobre la patología ósea en pacientes con enfermedad de Gaucher de tipo 1 que no han recibido tratamiento previo.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher Disease
    Estudio de fase 4, abierto, del efecto del tratamiento con velaglucerasa alfa sobre la patología ósea en pacientes con enfermedad de Gaucher de tipo 1 que no han recibido tratamiento previo.
    A.4.1Sponsor's protocol code numberSHP-GCB-402
    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 SponsorShire Human Genetic Therapies, Inc.
    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 supportShire Human Genetic Therapies, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationShire
    B.5.2Functional name of contact pointMs Elizabeth Sawicki
    B.5.3 Address:
    B.5.3.1Street Address300 Shire Way
    B.5.3.2Town/ cityLexington,
    B.5.3.3Post codeMA 02421
    B.5.3.4CountryUnited States
    B.5.4Telephone number+34900834223
    B.5.6E-mailRegistroEspanolDeEstudiosClinicos@druginfo.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 VPRIV 400 Units powder for solution of infusion
    D.2.1.1.2Name of the Marketing Authorisation holderShire Pharmaceuticals Ireland Limited
    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/3/10/752
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Powder 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 INNVELAGLUCERASE ALFA
    D.3.9.1CAS number 884604-91-5
    D.3.9.2Current sponsor codeGA-GCB
    D.3.9.3Other descriptive nameGene activated human glucocerebrosidase, velaglucerase alfa
    D.3.9.4EV Substance CodeSUB31010
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    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 No
    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
    Gaucher Disease
    Enfermedad de Gaucher
    E.1.1.1Medical condition in easily understood language
    Gaucher disease is caused by a defective enzyme. Due to this, fatty substances build up in the cells of the body, especially in liver, spleen and bone marrow.
    La enfermedad de Gaucher es causada por el deficit de una enzima. Debido a ello, sustancias grasas se acumulan en las células del cuerpo ,especialmente en el hígado, el bazo y la médula ósea
    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 18.1
    E.1.2Level PT
    E.1.2Classification code 10075697
    E.1.2Term Gaucher's disease type I
    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 effect of VPRIV therapy (60 U/kg every other week [EOW]) in treatment-naïve
    patients with type 1 Gaucher disease on change from baseline in lumbar spine (LS) BMD
    Z-score as measured by DXA after 12 months of treatment.
    Evaluar el efecto del tratamiento con VPRIV (60 U/kg cada dos semanas [C2S]) en pacientes
    que no han recibido tratamiento previo con enfermedad de Gaucher de tipo 1 sobre el cambio respecto al momento basal de la puntuación Z de DMO de la columna lumbar (CL) medida por DEXA después de 12 meses de tratamiento.
    E.2.2Secondary objectives of the trial
    To evaluate the effect of VPRIV therapy (60 U/kg EOW) on BMD in treatment-naïve patients
    with type 1 Gaucher disease as measured by the change from baseline in LS BMD Z-score at
    24 months, and BMB as measured by MRI of the LS and femur after 12 and 24 months of
    treatment.
    Evaluar el efecto del tratamiento con VPRIV (60 U/kg C2S) sobre la DMO en pacientes que no
    han recibido tratamiento previo con enfermedad de Gaucher de tipo 1 medido por el cambio
    respecto al momento basal de la puntuación Z de DMO de la CL a los 24 meses y de la CMO
    medida por RM de la CL y el fémur después de 12 y 24 meses de tratamiento
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Each patient must meet the following criteria to be enrolled in this study.
    1. The patient has a documented diagnosis of type 1 Gaucher disease, as documented by
    deficient GCB activity in leukocytes (whole blood only) or cultured skin fibroblasts.
    Diagnosis by dry blood spot test is not sufficient. Diagnosis may be based on results obtained
    prior to screening if documented in the patient?s medical history.
    2. Patients must have a LS BMD Z-score <-1 as measured by DXA during the screening phase.
    3. Patient is treatment-naïve, ie, has not received ERT or SRT in the 12 months prior to
    enrollment.
    4. The patient is ?16 and ?65 years of age. For patients ?16 and <18 years of age a Tanner
    stage of ?4 must be established.
    5. Female patients of child bearing potential must agree to use a medically acceptable method
    of contraception at all times during the study.
    6. The patient, and the patient?s parent(s) or legal guardian(s), if applicable, understands the
    nature, scope, and possible consequences of the study and has provided written informed
    consent that has been approved by the Institutional Review Board/Independent Ethics
    Committee (IRB/IEC).
    7. The patient must be sufficiently cooperative to participate in this clinical study as judged by
    the investigator.
    Los pacientes deberán cumplir todos los criterios siguientes para poder participar en este estudio.
    1. El paciente presenta un diagnóstico documentado de enfermedad de Gaucher de tipo 1,
    definido por una deficiencia de la actividad de la GCB en leucocitos (sangre entera
    exclusivamente) o fibroblastos de piel en cultivo. El diagnóstico por una prueba en gotas de
    sangre seca no es suficiente. Este podrá basarse en los resultados obtenidos antes de la
    selección si queda constancia en la historia clínica del paciente.
    2. Los pacientes deben tener una puntuación Z de DMO de la CL <-1 medida por DEXA
    durante la fase de selección.
    3. El paciente no ha sido tratado previamente, es decir, no ha recibido TES o TRS en los
    12 meses anteriores a la inclusión.
    4. El paciente tiene 16 años como mínimo y 65 como máximo. En el caso de los pacientes >=16 y <18 años de edad se debe establecer un estadio de Tanner >=4.
    5. Las mujeres en edad fértil deben acceder a utilizar un método anticonceptivo médicamente aceptable en todo momento durante el estudio.
    6. El paciente, y sus progenitores o tutores legales, si procede, entiende la naturaleza, alcance y posibles consecuencias del estudio y ha otorgado su consentimiento informado por escrito, que ha sido aprobado por el comité ético de investigación clínica (CEIC).
    7. El paciente debe ser lo suficientemente colaborador para participar en este estudio clínico, según el criterio del investigador.
    E.4Principal exclusion criteria
    Patients who meet any of the following criteria will be excluded from the study.
    1. Neurological symptoms indicating that the patient has type 3 Gaucher disease.
    2. A significant comorbidity, which, as determined by the investigator, might affect study data
    or confound the study results (eg, malignancies, primary biliary cirrhosis, autoimmune liver
    disease, etc).
    3. Any osteoporosis-specific treatment (eg, bisphosphonates) or treatment with erythropoietin
    (or erythropoietin-like substances) during the past year.
    4. Structural, joint-associated bone damage of such extent and severity that the investigator
    deems it could impact participation in the study and assessment of relevant study endpoints
    (eg, pain).
    5. The patient is pregnant or lactating.
    6. The patient has had a splenectomy.
    7. The patient is enrolled in another clinical study that involves clinical investigations or use of
    any investigational product (drug or device) within 30 days prior to study enrollment or at
    any time during the study.
    Se excluirá del estudio a los pacientes que cumplan alguno de los criterios citados a
    continuación.
    1. Síntomas neurológicos que indiquen que el paciente tiene enfermedad de Gaucher de tipo 3.
    2. Una comorbilidad significativa que, según lo determinado por el investigador, pudiera afectar a los datos del estudio o confundir sus resultados (p. ej., neoplasias malignas, cirrosis biliar primaria, enfermedad hepática autoinmunitaria, etc.).
    3. Cualquier tratamiento específico para la osteoporosis (p. ej., bisfosfonatos) o tratamiento con eritropoyetina (o compuestos similares a la eritropoyetina) durante el último año
    4. Lesión ósea estructural asociada a la articulación de una extensión e intensidad tales que el
    investigador considera que podría afectar a la participación en el estudio y a la evaluación de
    los criterios de valoración relevantes del estudio (p. ej., dolor).
    5. La paciente está embarazada o dando el pecho.
    6. El paciente se ha sometido a una esplenectomía.
    7. El paciente está participando en otro ensayo clínico que implica la investigación o el uso
    clínicos de algún producto en investigación (fármaco o dispositivo) en los 30 días previos a
    la inclusión en el estudio o en cualquier momento durante el mismo.
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline to 12 months (Week 51) in LS BMD Z-score as measured by DXA.
    Assessments will be performed at the screening visit and Weeks 25, 51, 77, and 103 (end of
    study).
    Cambio desde el momento basal hasta los 12 meses (semana 51) de la puntuación Z de
    DMO de la CL medida por DEXA. Las evaluaciones se realizarán en la visita de
    selección y en las semanas 25, 51, 77 y 103 (final del estudio).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Assessments will be performed at the screening visit and Weeks 25, 51, 77, and 103 (end of
    study).
    Las evaluaciones se realizarán en la visita de selección y en las semanas 25, 51, 77 y 103 (final del estudio).
    E.5.2Secondary end point(s)
    The key secondary endpoints of this study are:
    ? Change from baseline to 12 months (Week 51) and 24 months (Week 103 [end of study]) in
    BMB score (MRI of lumbar spine and femur). Assessments will be performed at the baseline
    visit and Weeks 25, 51, 77, and 103 (end of study).
    ? Change from baseline in LS BMD Z-score at 24 months (Week 103 [end of study]).
    Assessments will be performed at the screening visit and Weeks 25, 51, 77, and 103 (end of
    study).
    Las variables secundarias principales de este estudio son:
    . Cambio desde el momento basal hasta los 12 meses (semana 51) y los 24 meses
    (semana 103 [final del estudio]) de la puntuación de DMO (RM de la CL y el fémur).
    Las evaluaciones se realizarán en la visita basal y en las semanas 25, 51, 77 y 103
    (final del estudio).
    . Cambio desde el momento basal hasta los 24 meses (semana 103 [final del estudio])
    de la puntuación Z de DMO de la CL. Las evaluaciones se realizarán en la visita de
    selección y en las semanas 25, 51, 77 y 103 (final del estudio)
    E.5.2.1Timepoint(s) of evaluation of this end point
    . Assessments will be performed at the baseline visit and Weeks 25, 51, 77, and 103 (end of study).

    . Assessments will be performed at the screening visit and Weeks 25, 51, 77, and 103 (end of
    study).
    . Las evaluaciones se realizarán en la visita basal y en las semanas 25, 51, 77 y 103
    (final del estudio).

    . Las evaluaciones se realizarán en la visita de selección y en las semanas 25, 51, 77 y 103 (final del estudio).
    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 No
    E.6.7Pharmacodynamic No
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    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.8.3 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 EEA7
    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
    Canada
    Germany
    Israel
    Italy
    Spain
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee No
    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
    UVUP
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    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 years3
    E.8.9.2In all countries concerned by the trial months5
    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: 10
    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) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 30
    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 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 No
    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 12
    F.4.2.2In the whole clinical trial 40
    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)
    At the end of the study the patient?s doctor or primary care doctor will discuss the options for continued care
    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 Decision2016-02-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-02-04
    P. End of Trial
    P.End of Trial StatusOngoing
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