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    Summary
    EudraCT Number:2013-002554-78
    Sponsor's Protocol Code Number:PB-102-F02
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-09-20
    Trial results View 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 number2013-002554-78
    A.3Full title of the trial
    An Extension of Phase 1/2, Open Label, Dose Ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 38 Weeks (9 Months) to Adult Fabry Patients
    Una extensión del estudio de fase 1/2, abierto, de determinación de la dosis para evaluar la seguridad, la tolerabilidad, la farmacocinética y los parámetros de eficacia exploratoria de PRX-102 administrado mediante infusión intravenosa cada 2 semanas durante 38 semanas (9 meses) a pacientes adultos con enfermedad de Fabry
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Extension of the PB102F01 study: A clinical study in patients with Fabry disease to assess the safety, tolerability, and the body processing of the medication PRX102, which will be given as an infusion.
    Una extensión del estudio PB102F01: Estudio en pacientes con enfermedad de Fabry para evaluar la seguridad, la tolerabilidad, la farmacocinética y los parámetros de eficacia exploratoria de PRX-102 administrado mediante infusión intravenosa
    A.4.1Sponsor's protocol code numberPB-102-F02
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01769001
    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 SponsorProtalix Ltd.
    B.1.3.4CountryIsrael
    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 supportProtalix Ltd.
    B.4.2CountryIsrael
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCato Europe GmbH
    B.5.2Functional name of contact pointRainer Schuckelt
    B.5.3 Address:
    B.5.3.1Street AddressHertzstr. 7
    B.5.3.2Town/ cityKöln
    B.5.3.3Post code50859
    B.5.3.4CountryGermany
    B.5.4Telephone number004922343794411
    B.5.5Fax number004922343794425
    B.5.6E-mailr.schuckelt@catoeurope.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 namePRX102
    D.3.2Product code PRX102
    D.3.4Pharmaceutical form 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 INNPRX102
    D.3.9.1CAS number 1333358-30-7
    D.3.9.2Current sponsor codePRX102
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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 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 namePRX102
    D.3.2Product code PRX102
    D.3.4Pharmaceutical form 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 INNPRX102
    D.3.9.1CAS number 1333358-30-7
    D.3.9.2Current sponsor codePRX102
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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: 3
    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 namePRX102
    D.3.2Product code PRX102
    D.3.4Pharmaceutical form 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 INNPRX102
    D.3.9.1CAS number 1333358-30-7
    D.3.9.2Current sponsor codePRX102
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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
    Fabry disease
    Enfermedad de Fabry
    E.1.1.1Medical condition in easily understood language
    Fabry disease is an inherited condition caused by a deficiency of an enzyme, leading to a range of systemic symptoms.
    La enfermedad de Fabry es un trastorno genético heredado provocado por la deficiencia de una enzima que conduce a una amplia gama de síntomas.
    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 14.1
    E.1.2Level PT
    E.1.2Classification code 10016016
    E.1.2Term Fabry's disease
    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 ongoing safety, tolerability, pharmacokinetics and exploratory efficacy parameters of PRX102 in adult Fabry patients who have successfully completed treatment with PRX102 in the core study PB-102-F01, and are continuing to receive treatment at the dose assigned to each patient in the PB-102-F01 study. This extension study may provide important additional long term information on safety, tolerability and clinical outcome in patients treated with different doses of PRX102.
    Evaluar la seguridad, tolerabilidad, farmacocinética y los parámetros de eficacia exploratoria en curso de PRX-102 en pacientes adultos con enfermedad de Fabry que completaron de manera exitosa el tratamiento con PRX-102 en el estudio PB-102-F01, y continúan recibiendo tratamiento con la dosis asignada para cada paciente en el estudio PB-102-F01.
    Este estudio de extensión puede proporcionar información importante adicional a largo plazo, a la seguridad, la tolerabilidad y el resultado clínico en pacientes tratados con diferentes dosis de PRX102
    E.2.2Secondary objectives of the trial
    All exploratory endpoints that were evaluated during the Phase 1/2 study PB-102-F01 will continue to be assessed in the extension protocol .These include:
    -Gb3 concentration in kidney and in skin (assessed histologically in kidney biopsy and skin punch biopsy )
    -Gb3 concentrations in plasma and urine sediment
    -LysoGb3 concentration in plasma
    -Assessment of gastrointestinal symptoms
    -Kidney functions (eGFR and proteinuria)
    -Short Form Brief Pain Inventory (BPI)
    -Left ventricular mass (LVM) and myocardial fibrosis assessment by cardiac MRI
    -Cardiac function assessment by echocardiography and stress test
    -Cerebrovascular disease assessment (clinical and MRI evaluation)
    -Mainz Severity Score Index (MSSI)
    Todos los criterios de exploración que fueron evaluados en la Fase 1/2 del estudio PB-102-F01 seguirán siendo evaluados en el protocolo de extensión.
    Estos incluyen:
    -La concentración de Gb3 en el riñón (evaluada histológicamente con las muestras de la biopsia de riñón)
    -Las concentraciones de Gb3 en plasma y sedimento en orina
    -La concentración de globotriaosilesfingosina (Liso-Gb3) en plasma
    -Evaluación de síntomas gastrointestinales
    -Funciones renales (eGFR y proteinuria)
    -Cuestionario abreviado del dolor (Brief Pain Inventory, BPI)
    -La evaluación de la masa ventricular izquierda (Left ventricular mass, LVM) y de la fibrosis miocárdica mediante RM cardíaca.
    -Evaluación de la función cardíaca mediante ecocardiograma y prueba de esfuerzo
    -Evaluación de trastorno cerebrovascular (evaluación clínica y con RM)
    -Índice de severidad de Mainz (MSSI)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Completion of Phase 1/2 study PB-102-F01
    2.The patient signs informed consent
    3.Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically acceptable method of contraception, not including the rhythm method. Acceptable methods of contraception include hormonal products, intrauterine device, or male or female condoms. Contraception should be used for 1 month after termination of treatment.
    1. Haber completado el estudio de fase 1/2 PB-102-F01
    2. Que el paciente firme el consentimiento informado.
    3.Que los pacientes de sexo femenino y los pacientes de sexo masculino cuyas parejas tengan capacidad para concebir acepten usar un método anticonceptivo aceptable desde el punto de vista médico; se excluye el método del ritmo.
    Métodos anticonceptivos aceptables incluyen los productos hormonales, dispositivo intrauterino, o condones masculinos o femeninos. Los métodos anticonceptivos se deben continuar utilizando por un mes después de completar el tratamiento.
    Anticoncepción se deben continuar utilizando por un mes después de completar el tratamiento.
    E.4Principal exclusion criteria
    1.Pregnant or nursing
    2.Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with the patient?s compliance with the requirements of the study
    1.Embarazo o lactancia
    2.Presencia de cualquier afección médica, emocional, conductual o psicológica que, según el criterio del Investigador y/o Director Médico, interfiera en el cumplimiento de los requisitos del estudio por parte del paciente.
    E.5 End points
    E.5.1Primary end point(s)
    The main outcome will be evaluation of safety in PRX102 treated subjects at each dose. Safety will be assessed by the frequency, severity, and duration of treatment-emergent adverse events (TEAEs), including clinically significant laboratory abnormalities, ECG changes from baseline, physical examination findings and assessment of the injection
    site reactions after administration of the study drug. Also AntiPRX102
    antibodies will be assessed.
    El efecto principal es la evaluación de la seguridad de los pacientes tratados en PRX102 en cada dosis.
    La seguridad se evaluará mediante la frecuencia, la gravedad y la duración de los acontecimientos adversos emergentes del tratamiento (TEAE), incluidas las anomalías de laboratorio significativas desde el punto de vista clínico, los cambios en el ECG respecto del nivel basal, los hallazgos del examen físico y la evaluación de reacciones en el lugar de inyección después de la administración del medicamento en investigación del estudio.
    También se evaluará la presencia de anticuerpos contra PRX-102.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Clinical lab tests, Physical Examination, ECG will be assessed at Screening, Visit 7 (total treatment of ~ 6 months), 13 and 20 (total treatment of ~12 months). Adverse events will be assessed at every visit.
    Injection site reactions will be assessed at every visit up to visit 20.
    Anti-PRX-102 antibodies will be assessed at Screening, Visit 2, 7, 11, 15, 20 and 2 months after visit 20.
    Análisis de Laboratorio, Examen Físico, ECG at Screening, Visita 7 (tratamiento total aproximadamente ~ 6 meses) 13 y 20 (tratamiento total aproximadamente ~ 12 meses). Los Acontecimientos Adversos se evaluaran en cada visita. Se evaluará el lugar de aplicación de la inyección en cada visita hasta la visita 20.También se evaluará la presencia de anticuerpos contra PRX-102, en las visitas 2, 7, 11, 15, 20 y 2 meses después de la visita 20.
    E.5.2Secondary end point(s)
    PHARMACOKINETIC ENDPOINTS:
    The following PK parameters will be derived from the plasma concentration versus time profiles to determine the profile of the study drug: Cmax, t1/2, Tmax, AUC0t, and AUC0?.
    EXPLORATORY EFFICACY ENDPOINTS:
    -Globotriaosylceramide (Gb3) concentration in kidney (assessed histologically in kidney biopsy)
    -Gb3 concentration in skin (assessed histologically in skin punch biopsy)
    -Gb3 concentrations in plasma and urine sediment
    -Globotriaosylsphingosine (LysoGb3) concentration in plasma
    -Assessment of gastrointestinal symptoms
    -Kidney functions (eGFR and proteinuria)
    -Short Form Brief Pain Inventory (BPI)
    -Left ventricular mass (LVM) and myocardial fibrosis assessment by cardiac MRI
    -Cardiac function assessment (echocardiography and stress test)
    -Cerebrovascular disease assessment (clinical and MRI evaluation)
    -Mainz Severity Score Index (MSSI)
    VARIABLES FARMACOCINETICAS:
    Los siguientes parámetros farmacocinéticos se derivarán de los perfiles de concentración plasmática-tiempo a fin de determinar el perfil del medicamento en investigación del estudio: Cmáx, t1/2, Tmáx, AUC0-t y AUC0-?
    VARIABLES DE EFICACIA:
    -Gb3 en el riñón (evaluada histológicamente con las muestras de la biopsia de riñón)
    Gb3 en la piel (evaluada histológicamente con las muestras de la biopsia de riñón)
    -Gb3 en plasma y el sedimento en orina
    -globotriaosilesfingosina (Liso-Gb3) en plasma
    -Síntomas gastrointestinales
    -Funciones renales (eGFR y proteinuria)
    -Cuestionario abreviado del dolor (Brief Pain Inventory, BPI)
    -Evaluación de la masa ventricular izquierda y de la fibrosis miocárdica mediante RM cardíaca.
    -Evaluación de la función cardíaca (mediante ecocardiograma)
    -Evaluación de trastorno cerebrovascular (evaluación clínica y con RM)
    -Índice de severidad de Mainz (MSSI)
    E.5.2.1Timepoint(s) of evaluation of this end point
    EXPLORATORY:
    Visit 7: Kidney biopsy & skin punch biopsy
    Screening, visit 7, 13 & 20 (after total treatment of 12 months): Gb3 concentrations in plasma and urine sediment, Globotriaosylsphingosine (LysoGb3) concentration, Assessment of gastrointestinal symptoms, Kidney functions,Short Form Brief Pain Inventory
    Visit 7 & 20: Left ventricular mass (LVM) and myocardial fibrosis, Cardiac function Mainz Severity Score Index
    Visit 20: Cerebrovascular disease assessment
    PK: preinfusion, 1 hour after infusion starts, end of infusion, 1,4,8,24,48,72,96 hours post infusions and 2 weeks post infusión.
    EXPLORATORIA:
    V7: Biopsia de riñon y de piel con sacabocados.
    Visita 7,13,& 20 (después del tratamiento total de 12 meses):Gb3 en orina y plasma, concentración de globotriaosilesfingosina (Liso-Gb3), Síntomas gastrointestinales,Funciones renales,Cuestionario abreviado del dolor (Brief Pain Inventory, BPI).V 7 & 20:Evaluación de la masa ventricular izquierda y de la fibrosis miocárdica mediante RM cardíaca, Evaluación de la función cardíaca Índice de severidad de Mainz (MSSI).V 20 Evaluación de trastorno cerebrovascular
    PK:
    Las muestras PK se recolectarán en los siguientes puntos cronológicos:
    Preinfusión, 1 hora después del inicio de la infusión y al final de la infusión. 1,4,8,24,48,72,96 horas después de la infusión y 2 semanas después de la infusión.
    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 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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Extension of the study PB-102-F01 which first administration to humans took place
    Extensión del estudio PB-102-F01 donde ya fue realizada la primera administración en humanos
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 EEA2
    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
    Australia
    Israel
    Paraguay
    Serbia
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months7
    E.8.9.1In the Member State concerned days30
    E.8.9.2In all countries concerned by the trial years2
    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 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 17
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 8
    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)
    Subjects who tolerate the infusions well and complete the study are eligible to enroll in the next extension study.
    Los pacientes que toleren bien las dosis y completen este estudio, se les dará la opción de participar en la siguiente extensión del estudio.
    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 Decision2013-11-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-11-13
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-02-17
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