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The European Union Clinical Trials Register allows you to search for protocol and results information on:
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    The EU Clinical Trials Register currently displays   43207   clinical trials with a EudraCT protocol, of which   7151   are clinical trials conducted with subjects less than 18 years old.
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    Summary
    EudraCT Number:2018-001148-67
    Sponsor's Protocol Code Number:PB-102-F60
    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:2018-11-16
    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 number2018-001148-67
    A.3Full title of the trial
    Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease
    Estudio de extensión abierto para evaluar la seguridad y la eficacia a largo plazo de pegunigalsidasa alfa (PRX-102) en pacientes con la enfermedad de Fabry
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease
    Estudio de evaluación de la seguridad y la eficacia en pacientes de pegunigalsidasa alfa (PRX-102) con la enfermedad de Fabry
    A.4.1Sponsor's protocol code numberPB-102-F60
    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 organisationProtalix Ltd.
    B.5.2Functional name of contact pointRaul Chertkoff
    B.5.3 Address:
    B.5.3.1Street Address2 Snunit St, Science Park, POB 455
    B.5.3.2Town/ cityCarmiel
    B.5.3.3Post code20100
    B.5.3.4CountryIsrael
    B.5.4Telephone number+34976468041
    B.5.6E-mailraul@protalix.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/3/17/1953
    D.3 Description of the IMP
    D.3.1Product namePegunigalsidase alfa
    D.3.2Product code PRX-102
    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 INNPEGUNIGALSIDASE ALFA
    D.3.9.1CAS number 1644392-61-9
    D.3.9.2Current sponsor codePRX-102
    D.3.9.4EV Substance CodeSUB188654
    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 (α-galactosidase A deficiency)
    Enfermedad de Fabry (déficit de alfa-galactosidasa)
    E.1.1.1Medical condition in easily understood language
    Fabry disease (α-galactosidase A deficiency)
    Enfermedad de Fabry (déficit de alfa-galactosidasa)
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    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, and efficacy parameters of 1 mg/kg pegunigalsidase alfa (PRX-102) every other week in adult Fabry patients who have successfully completed studies PB-102-F20 or PB-102-F30.
    Evaluar los parámetros de seguridad, tolerabilidad y eficacia en curso de 1 mg/kg de pegunigalsidasa alfa (PRX-102) cada dos semanas en pacientes adultos con la enfermedad de Fabry que han completado satisfactoriamente los estudios PB-102-F20 o PB-102-F30.
    E.2.2Secondary objectives of the trial
    N/A
    N/A
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Completion of study PB-102-F20 or PB-102-F30
    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 treatment termination.
    1. Finalización del estudio PB-102-F20 o PB-102-F30
    2. El paciente firma el consentimiento informado
    3. Pacientes de sexo femenino y masculino cuyas parejas estén en edad fértil y acepten el uso de un método anticonceptivo altamente eficaz aceptable desde el punto de vista médico, sin incluir el método del ritmo. Los métodos anticonceptivos aceptables incluyen productos hormonales, dispositivos intrauterinos o condones masculinos o femeninos. La anticoncepción debe usarse durante 1 mes después de la finalización del tratamiento.
    E.4Principal exclusion criteria
    1. Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with patient compliance with the requirements of the study.
    1. Presencia de cualquier problema médico, emocional, conductual o psicológico que, a juicio del investigador y/o del director médico, interferiría en el cumplimiento del paciente de los requisitos del estudio.
    E.5 End points
    E.5.1Primary end point(s)
    SAFETY ENDPOINTS:
    Changes from baseline in:
    *Clinical laboratory tests
    *Physical examination
    *Assessment of the injection site
    *Electrocardiography (ECG)
    *Treatment-emergent adverse events (TEAE)
    *Ability to taper off infusion pre-medication at the start of the study
    *Requirement for use of pre-medication overall to manage infusion reactions
    *Treatment-emergent anti-pegunigalsidase alfa antibodies
    CRITERIOS DE VALORACIÓN DE LA SEGURIDAD:
    Cambios desde el inicio en:
    • Análisis clínicos
    • Exploración física
    • Evaluación de la zona de inyección
    • Electrocardiograma (ECG)
    • Acontecimientos adversos surgidos durante el tratamiento (AAST)
    • Capacidad para disminuir de forma gradual la premedicación para la infusión al inicio del estudio
    • Requisito de uso de la premedicación en general para tratar las reacciones a la infusión
    • Anticuerpos contra la pegunigalsidasa alfa surgidos durante el tratamiento
    E.5.1.1Timepoint(s) of evaluation of this end point
    Patients who received at least one dose of study drug will be included in the safety analysis. Results of safety evaluations (including TEAEs, injection site reactions, physical examinations, ECG, and laboratory analyses, anti-drug antibodies) will be summarized and described.
    No formal statistical analyses will be performed. The study endpoints will be evaluated by various summary analyses by study visit and by change from baseline data for each efficacy endpoint. Safety and efficacy endpoints will be compared to baseline using summary statistics. Data will not be analysed by inferential statistics.
    An interim analysis may be performed for administrative purposes during the course of the study; available efficacy and safety parameters will be summarized using descriptive statistics.
    Los pacientes que reciban al menos una dosis del fármaco del estudio se incluirán en el análisis de la seguridad. Se resumirán y describirán los resultados de las evaluaciones de seguridad (incluidos AAST, reacciones en la zona de la inyección, exploraciones físicas, ECG, pruebas analíticas y anticuerpos antifármaco). No se realizarán análisis estadísticos formales en este estudio. Los criterios de valoración del estudio se evaluarán por diversos análisis resumidos por visita del estudio y por el cambio con respecto a los datos del inicio de cada criterio de valoración de la eficacia. Se compararán los criterios de valoración de seguridad y eficacia con respecto al inicio mediante estadísticas resumidas. Los datos no se analizarán mediante estadística inferencial. *Consultar Protocolo
    E.5.2Secondary end point(s)
    EFFICACY ENDPOINTS:
    *Estimated glomerular filtration rate (eGFRCKD-EPI)
    *Left Ventricular Mass Index (g/m2) by magnetic resonance imaging (MRI)
    *Plasma Gb3
    *Plasma Lyso-Gb3
    *Urine Lyso-Gb3
    *Protein/Creatinine ratio, spot urine test (UPCR)
    *Frequency of pain medication use
    *Exercise tolerance (Stress Test)
    *Short Form Brief Pain Inventory (BPI)
    *Mainz Severity Score Index (MSSI)
    *Quality of life (EQ-5D-5L)
    *Clinical events
    CRITERIOS DE VALORACIÓN DE LA EFICACIA:
    * Tasa de filtración glomerular estimada (TFGeCKD-EPI)
    *Índice de masa del ventrículo izquierdo (g/m2) mediante imágenes de resonancia magnética (IRM)
    * Gb3 en plasma
    * Lyso-Gb3 en plasma
    * Lyso-Gb3 en orina
    * Cociente proteínas/creatinina en prueba puntual de orina
    * Frecuencia de uso de analgésicos
    * Tolerancia al ejercicio (prueba de esfuerzo)
    * Cuestionario abreviado para la evaluación del dolor (Brief Pain Inventory, BPI)
    * Índice de valoración de la gravedad de Mainz (Mainz Severity Score Index, MSSI)
    * Calidad de vida (EQ-5D-5L)
    * Eventos clínicos
    E.5.2.1Timepoint(s) of evaluation of this end point
    N/A
    N/A
    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) 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 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.8.2.4Number 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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA17
    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
    Australia
    Belgium
    Brazil
    Canada
    Czech Republic
    Denmark
    Germany
    Hungary
    Italy
    Netherlands
    Norway
    Slovenia
    Spain
    Switzerland
    Turkey
    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
    Ultimo paciente ultima visita
    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 months0
    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 months0
    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: 110
    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 state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 36
    F.4.2.2In the whole clinical trial 110
    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)
    N/A
    N/A
    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 Decision2019-02-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-02-04
    P. End of Trial
    P.End of Trial StatusOngoing
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