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    Summary
    EudraCT Number:2014-005544-18
    Sponsor's Protocol Code Number:PB-102-F03
    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:2015-02-16
    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 number2014-005544-18
    A.3Full title of the trial
    A Multi Center Extension Study of PRX-102 Administered by Intravenous Infusions Every 2 Weeks for 24 Months to Adult Fabry Patients
    Estudio de extensión multicéntrico de PRX-102 administrado mediante infusión intravenosa cada 2 semanas durante 24 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
    Study of PRX-102 Administered by Intravenous Infusions Every 2 Weeks for 24 Months to Adult Fabry Patients
    Estudio de PRX-102 administrado mediante infusión intravenosa cada 2 semanas durante 24 meses a pacientes adultos con enfermedad de Fabry
    A.4.1Sponsor's protocol code numberPB-102-F03
    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 organisationSERMES PLANIFICACION S.L
    B.5.2Functional name of contact pointUnidad de Puesta en Marcha
    B.5.3 Address:
    B.5.3.1Street AddressC/Rufino González 14, Esc.1ª-2ºD
    B.5.3.2Town/ cityMadrid
    B.5.3.3Post code28027
    B.5.3.4CountrySpain
    B.5.4Telephone number+34913275025
    B.5.5Fax number+34917542721
    B.5.6E-mailstart-up@sermescro.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 18.0
    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 PRX-102 in adult Fabry patients who have successfully completed treatment with PRX-102 in studies PB-102-F01 and PB-102-F02- and are continuing to receive treatment at the dose each patient reeceived in study PB-102-F02.
    Evaluar los parámetros de seguridad, tolerabilidad y eficacia de PRX-102 en pacientes adultos con enfermedad de Fabry que completaron de manera exitosa el tratamiento con PRX-102 en los estudios PB-102-F01 y PB-102-F02 y continúan recibiendo tratamiento con la dosis que cada paciente recibió en el estudio PB-102-F02.
    E.2.2Secondary objectives of the trial
    Not applicable
    No aplica
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Completion of study PB-102-F02
    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. Que se haya completado el estudio PB-102-F02
    2. Que el paciente firme el consentimiento informado
    3. Que las pacientes y los pacientes cuyas parejas tengan capacidad para concebir acepten usar un método anticonceptivo aceptable desde el punto de vista médico; se excluye el método del ritmo. Los métodos anticonceptivos aceptables incluyen productos hormonales, dispositivos intrauterinos o condones masculinos o femeninos. Deben utilizarse durante 1 mes después de la finalización del 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)
    Efficacy Variables
    The efficacy endpoints evaluated in Study PB-102-F02 will continue to be measured in this extension study. Baseline values for this extension study will be the values from the last infusion of study PB-102-F02.
    Safety Variables: 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, assessment of the injection site reactions and existence of Anti PRX-102 antibodies after administration of the study drug.
    Variables de eficacia
    Los criterios de valoración de la eficacia evaluados en el estudio PB-102-F02 se continuarán midiendo en este estudio de extensión. Los valores basales para este estudio de extensión serán los valores de la última infusión del estudio PB-102-F02.
    Variables de seguridad: La seguridad se evaluará mediante la frecuencia, la gravedad y la duración de los eventos adversos emergentes del tratamiento (EAET), 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, la evaluación del lugar de la inyección y la existencia de anticuerpos contra PRX-102 después de la administración del fármaco del estudio.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Efficacy Variables
    Gb3 and Lyso-Gb3 concentrations in plasma: baseline/every 3 months
    Gastrointestinal symptoms, Kidney functions, BPI: baseline/every 3 months
    Gb3 concentration in kidney and in skin: last treatment visit
    Left ventricular mass and myocardial fibrosis: baseline/ every 12 months
    Cardiac function and stress test: baseline/every 12 months
    Cerebrovascular disease: baseline/last treatment of study
    MSSI: baseline/every 6 months
    Safety Variables
    Laboratory values and physical examination: screening and visits 7, 14, 20, 27, 33, 40, 46, 53
    ECG: screening and visits 14, 40, 53
    Anti PRX-102 antibodies: screening and visits 5, 7, 14, 20, 27, 33, 40,46,53 and 3 months after last infusion
    AE: every study visit
    Injection site reactions: Every study treatment visit
    Variables de eficacia
    Concentraciones de Gb3 y Liso-Gb3 en plasma: nivel basal/cada 3 meses
    Sínt. gastrointestinales, Fun. renales, BPI: nivel basal/cada 3 meses
    Concentración Gb3 en riñón y piel: última visita del trat.
    LVM y fibrosis miocárdica: nivel basal/cada 12 meses
    Función cardíaca y prueba de esfuerzo: nivel basal/cada 12 meses
    Trastorno cerebrovascular: nivel basal y última visita del trat.
    MSSI: nivel basal/cada 6 meses
    Variables de seguridad
    Valores de lab. Y examen físico: screening y visitas 7, 14, 20, 27, 33, 40, 46, 53
    ECG: screening y visitas 14, 40, 53
    Anticuerpos PRX-102: screening y visitas 5, 7, 14, 20, 27, 33, 40,46,53 y 3 meses después de la última infusión
    AE: todas las visitas
    Reacciones lugar de la inyección: Todas las visitas de trat.
    E.5.2Secondary end point(s)
    Not applicable
    No aplica
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not applicable
    No aplica
    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) 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 and PB-102-F02
    Extensión del estudio PB-102-F01 y PB-102-F02
    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 No
    E.8.1.2Open No
    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) 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
    Paraguay
    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 years3
    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 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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 3
    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 Decision2015-04-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-04-15
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-08-26
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