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    Summary
    EudraCT Number:2016-001318-11
    Sponsor's Protocol Code Number:PB-102-F30
    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:2016-10-24
    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 number2016-001318-11
    A.3Full title of the trial
    An Open Label Study of the Safety and Efficacy of PRX 102 in Patients with Fabry Disease Currently Treated With REPLAGAL® (Agalsidase alfa)
    Estudio abierto sobre la seguridad y la eficacia de PRX-102 en pacientes con enfermedad de Fabry tratados actualmente con REPLAGAL® (Agalsidasa alfa)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety and Efficacy study assessing PRX 102 in Patients with Fabry Disease currently treated with REPLAGAL® (Agalsidase alfa)
    Estudio sobre la seguridad y la eficacia para evaluar el PRX-102 en pacientes con enfermedad de Fabry tratados actualmente con REPLAGAL® (Agalsidasa alfa)
    A.4.1Sponsor's protocol code numberPB-102-F30
    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+3497646 80 41NA
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namePegunigalsidase alfa
    D.3.2Product code PRX-102
    D.3.4Pharmaceutical form Solution for injection
    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 alpha
    D.3.9.1CAS number 1333358-30-7
    D.3.9.2Current sponsor codePRX-102
    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-galactosidase A deficiency)
    E.1.1.1Medical condition in easily understood language
    Fabry disease
    Enfermedad de Fabry
    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 19.0
    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 safety and efficacy of PRX-102 in patients with Fabry disease currently treated with agalsidase alfa
    Evaluar la seguridad y la eficiacia de PRX-102 en pacientes con enfermedad de Fabry actualmente tratados con agalsidasa alfa
    E.2.2Secondary objectives of the trial
    N/A
    NA
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age: 18-60 years
    2. A documented diagnosis of Fabry disease.
    3. Males: plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal according to laboratory range and one or more of the characteristic features of Fabry disease
    i. Neuropathic pain
    ii. Cornea verticillata
    iii. Clustered angiokeratoma
    4. Females: historical genetic test results consistent with Fabry mutations, or in the case of novel mutations a first degree male relative with Fabry disease, and one or more of the characteristic features of Fabry disease
    i. Neuropathic pain
    ii. Cornea verticillata
    iii. Clustered angiokeratoma
    5. Treatment with agalsidase alfa for at least 2 years and on a stable dose (>80% labelled dose/kg) for at least 6 months
    6. eGFR ≥ 40 ml/min/1.73 m2 by CKD-EPI equation
    7. Availability of at least 2 historical serum creatinine evaluations since starting agalsidase alfa treatment and not more than 2 years
    8. 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
    1. Edad: 18-60 años.
    2. Diagnóstico documentado de enfermedad de Fabry.
    3. Hombres. Actividad alfa-galactosidasa en plasma o en leucocitos (mediante análisis de la actividad) por debajo del límite inferior de la normalidad según el intervalo analítico y uno o varios de los rasgos característicos de la enfermedad de Fabry:
    i. Dolor neuropático
    ii. Córnea verticillata
    iii. Angioqueratoma en grupos
    4. Mujeres. Resultados de pruebas genéticas históricas coherentes con las mutaciones de Fabry o, en caso de nuevas mutaciones, familiar de primer grado varón con enfermedad de Fabry y uno o varios de los rasgos característicos de la enfermedad de Fabry:
    i. Dolor neuropático
    ii. Córnea verticillata
    iii. Angioqueratoma en grupos
    5. Tratamiento con agalsidasa alfa durante un mínimo de 2 años con una dosis estable (>80 % de la dosis autorizada/kg) durante un mínimo de 6 meses.
    6. TFGe ≥40 ml/min/1,73 m2 mediante la ecuación CKD-EPI.
    7. Disponibilidad de un mínimo de 2 evaluaciones históricas de la creatinina sérica desde el inicio del tratamiento con agalsidasa alfa de no más de 2 años.
    8. Pacientes de sexo femenino y masculino cuyas parejas estén en edad fértil y acepten el uso de un método anticonceptivo aceptable desde el punto de vista médico, sin incluir el método del ritmo.
    E.4Principal exclusion criteria
    1. History of anaphylaxis or Type 1 hypersensitivity reaction to agalsidase alfa
    2. History of renal dialysis or transplantation
    3. History of acute kidney injury in the 12 months prior to screening, including specific kidney diseases (e.g., acute interstitial nephritis, acute glomerular and vasculitic renal diseases); non-specific conditions (e.g, ischemia, toxic injury); as well as extrarenal pathology (e.g., prerenal azotemia, and acute postrenal obstructive nephropathy)
    4. Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening
    5. Urine protein to creatinine ratio (UPCR) > 0.5 g/g and not treated with an ACE inhibitor or ARB
    6. Known history of hypersensitivity to Gadolinium contrast agent
    7. Females who are pregnant, planning to become pregnant during the study, or are breast feeding
    8. Cardiovascular event (myocardial infarction, unstable angina) in the 6 month period before screening
    9. Congestive heart failure NYHA Class IV
    10. Cerebrovascular event (stroke, transient ischemic attack) in the 6 month period before screening
    11. 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. Antecedentes de anafilaxis o reacción de hipersensibilidad de tipo 1 a la agalsidasa alfa.
    2. Antecedentes de diálisis o trasplante renal.
    3. Antecedentes de lesión renal aguda en los 12 meses anteriores a la selección, incluidas nefropatías específicas (p. ej., nefritis intersticial aguda, enfermedades renales glomerulares y vasculares agudas); afecciones inespecíficas (p. ej., isquemia, lesión tóxica); así como patología extrarrenal (p. ej., azotemia prerrenal y nefropatía obstructiva posrenal aguda).
    4. Tratamiento con inhibidor de la enzima de conversión de la angiotensina (ECA) o bloqueador del receptor de la angiotensina (BRA) iniciado o dosis cambiada en las 4 semanas previas a la selección.
    5. Cociente proteínas/creatinina en orina (UPCr) >0,5 g/g y no tratado con un inhibidor de la ECA ni un BRA.
    6. Antecedentes conocidos de hipersensibilidad a un medio de contraste con gadolinio.
    7. Mujeres que estén embarazadas, planeen quedarse embarazadas durante el estudio o que estén en periodo de lactancia.
    8. Acontecimiento cardiovascular (infarto de miocardio, angina inestable) en el periodo de 6 meses previo a la selección.
    9. Insuficiencia cardiaca congestiva de clase IV de la NYHA.
    10. Acontecimiento cerebrovascular (ictus, accidente isquémico transitorio) en el periodo de 6 meses previo a la selección.
    11. Presencia de cualquier problema médico, emocional, conductual o psicológico que, a juicio del investigador 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:
    Change from baseline in:
    • Clinical laboratory tests
    • Physical examination
    • Assessment of the injection site
    • Electrocardiogram
    • Treatment-emergent adverse events
    • Ability to taper off infusion premedication throughout the first 2 months of the study
    • Requirement for use of premedication overall to manage infusion reactions
    • Treatment-emergent anti-PRX-102 antibodies

    EFFICACY ENDPOINTS:
    • Mean annualised change in estimated glomerular filtration rate (eGFRCKD-EPI)
    • Left Ventricular Mass Index (g/m2) preferably by MRI (echocardiogram can be used as an alternative)
    • Plasma Lyso-Gb3
    • Plasma Gb3
    • Urine Lyso-Gb3
    • Protein/Creatinine ratio spot urine test
    • 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
    CRITERIOS DE VALORACIÓN DE LA SEGURIDAD:
    Cambio desde el inicio en:
    • Análisis clínicos
    • Exploración física
    • Evaluación de la zona de inyección
    • Electrocardiograma
    • Acontecimientos adversos surgidos durante el tratamiento
    • Capacidad para disminuir de forma gradual la premedicación para la infusión a lo largo de los primeros 2 meses del estudio
    • Requisito de uso de la premedicación en general para tratar las reacciones a la infusión
    • Anticuerpos anti-PRX-102 surgidos durante el tratamiento
    E.5.1.1Timepoint(s) of evaluation of this end point
    As per protocol
    Segun el protocolo
    E.5.2Secondary end point(s)
    N/A
    NA
    E.5.2.1Timepoint(s) of evaluation of this end point
    N/A
    NA
    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 No
    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 EEA9
    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
    Canada
    Germany
    Netherlands
    Norway
    Spain
    United Kingdom
    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
    LPLV
    Ultima visita del último paciente
    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 months8
    E.8.9.1In the Member State concerned days15
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months8
    E.8.9.2In all countries concerned by the trial days15
    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: 22
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 22
    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)
    Treatment on PRX-102, with the option to be enrolled in an extension study upon completion of this study.
    El tratamiento de PRX-102, con la opción de estar inscrito en un estudio de extensión tras la finalización de este 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 Decision2017-01-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-12-28
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-12-17
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