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    Summary
    EudraCT Number:2017-001528-23
    Sponsor's Protocol Code Number:PB-102-F50
    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:2017-10-16
    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 number2017-001528-23
    A.3Full title of the trial
    A Phase 3, Open Label, Switch Over Study to Assess the Safety, Efficacy and Pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks for 52 weeks in Patients with Fabry Disease Currently Treated with Enzyme Replacement Therapy; Fabrazyme® (agalsidase beta) or Replagal™ (agalsidase alfa)
    Estudio en fase III, abierto y de grupos cruzados para evaluar la seguridad, la eficacia y la farmacocinética de pegunigalsidasa alfa (PRX-102), en dosis de 2 mg/kg, administrada mediante infusión intravenosa cada 4 semanas durante 52 semanas en pacientes con enfermedad de Fabry que actualmente reciben tratamiento de sustitución enzimática: Fabrazyme® (agalsidasa beta) o Replagal™ (agalsidasa alfa)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered Every 4 Weeks in Fabry Disease Patients (BRIGHT)
    Estudio de fase III de seguridad, eficacia y FC de pegunigalsidasa alfa (PRX-102), en dosis de 2 mg/kg, administrada cada 4 semanas en pacientes con enfermedad de Fabry
    (BRIGHT)
    A.4.1Sponsor's protocol code numberPB-102-F50
    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 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 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 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-galactosidasa).
    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 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 safety, efficacy and pharmacokinetics of pegunigalsidase alfa (PRX-102) in patients with Fabry disease currently treated with currently commercially available ERT (agalsidase alfa or agalsidase beta).
    Evaluar la seguridad, la eficacia y la farmacocinética de pegunigalsidasa alfa
    (PRX-102) en pacientes con enfermedad de Fabry tratados actualmente con un TSE comercialmente disponible en la actualidad (agalsidasa alfa o agalsidasa beta).
    E.2.2Secondary objectives of the trial
    Not applicable
    No aplica
    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 the laboratory reference ranges and one or more of the characteristic features of Fabry disease
    a. Neuropathic pain
    b. Cornea verticillata
    c. 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
    a. Neuropathic pain
    b. Cornea verticillata
    c. Clustered angiokeratoma
    5. Treatment with agalsidase alfa or agalsidase beta for at least 3 years and on a stable dose (>80% labelled dose/kg) for at least 6 months
    6. eGFR ≥ 30 mL/min/1.73 m2 by CKD-EPI equation at screening visit
    7. Availability of at least 3 historical serum creatinine evaluations since starting agalsidase alfa or agalsidase beta treatment and not more than 2 years old
    8. Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted, highly effective method of contraception. These include combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomised partner, or sexual abstinence
    9. Patients whose clinical condition, in the opinion of the Investigator, is suitable for treatment with ERT every 4 weeks.
    1. Edad: 18-60 años.
    2. Diagnóstico documentado de enfermedad de Fabry
    3. Hombres: actividad de la alfa-galactosidasa en plasma y/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
    a. Dolor neuropático
    b. Córnea verticillata
    c. 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
    a. Dolor neuropático
    b. Córnea verticillata
    c. Angioqueratoma en grupos
    5. Tratamiento con agalsidasa alfa o agalsidasa beta durante un mínimo de 3 años y con una dosis estable (>80 % de la dosis autorizada/kg) durante un mínimo de 6 meses
    6. TFGe ≥30 ml/min/1,73 m2 mediante la ecuación CKD-EPI en la visita de selección
    7. Disponibilidad de un mínimo de 3 evaluaciones históricas de la creatinina sérica desde el inicio del tratamiento con agalsidasa alfa o agalsidasa beta 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 de alta eficacia aceptado desde el punto de vista médico. Estos incluyen una anticoncepción hormonal combinada (con estrógenos o progesterona) asociada a una inhibición de la ovulación (oral, intravaginal o transdérmica), anticoncepción hormonal basada solo en la progesterona asociada a una inhibición de la ovulación (oral, inyectable o implantable), dispositivos intrauterinos (DIU), sistema de liberación hormonal intrauterina (SIU), oclusión bilateral de trompas, pareja vasectomizada o abstinencia sexual
    9. Pacientes cuyo estado clínico, en opinión del investigador, es adecuado para el TSE cada 4 semanas.
    E.4Principal exclusion criteria
    1. History of anaphylaxis or Type 1 hypersensitivity reaction to agalsidase alfa or agalsidase beta
    2. History of renal dialysis or transplantation
    3. Linear negative slope of eGFR of ≥ 2 mL/min/1.73 m2 based on at least 4 serum creatinine values over approximately 2 years (including the value obtained at the screening visit)
    4. 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 post renal obstructive nephropathy)
    5. Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening
    6. Urine protein to creatinine ratio (UPCR) at screening > 0.5 g/g or mg/mg or 500 mg/g and not treated with an ACE inhibitor or ARB
    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. Cerebrovascular event (stroke, transient ischemic attack) in the 6-month period before screening
    10. 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 o la agalsidasa beta
    2. Antecedentes de diálisis o trasplante renal
    3. Pendiente lineal negativa de la TFGe de ≥2 ml/min/1,73 m2 en función de al menos 4 valores de creatinina en suero en aproximadamente 2 años (incluido el valor obtenido en la visita de selección)
    4. 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)
    5. 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
    6. Cociente proteínas/creatinina en orina (UPCr) >0,5 g/g o mg/mg 500 mg/g, y no tratado con un inhibidor de la ECA ni un BRA
    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. Acontecimiento cerebrovascular (ictus, accidente isquémico transitorio) en el periodo de 6 meses previo a la selección
    10. 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
    - Electrocardiogram
    - Treatment-emergent adverse events
    - Ability to taper off infusion premedication
    - Requirement for use of premedication overall to manage infusion reactions
    - Treatment-induced anti-pegunigalsidase alfa antibodies

    EFFICACY EXPLORATORY ENDPOINTS:
    - Estimated glomerular filtration rate (eGFRCKD-EPI)
    - Left Ventricular Mass Index (g/m2) by echocardiogram
    - 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 SEGURIDAD

    Cambios desde el inicio en:
    • Análisis clínicos
    • Exploración física
    • Evaluación de la zona de inyección
    • Electrocardiograma
    • Acontecimientos adversos surgidos durante el tratamiento
    • Capacidad de disminuir gradualmente la premedicación de la infusión
    • Requisito de uso de la premedicación en general para tratar las reacciones a la infusión
    • Anticuerpos contra la pegunigalsidasa alfa inducidos por el tratamiento
    CRITERIOS DE VALORACIÓN EXPLORATORIOS DE LA EFICACIA:
    • Tasa de filtración glomerular estimada (TFGeCKD-EPI)
    • Índice de masa del ventrículo izquierdo (g/m2) mediante ecocardiograma
    • Lyso-Gb3 en plasma
    • 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)
    • Índice de calidad de vida EQ-5D-5L
    E.5.1.1Timepoint(s) of evaluation of this end point
    As per protocol
    Acorde a protocolo
    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 No
    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) 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.8.3 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 EEA6
    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
    Belgium
    Canada
    Germany
    Netherlands
    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
    LVLP
    Última visita 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 months9
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months9
    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: 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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 15
    F.4.2.2In the whole clinical trial 30
    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)
    There will be the option to be enrolled in an open-label extension study upon completion of the study.
    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 Decision2017-12-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-12-15
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-08-01
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