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    EudraCT Number:2018-001148-67
    Sponsor's Protocol Code Number:PB-102-F60
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-10-21
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    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
    Studio di estensione in aperto per valutare la sicurezza e l’efficacia a lungo termine di pegunigalsidasi alfa (PRX-102) in pazienti affetti da malattia di 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
    Studio per la valutazione di sicurezza ed efficacia di Pegunigalsidase Alfa (PRX-102) in pazienti con patologia di Fabry
    A.3.2Name or abbreviated title of the trial where available
    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.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.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
    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.3.9.1CAS 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. cell therapy medicinal product Information not present in EudraCT
    D. therapy medical product Information not present in EudraCT
    D. Engineered Product Information not present in EudraCT
    D. ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D. on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 (a-galactosidase A deficiency)
    Malattia di Fabry (deficienza di a- galattosidasi A)
    E.1.1.1Medical condition in easily understood language
    Fabry disease (a-galactosidase A deficiency)
    Malattia di Fabry (deficienza di a- galattosidasi A)
    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, or completed at least 48 months in study PB-102-F03.
    Valutare i parametri continui di sicurezza, tollerabilità ed efficacia di pegunigalsidasi alfa (PRX-102) 1 mg/kg ogni due settimane in pazienti adulti affetti da malattia di Fabry che abbiano completato con successo lo studio PB-102-F20 o PB-102-F30 oppure abbiano completato almeno 48 mesi dello studio PB-102-F03
    E.2.2Secondary objectives of the trial
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Completion of study PB-102-F20 or PB-102-F30, or completed at least 48 months in study PB-102-F03.
    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. Completamento dello studio PB-102-F20 o PB-102-F30, oppure completamento di almeno 48 mesi dello studio PB-102-F03.
    2. Firma da parte del paziente del consenso informato
    3. Consenso delle pazienti e dei pazienti con compagne in età fertile a utilizzare un metodo contraccettivo accettabile dal punto di vista medico, ad esclusione del metodo Ogino-Knaus. I metodi contraccettivi accettabili comprendono prodotti ormonali, dispositivo intrauterino o preservativi maschili o femminili. La contraccezione deve proseguire per 1 mese dopo la conclusione del trattamento.
    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.
    Presenza di qualsiasi condizione medica, emotiva, comportamentale o psicologica che, a giudizio dello sperimentatore e/o del Dirigente Medico, potrebbe interferire con la conformità del paziente ai requisiti dello studio.
    E.5 End points
    E.5.1Primary end point(s)
    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
    Variazioni rispetto al basale in:
    *Analisi cliniche di laboratorio
    *Esame obiettivo
    *Valutazione della sede di iniezione
    *Elettrocardiogramma (ECG)
    *Eventi avversi emergenti dal trattamento (TEAE)
    *Possibilità di ridurre la premedicazione all’infusione all’avvio dello studio
    *Necessità di utilizzare complessivamente una premedicazione per gestire le reazioni all’infusione
    *Anticorpi anti-pegunigalsidasi alfa emergenti dal trattamento
    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.
    L’analisi di sicurezza includerà pazienti che hanno ricevuto almeno una dose di farmaco. Saranno descritti i risultati delle valutazioni di sicurezza compresi TEAE reazioni nella sede di iniezione esami obiettivi ECG analisi di laboratorio anticorpi anti-farmaco. Non verranno eseguite analisi statistiche formali Gli endpoint saranno valutati con analisi riassuntive per visita dello studio e variazione rispetto ai dati al basale per ciascun endpoint di efficacia. Gli endpoint di efficacia e sicurezza saranno confrontati al basale tramite statistiche descrittive I dati non saranno analizzati tramite statistiche inferenziali Durante lo studio potrebbe essere eseguita un’analisi ad interim per finalità amministrative con statistiche descrittive per riassumere parametri di efficacia e sicurezza
    E.5.2Secondary end point(s)
    *Estimated glomerular filtration rate (eGFRCKD-EPI)
    *Left Ventricular Mass Index (g/m2) by magnetic resonance imaging (MRI)
    *Plasma Lyso-Gb3
    *Plasma Gb3 concentration
    *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)
    *Fabry disease clinical events
    *Velocità di filtrazione glomerulare stimata (eGFR mediante CKD-EPI)
    *Indice di massa ventricolare sinistra (g/m2) misurato mediante risonanza magnetica (RM)
    *Livelli plasmatici di liso-Gb3
    *Concentrazione plasmatica di Gb3
    *Rapporto proteine/creatinina nell’analisi su campione estemporaneo di urina (UPCR)
    *Frequenza dell’uso di farmaci analgesici
    *Tolleranza all’esercizio (test da sforzo)
    *Breve inventario per la valutazione del dolore (BPI) in forma abbreviata
    *Indice del punteggio di gravità di Mainz (MSSI)
    *Questionario europeo sulla qualità della vita a 5 dimensioni e 5 livelli (EQ-5D-5L)
    *Eventi clinici di malattia di Fabry
    E.5.2.1Timepoint(s) of evaluation of this end point
    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. 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 Yes
    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 Yes
    E. description
    E.8.2.4Number of treatment arms in the trial1
    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.8.4.1Number 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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Czech Republic
    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
    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)
    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 Decision2020-07-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-11-11
    P. End of Trial
    P.End of Trial StatusOngoing
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