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    Summary
    EudraCT Number:2018-001947-30
    Sponsor's Protocol Code Number:PB-102-F51
    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:2021-09-01
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2018-001947-30
    A.3Full title of the trial
    Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks 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) 2 mg/kg somministrato mediante infusione endovenosa ogni 4 settimane in pazienti con 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) Administered by Intravenous Infusion Every 4 Weeks in Patients With Fabry Disease
    Studio per la valutazione di sicuezza ed efficacia di di pegunigalsidasi alfa (PRX-102) somministrato mediante infusione endovenosa ogni 4 settimane in pazienti con malattia di Fabry
    A.3.2Name or abbreviated title of the trial where available
    PB-102-F51
    PB-102-F51
    A.4.1Sponsor's protocol code numberPB-102-F51
    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 number00009028
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee 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 (carenza di a-galattosidasi A)
    E.1.1.1Medical condition in easily understood language
    Fabry disease (a-galactosidase A deficiency)
    Malattia di Fabry (carenza 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 and efficacy parameters of 2 mg/kg pegunigalsidase alfa every 4 weeks in adult Fabry patients.
    Valutare la sicurezza e l’efficacia a lungo termine di pegunigalsidasi alfa (PRX-102) 2 mg/kg somministrato mediante infusione endovenosa ogni 4 settimane in pazienti con malattia di Fabry
    E.2.2Secondary objectives of the trial
    N/A
    Non Applicabile
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Completion of study PB-102-F50.
    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 ccepted, 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.
    Principali criteri di inclusione:
    i soggetti eleggibili devono soddisfare i seguenti criteri di inclusione:
    1. Completamento dello studio PB-102-F50.
    2. Firma da parte del paziente del consenso informato.
    3. Le pazienti di sesso femminile e i pazienti di sesso maschile le cui compagne siano potenzialmente fertili acconsentono a usare un metodo contraccettivo altamente efficace e accettabile dal punto di vista medico. Questi metodi includono la contraccezione ormonale combinata (contenente estrogeni o progestinici) associata all’inibizione dell’ovulazione (orale, intravaginale o transdermica), contraccezione ormonale a base di solo progesterone associata all’inibizione dell’ovulazione (orale, iniettabile o impiantabile), dispositivo intrauterino (IUD), sistema intrauterino a rilascio di ormoni (IUS), occlusione tubarica bilaterale, compagno vasectomizzato o astinenza sessuale.
    E.4Principal exclusion criteria
    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.
    Principali criteri di esclusione:
    i seguenti criteri escludono l’arruolamento del paziente nello studio: presenza di qualsiasi condizione medica, emotiva, comportamentale o psicologica che, a giudizio dello sperimentatore e/o del responsabile medico, interferirebbe con la conformità del paziente ai requisiti dello studio.
    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
    *Requirement for use of pre-medication overall to manage infusion reactions
    *Treatment-induced anti-pegunigalsidase alfa antibodies
    ENDPOINT DI SICUREZZA:
    Variazioni rispetto al basale in:
    • esami clinici di laboratorio
    • esame obiettivo
    • valutazione del sito di iniezione
    • elettrocardiogramma
    • eventi avversi emergenti dal trattamento
    • necessità di utilizzare complessivamente una pre-medicazione per gestire le reazioni all’infusione
    • anticorpi anti-pegunigalsidasi alfa indotti dal trattamento
    E.5.1.1Timepoint(s) of evaluation of this end point
    Results of safety evaluations (including TEAEs, injection site reactions, physical examinations, ECG, laboratory analyses, and anti-drug antibodies) will be summarized and described. No formal statistical analyses will be performed in this study. 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.
    I Risultati delle valutazioni di sicurezza (compresi i TEAE, le reazioni al sito di iniezione, esami fisici, ECG, analisi di laboratorio e anti-drug antibodies) saranno riassunti e descritti. Nessuna
    analisi statistica formale sarà eseguita in questo studio. Gli endpoint dello studio saranno valutati da varie analisi riassuntive per visita di studio e per cambiamento dai dati di riferimento per ciascun endpoint di efficacia. Gli endpoint di Sicurezza ed efficacia saranno confrontati con la baseline usando le statistiche riassuntive. I Dati non saranno analizzaio da statistiche inferenziali.
    E.5.2Secondary end point(s)
    EFFICACY EXPLORATORY ENDPOINTS:
    *Estimated glomerular filtration rate (eGFRCKD-EPI)
    *Left Ventricular Mass Index (g/m2) by echocardiogram
    * Plasma Gb3 concentration
    *Plasma Lyso-Gb3 concentration
    *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
    ENDPOINT DI EFFICACIA ESPLORATIVI:
    • velocità di filtrazione glomerulare stimata (eGFRmediante CKD-EPI)
    • indice di massa ventricolare sinistra (g/m2) mediante ecocardiogramma
    - concentrazione plasmatica Gb-3
    • concentrazione plasmatica di liso-Gb3
    • rapporto proteine/creatinina nel test su campione estemporaneo di urina
    • 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)
    E.5.2.1Timepoint(s) of evaluation of this end point
    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.
    Gli endpoint dello studio saranno valutati con diverse 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.
    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 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 Yes
    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 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 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 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
    Australia
    Canada
    Taiwan
    Turkey
    United States
    Belgium
    Denmark
    Finland
    France
    Italy
    Netherlands
    Norway
    Spain
    Sweden
    United Kingdom
    Czechia
    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 years3
    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: 40
    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 20
    F.4.2.2In the whole clinical trial 40
    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
    Non Applicabile
    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 Decision2019-10-31
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-10-09
    P. End of Trial
    P.End of Trial StatusOngoing
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