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    Summary
    EudraCT Number:2017-001528-23
    Sponsor's Protocol Code Number:PB-102-F50
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-11-10
    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 ConcernedItaly - Italian Medicines Agency
    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)
    Studio switch-over, in aperto, di fase 3 per valutare la sicurezza, l¿efficacia e la farmacocinetica di pegunigalsidasi alfa (PRX-102) 2 mg/kg somministrato mediante infusione endovenosa ogni 4 settimane per 52 settimane in pazienti adulti con malattia di Fabry attualmente in trattamento con terapia di sostituzione enzimatica: Fabrazyme¿ (agalsidasi beta) o Replagal¿ (agalsidasi 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)
    Studio di fase 3 per la sicurezza, efficacia & PK di pegunigalsidasi alfa (PRX-102) 2 mg/kg somministrato mediante infusione endovenosa ogni 4 settimane in pazienti affetti da malattia di Fabry (BRIGHT)
    A.3.2Name or abbreviated title of the trial where available
    Phase 3 Study of the Safety, Efficacy & PK of pegunigalsidase alfa (PRX-102) 2 mg/kg IV Administered
    Studio di fase 3 per la sicurezza, efficacia & PK di pegunigalsidasi alfa (PRX-102) 2 mg/kg somminis
    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.5.6E-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.3.10 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 (deficienza di a-galattosidasi-A)
    E.1.1.1Medical condition in easily understood language
    Fabry disease
    Malattia di 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).
    valutare la sicurezza, l¿efficacia e la farmacocinetica di pegunigalsidasi alfa (PRX-102) in pazienti con malattia di Fabry attualmente in trattamento con ERT attualmente disponibile in commercio (agalsidasi alfa o agalsidasi beta).
    E.2.2Secondary objectives of the trial
    Not applicable
    non applicabile
    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. Età: 18-60 anni
    2. Diagnosi documentata di malattia di Fabry
    3. Soggetti di sesso maschile: attività dell’alfa galattosidasi leucocitica e/o plasmatica (valutata tramite saggio dell’attività) inferiore al limite inferiore dell’intervallo normale ai sensi degli intervalli di riferimento del laboratorio e una o più caratteristiche tipiche della malattia di Fabry
    a. Dolore neuropatico
    b. Cornea verticillata
    c. Angiocheratoma a grappolo
    4. Soggetti di sesso femminile: riscontri storici agli esami genetici compatibili con mutazioni di Fabry, o nell’eventualità di nuove mutazioni, un parente di primo grado di sesso maschile con malattia di Fabry, e una o più caratteristiche tipiche della malattia di Fabry
    a. Dolore neuropatico
    b. Cornea verticillata
    c. Angiocheratoma a grappolo
    5. Trattamento con agalsidasi alfa o agalsidasi beta per almeno 3 anni e con dose stabile (>80% della dose/kg riportata in etichetta) per almeno gli ultimi 6 mesi
    6. Velocità stimata di filtrazione glomerulare (eGFR) alla visita di screening =30 ml/min/1,73 m2 misurata mediante l’equazione della Collaborazione per l’epidemiologia della malattia renale cronica (CKD-EPI)
    7. Disponibilità di almeno 3 valutazioni storiche della creatinina sierica dall’avvio del trattamento con agalsidasi alfa o agalsidasi beta, e che non siano più datate di 2 anni
    8. 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
    9. Pazienti la cui condizione clinica, a giudizio dello sperimentatore, sia idonea al trattamento con ERT ogni 4 settimane.
    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. Anamnesi di anafilassi o reazione da ipersensibilità di Tipo 1 all’agalsidasi alfa o agalsidasi beta
    2. Anamnesi di dialisi o trapianto renale
    3. Pendenza lineare negativa dell’eGFR =2 ml/min/1,73 m2 sulla base di almeno 4 valori di creatinina sierica nell’arco di circa 2 anni (compreso il valore ottenuto alla visita di screening)
    4. Anamnesi di lesione renale acuta nei 12 mesi precedenti lo screening, comprese malattie renali specifiche (ad es., nefrite interstiziale acuta, malattie renali glomerulari e vasculitiche acute), condizioni non specifiche (ad es., ischemia, lesione tossica), nonché patologia extra-renale (ad es., azotemia pre-renale e nefropatia ostruttiva acuta post-renale)
    5. Terapia con inibitore dell’enzima di conversione dell’angiotensina (ACE) o con bloccante del recettore dell’angiotensina (ARB) avviata o la cui dose sia stata cambiata nelle 4 settimane precedenti lo screening
    6. Rapporto proteine/creatinina nelle urine (UPCR) allo screening >0,5 g/g o mg/mg oppure 500 mg/g e non trattato con un inibitore dell’ACE o un ARB
    7. Soggetti di sesso femminile in stato di gravidanza, che prevedono di iniziare una gravidanza durante lo studio o in allattamento
    8. Evento cardiovascolare (infarto miocardico, angina instabile) nel periodo di 6 mesi precedente lo screening
    9. Evento cerebrovascolare (ictus, attacco ischemico transitorio) nel periodo di 6 mesi precedente lo screening
    10. 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 - 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
    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 - Capacità di ridurre la premedicazione tramite infusione - Necessità di utilizzare complessivamente una premedicazione per gestire le reazioni all’infusione - Anticorpi anti-pegunigalsidasi alfa indotti dal trattamento - ENDPOINT DI EFFICACIA ESPLORATIVI: - Velocità di filtrazione glomerulare stimata (eGFRmediante CKD-EPI) - Indice di massa ventricolare sinistra (g/m2) mediante ecocardiogramma - Livelli plasmatici di liso-Gb3 - Livelli plasmatici di Gb3 -Livelli urinari 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.5.1.1Timepoint(s) of evaluation of this end point
    Come da protocollo
    As per protocol
    E.5.2Secondary end point(s)
    Not applicable
    Not applicable
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not applicable
    Not applicable
    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 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) 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 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
    Austria
    Belgium
    Canada
    Czechia
    Denmark
    Germany
    Italy
    Netherlands
    Norway
    Spain
    Taiwan
    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
    LVLP
    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 days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months9
    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: 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.
    Ci sar¿ la possibilit¿ di essere arruolati in uno studio di estensione open-label al completamento dello studio
    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 Decision2018-07-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-05-23
    P. End of Trial
    P.End of Trial StatusCompleted
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