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    Summary
    EudraCT Number:2013-002554-78
    Sponsor's Protocol Code Number:PB-102-F02
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-09-09
    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 ConcernedUK - MHRA
    A.2EudraCT number2013-002554-78
    A.3Full title of the trial
    An Extension of Phase 1/2, Open-Label, Dose Ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 38 Weeks (9 Months) to Adult Fabry Patients
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Extension of the PB-102-F01 study: A clinical study in patients with Fabry disease to assess the safety, tolerability, and the body processing of the medication PRX102, which will be given as an infusion.
    A.3.2Name or abbreviated title of the trial where available
    PB-102-F02: Extension of the PB-102-F01 study (PRX-102 for ERT in Fabry Disease)
    A.4.1Sponsor's protocol code numberPB-102-F02
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01769001
    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 organisationCATO Europe GmbH
    B.5.2Functional name of contact pointRainer Schuckelt
    B.5.3 Address:
    B.5.3.1Street AddressHertzstr. 7
    B.5.3.2Town/ cityKoeln
    B.5.3.3Post code50859
    B.5.3.4CountryGermany
    B.5.4Telephone number00492234379440
    B.5.5Fax number004922343794425
    B.5.6E-mailr.schuckelt@cato-europe.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 namePRX-102
    D.3.2Product code PRX-102
    D.3.4Pharmaceutical form 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 INNPRX-102
    D.3.9.1CAS number 1333358-30-7
    D.3.9.2Current sponsor codePRX-102
    D.3.9.4EV Substance CodeAS1
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Fabry disease
    E.1.1.1Medical condition in easily understood language
    Fabry disease is an inherited condition caused by a deficiency of an enzyme, leading to a range of systemic symptoms (potentially life-threatening), such as kidney failure, heart attacks and strokes.
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.0
    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, pharmacokinetics and exploratory efficacy parameters of PRX-102 in adult Fabry patients who have successfully completed treatment with PRX-102 in the core study PB-102-F01, and are continuing to receive treatment at the dose assigned to each patient in the PB-102-F01 study. This extension study may provide important additional long term information on safety, tolerability and clinical outcome in patients treated with different doses of PRX102.
    E.2.2Secondary objectives of the trial
    All exploratory endpoints that were evaluated during the Phase 1/2 study PB-102-F01 will continue to be assessed in the extension protocol (study PB-102-F02). These include:

    Globotriaosylceramide (Gb3) concentration in kidney (assessed histologically in kidney biopsy)
    Gb3 concentration in skin (assessed histologically in skin punch biopsy)
    Gb3 concentrations in plasma and urine sediment
    Globotriaosylsphingosine (Lyso-Gb3) concentration in plasma
    Assessment of gastrointestinal symptoms
    Kidney functions (eGFR and proteinuria)
    Short Form Brief Pain Inventory (BPI)
    Left ventricular mass (LVM) and myocardial fibrosis assessment by cardiac MRI
    Cardiac function assessment by echocardiography and stress test
    Cerebrovascular disease assessment (clinical and MRI evaluation)
    Mainz Severity Score Index (MSSI)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Eligible patients must fulfill the following inclusion criteria:
    1. Completion of Phase 1/2 study PB-102-F01
    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 termination of treatment.
    E.4Principal exclusion criteria
    The presence of any of the following excludes a patient from study enrollment:
    1. Pregnant or nursing
    2. 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
    E.5 End points
    E.5.1Primary end point(s)
    The main outcome will be evaluation of safety in PRX-102 treated subjects at each dose. Safety will be assessed by the frequency, severity, and duration of treatment-emergent adverse events (TEAEs), including clinically significant laboratory abnormalities, ECG changes from baseline, physical examination findings and assessment of the injection site reactions after administration of the study drug. Also Anti-PRX-102 antibodies will be assessed.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Clinical lab tests, Physical Examination, ECG will be assessed at Screening, Visit 7 (~ 6 months after treatment), 13 and 20 (~ 12 months after treatment).

    Adverse events will be assessed at every visit.

    Injection site reactions will be assessed at every visit up to visit 20.

    Anti-PRX-102 antibodies will be assessed at Screening, Visit 2, 7, 11, 15, 20 and 2 months after visit 20.
    E.5.2Secondary end point(s)
    PHARMACOKINETIC ENDPOINTS:
    The following PK parameters will be derived from the plasma concentration versus time profiles to determine the profile of the study drug: Cmax, t1/2, Tmax, AUC0t, and AUC0∞.

    EXPLORATORY EFFICACY ENDPOINTS:
    • Globotriaosylceramide (Gb3) concentration in kidney (assessed histologically in kidney biopsy)
    • Gb3 concentration in skin (assessed histologically in skin punch biopsy)
    • Gb3 concentrations in plasma and urine sediment
    • Globotriaosylsphingosine (Lyso-Gb3) concentration in plasma
    • Assessment of gastrointestinal symptoms
    • Kidney functions (eGFR and proteinuria)
    • Short Form Brief Pain Inventory (BPI)
    • Left ventricular mass (LVM) and myocardial fibrosis assessment by cardiac MRI
    • Cardiac function assessment (echocardiography and stress test)
    • Cerebrovascular disease assessment (clinical and MRI evaluation)
    • Mainz Severity Score Index (MSSI)
    E.5.2.1Timepoint(s) of evaluation of this end point
    EXPLORATORY:
    Visit 7: Kidney biopsy & skin punch biopsy

    Screening, visit 7, 13 & 20 (after total treatment of 12 months): Gb3 concentrations in plasma and urine sediment, Globotriaosylsphingosine (Lyso-Gb3) concentration, Assessment of gastrointestinal symptoms, Kidney functions,Short Form Brief Pain Inventory

    Visit 7 & 20: Left ventricular mass (LVM) and myocardial fibrosis, Cardiac function
    Mainz Severity Score Index

    Visit 20: Cerebrovascular disease assessment

    PHARMACOKINETIC: samples will be collected at the following time points: pre-infusion (baseline); 1±0.5 hour after the beginning of the infusion; at the end of the infusion; 1±0.25, 4±0.25, 8±0.25, 24±0.5, 48±3, 72±3, 96±3 hours and 2 weeks±3 days post-infusion at the 3 and 9 month visit
    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 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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Extension of the study PB-102-F01 in which first administration to humans took place
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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.8.3 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 No
    E.8.5.1Number of sites anticipated in the EEA1
    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
    Argentina
    Australia
    Chile
    Israel
    Paraguay
    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
    LVLS
    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 months7
    E.8.9.1In the Member State concerned days30
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months1
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1Number of subjects for this age range: 0
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 0
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 17
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 4
    F.4.2.2In the whole clinical trial 18
    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)
    Subjects who tolerate the infusions well and complete the study are eligible to enroll in the next extension study.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2013-07-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-08-29
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-02-17
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