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    EudraCT Number:2012-002365-35
    Sponsor's Protocol Code Number:GC1008FSGS03110
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2013-03-20
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2012-002365-35
    A.3Full title of the trial
    A Phase 2, Multicenter, Double-Blind, Parallel Dosing, Randomized Study of Fresolimumab or Placebo in Patients with Steroid-Resistant Primary Focal Segmental Glomerulosclerosis
    Studio di fase II multicentrico, randomizzato, in doppio cieco, a gruppi dose paralleli con fresolimumab o placebo in pazienti affetti da glomerulosclerosi focale segmentaria primitiva resistenti agli steroidi
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of Fresolimumab in Patients With Steroid-Resistant Primary Focal Segmental Glomerulosclerosis (FSGS)
    Studio di Frsolimumab in pazienti affetti da glomerulosclerosi focale segmentaria primitiva resistenti agli steroidi
    A.4.1Sponsor's protocol code numberGC1008FSGS03110
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01665391
    A.5.4Other Identifiers
    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 SponsorGenzyme Corporation
    B.1.3.4CountryUnited States
    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 supportGenzyme Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationTo be provided locally
    B.5.2Functional name of contact point
    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/3/11/882
    D.3 Description of the IMP
    D.3.1Product nameFresolimumab
    D.3.2Product code GC1008
    D.3.4Pharmaceutical form Powder 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 INNFresolimumab
    D.3.9.2Current sponsor codeGC1008
    D.3.9.3Other descriptive nameGZ402669
    D.3.9.4EV Substance CodeSUB31224
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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 No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    D.8.3Pharmaceutical form of the placeboPowder for solution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Focal segmental glomerulosclerosis
    Glomerulosclerosi focale segmentaria
    E.1.1.1Medical condition in easily understood language
    Focal segmental glomerulosclerosis
    Glomerulosclerosi focale segmentaria
    E.1.1.2Therapeutic area Not possible to specify
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 15.1
    E.1.2Level PT
    E.1.2Classification code 10067757
    E.1.2Term Focal segmental glomerulosclerosis
    E.1.2System Organ Class 10038359 - Renal and urinary disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objectives of this trial are as follows:
    • To compare the achievement of a partial remission (PR) or complete remission (CR) in urinary protein: creatinine ratio (Up/c ratio) in patients treated with fresolimumab versus placebo
    • To compare the safety profile of patients treated with fresolimumab versus placebo
    Gli obiettivi primari dello studio sono i seguenti:
    - Confrontare il raggiungimento di una remissione parziale (PR) o completa (CR) delle proteine del sistema urinario: rapporto di creatinina (rapporto Up/c) in pazienti trattati con fresolimumab versus placebo;
    - Confrontare il profilo di sicurezza dei pazienti trattati con fresolimumab versus placebo
    E.2.2Secondary objectives of the trial
    The secondary objectives are as follows:
    • To compare the reduction in proteinuria in patients treated with fresolimumab versus placebo
    • To evaluate fresolimumab dose-dependent reduction in proteinuria
    • To compare the change in renal function (estimated glomerular filtration rate [eGFR]) in patients treated with fresolimumab versus placebo
    • To evaluate the multiple-dose pharmacokinetics of fresolimumab
    Gli obiettivi secondari sono i seguenti:
    - Confrontare la riduzione della proteinuria nei pazienti trattati con fresolimumab versus placebo;
    - Valutare la riduzione dose-dipendente di fresolimumab nella proteinuria;
    - Confrontare il cambiamento della funzionalità renale (velocità di filtrazione glomerulare stimata [eGFR]) nei pazienti trattati con fresolimumab versus placebo;
    - Valutare la farmacocinetica a dose multipla di fresolimumab
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Ages Eligible for Study: 18 Years and older
    Genders Eligible for Study: Both

    Inclusion Criteria:
    • The patient's renal biopsy is consistent with the diagnosis of primary Focal Segmental Glomerulosclerosis (FSGS) including all histological subtypes.
    • The patient has an eGFR ≥ 30 mL/min/1.73 m2
    • The patient has a urinary total protein:creatinine ratio ≥ 3 mg protein/mg creatinine
    • In the opinion of the Investigator, the patient has steroid-resistant FSGS. The patient must have been treated for FSGS with a course of high-dose steroid therapy for a minimum of 4 weeks
    • The patient has been treated with an ACEi and/or ARB at a stable dose for a minimum of 4 weeks prior to Visit 2 (treatment start)
    Età eleggibile per lo studio: 18 anni e oltre
    Generi eleggibili per lo studio: entrambi

    Criteri di inclusione:
    - La biopsia renale del paziente è coerente con la diagnosi di FSGS primitiva, compresi i sottotipi istologici;
    - Il paziente ha una eGFR  30 ml/min/1,73 m2;
    - Il paziente presenta un rapporto proteine urinarie totali: creatinina ≥ 3 mg proteina/mg creatinina in almeno 1 dei campioni di urina raccolti durante il periodo di screening e un apporto proteine urinarie totali: creatinina ≥ 2 mg proteina/mg creatinina in 2 campioni raccolti urante il periodo di screening;
    - Secondo l’opinione dello sperimentatore, il paziente è affetto da FSGS resistente agli steroidi. I pazienti devono essere stati curati per la FSGS con una terapia ad alte dosi di steroidi per almeno 4 settimane;
    - ll paziente è stato trattato con un ACE inibitore e/o un ARB ad un dosaggio stabile per almeno 4 settimane prima della Visita 2 (avvio del trattamento).
    E.4Principal exclusion criteria
    • The patient has FSGS which in the Investigator's opinion is secondary to another condition
    • The patient has been taking prednisone at a dose > 10 mg/day (or equivalent dose of an alternative glucocorticoid) within 4 weeks prior to Visit 1 (Screening Visit).
    • The patient has received any other systemically administered immunosuppressive drugs (other than glucocorticoids) within 8 weeks prior to Visit 1.
    • The patient has received rituximab within 6 months prior to Visit 1.
    • The patient has a history of organ transplantation.
    - Il paziente è affetto da FSGS che secondo l’opinione dello sperimentatore è secondaria ad un’altra condizione;
    - Il paziente ha assunto prednisone ad una dose > 10 mg/giorno (o una dose equivalente di un altro glucocorticoide) nelle 4 settimane prima della Visita 1 (visita di selezione);
    - Il paziente ha ricevuto qualsiasi altro farmaco immunosoppressore (diverso dai glucocorticoidi) somministrato per via sistemica nelle 8 settimane prima della Visita 1;
    - Il paziente ha ricevuto rituximab nei 6 mesi prima della Visita 1;
    - Il paziente ha ricevuto un trapianto di organi
    E.5 End points
    E.5.1Primary end point(s)
    1) Percentage of patients achieving partial remission (PR) or complete remission (CR) in urinary protein:creatinine ratio (Up/c ratio)
    2) Number of patients reporting adverse events (AEs), serious adverse events (SAEs), and medical events of interest (MEOIs)
    1) percentuale di pazienti che raggiungono la remissione parziale (RP) o la remissione completa (RC) nella proteina urinaria: rapporto Up/c
    2) numero di pazienti che riportano eventi avversi (EA), eventi avversi seri (EAS) e eventi clinici di interesse
    E.5.1.1Timepoint(s) of evaluation of this end point
    1/2) Up to Day 112
    1/2) Fino al giorno 112
    E.5.2Secondary end point(s)
    1) Percentage of patients achieving CR in Up/c ratio
    2) Percentage of patients achieving PR in Up/c ratio
    3) Change from baseline in Up/c ratio and urinary protein excretion rate
    4) Time to first PR or CR
    5) Change from baseline in eGFR
    6) Percentage of patients achieving PR or CR with stable eGFR
    7) Pharmacokinetics as measured by Cmax, tmax, AUC 0-last, C trough, t 1/2z
    1) Percentuale di pazienti che raggiungo remissione completa nel Up/c ratio;
    2) Percentuale di pazienti che raggiungono la remissione parziale nel rapporto Up/c ;
    3) Cambiamenti rispetto al basale nel Up/c ratio e nel valore di escrezione della proteina urinaria;
    4) tempo alla prima remissione parziale o completa
    5) Cambiamenti rispetto al basale nell'eGFR;
    6) Percentuale di pazienti che raggiungono remissione parziale o completa con eGFR stabile;
    7) Faramcocinetica misurata in base a Cmax, tmax, AUC-0-last e C trough, t 1/2z
    E.5.2.1Timepoint(s) of evaluation of this end point
    1 to 6) Up to Day 112
    7) Up to Day 252
    Dall'endpoint 1 al 6: Fino al giorno 112
    per l'endpoint 7: Fino al giorno 252
    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 Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 trial3
    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 No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA18
    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
    United States
    E.8.7Trial has a data monitoring committee Yes
    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
    Ultima visita dell'ultimo paziente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months24
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months24
    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: 120
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 No
    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 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 state
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 36
    F.4.2.2In the whole clinical trial 125
    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 Decision2013-05-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-05-22
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2015-01-27
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