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    EudraCT Number:2016-001028-80
    Sponsor's Protocol Code Number:ACT14604
    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:2020-11-05
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2016-001028-80
    A.3Full title of the trial
    Efficacy and safety of SAR156597 in the treatment of diffuse cutaneous Systemic Sclerosis (dcSSc): A randomized, double-blind, placebo-controlled, 24-week, proof of concept study
    Efficacia e sicurezza di SAR156597 nel trattamento della sclerosi sistemica cutanea diffusa (dcSSc): studio "proof of concept" della durata di 24 settimane, randomizzato, in doppio cieco, controllato verso placebo
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Effectiveness and Safety of SAR156597 in Treating Diffuse Systemic Sclerosis
    Efficacia e sicurezza di SAR 156597 nel trattamento della sclerosi sistemica diffusa
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberACT14604
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1179-4690
    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.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 supportSANOFI-AVENTIS RECHERCHE ET DEVELOPPEMENT
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSANOFI S.p.A.
    B.5.2Functional name of contact pointCONTACT POINT
    B.5.3 Address:
    B.5.3.1Street AddressVIALE BODIO, 37/ B
    B.5.3.2Town/ cityMILANO
    B.5.3.3Post code20158
    B.5.4Telephone number800226343
    B.5.5Fax number0239394168
    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 nameNA
    D.3.2Product code SAR156597
    D.3.4Pharmaceutical form Powder for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNnon applicabile
    D.3.9.2Current sponsor codeSAR156597
    D.3.9.4EV Substance CodeSUB33159
    D.3.10 Strength
    D.3.10.1Concentration unit mg/g milligram(s)/gram
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number100
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboPowder for solution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Systemic sclerosis
    Sclerosi sistemica
    E.1.1.1Medical condition in easily understood language
    Systemic sclerosis
    Sclerosi sistemica
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10042953
    E.1.2Term Systemic sclerosis
    E.1.2System Organ Class 100000004859
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate, in comparison with placebo, the efficacy of SAR156597 administered subcutaneously on skin fibrosis in patients with dcSSc.
    Valutare, rispetto a placebo, l¿efficacia di SAR156597,somministrato per via sottocutanea sulla fibrosi cutanea di pazienti affetti da sclerosi sistemica cutanea diffusa (dcSSc).
    E.2.2Secondary objectives of the trial
    -To evaluate the efficacy of SAR156597 compared to placebo on
    physical/functional disability in patients with dcSSc.
    -To evaluate the efficacy of SAR156597 compared to placebo on
    respiratory function in patients with dcSSc.
    -To evaluate the safety profile of SAR156597 compared to placebo in
    patients with dcSSc.
    -To evaluate the potential for immunogenicity (anti-drug antibodies
    [ADA] response) of SAR156597 in patients with dcSSc.
    -To evaluate the pharmacokinetics (PK) (trough plasma concentrations)
    of SAR156597
    administered subcutaneously.
    - Valutare l¿efficacia di SAR156597 rispetto a placebo sulla disabilit¿ fisica/funzionale in pazienti affetti da dcSSc.
    - Valutare l¿efficacia di SAR156597 rispetto a placebo sulla funzionalit¿ respiratoria in pazienti affetti da dcSSc.
    - Valutare il profilo di sicurezza di SAR156597 rispetto a placebo in pazienti affetti da dcSSc.
    - Valutare il potenziale di SAR156597 per lo sviluppo di una risposta immunogenica mediata da anticorpi anti-farmaco (ADA) in pazienti affetti da dcSSc.
    - Valutare la farmacocinetica (PK) attraverso le concentrazioni plasmatiche di SAR156597 somministrato per via sottocutanea.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Systemic Sclerosis according to the American College of Rheumatology/The European League against Rheumatism (ACR/EULAR) 2013 criteria.
    -Diffuse cutaneous form of SSc according to Leroy's criteria.
    -Able and willing to sign the written informed consent form with comprehension of its contents and comply with the requirements of the
    study protocol.
    Sclerosi sistemica secondo i criteri 2013 dell’American College of Rheumatology/European League against Rheumatism
    (ACR/EULAR) [Collegio americano di reumatologia/Lega Europea contro le malattie reumatiche];
    - Forma cutanea diffusa di SSc in base ai criteri di Leroy.
    - Soggetto in grado e disposto a firmare il Modulo di consenso informato scritto, previa comprensione dei suoi contenuti, e a rispettare i requisiti del protocollo dello studio.
    E.4Principal exclusion criteria
    Disease duration>36mths from time of 1nonRaynaud's phenomenon manifestation
    mRSS<10 or>35 at screen and baseline visits
    History of vasculitis,active or in remission
    Diag connective tissue diseases(other than SSc)or PM/Scl
    Pos HIV serology/known history of HIV active or in remission
    Abnormal hepB and/or hepC tests indicative active/chronic infection:Abnormal HepB tests:Pos HBsAg OR pos total HBcAb with neg hepB surface Ab(HBsAb)/pos total hepB core antibody with pos HBsAb and presence of hepB DNA(HBV DNA)
    Abnormal HepC tests:Pos antiHCV Ab and pos HCV RNA
    Pos or 2confirmed indeterminate QTF tests at screen(regardless of prior treatment status)
    Serious infection(eg pneumonia, pyelonephritis)within 4wk of screen,infection requiring hospitaliz/IV antibiotics within 4wk of screen or chronic bacterial infection(eg osteomyelitis)
    History of anaphylaxis to any biologic therapy
    Evidence of any clinically significant,severe/unstable,acute/chronically progressive,uncontrolled infection or medical condition(eg, cerebral,cardiac,pulmonary,renal,hepatic,gastrointestinal or neurologic other than SSc or SScILD)or previous,active/pending surgical disorder,or any condition that may affect pt safety in the judgment of the PI
    At screen, the% predicted FVC <=75% AND%predicted DLCO after Hgb correction is<=40%
    History of HF, LVEF <=45%, coronary artery disease,angina,myocardial infarction,ischemic/HCM
    Any prior history of malignancy or active malignancy including lymphoproliferative diseases(except successfullytreated carcinoma insitu of the cervix,nonmetastatic squamous cell/basal cell carcinoma of the skin)within 5yrs prior to baseline
    Ischemic ECG changes and/or other clinical significant ECG findings at screen
    High dose steroids(>10mg/day prednisolone equiv)or change in steroid dose within 4wk prior to screen or during the screen period or expected changes during the study course
    Previous treat with rituximab within 12mths prior to screen
    Previous treat with bone marrow transplantation,tot lymphoid irradiation/ablative ultrahigh dose cyclophosphamide
    Treat with high dose immunosuppressive drug(eg cyclophosphamide>1mg/kg oral/day or>750mg IV/mth;azathioprine>100mg/day;methotrexate>15mg/week;mycophenolate mofetil>2g/day)within 3mths of screen or change in dose within 4wk prior to baseline
    Treat with etanercept,cyclosporine A,IVIG,rapamycin,Dpenicillamine,tyrosine K inhibitors within 4wk of screen or antithymocyte globulin within 6mths of screen
    Treat with infliximab,certolizumab,golimumab,abatacept,or adalimumab,tocilizumab within 8wk of screen or anakinra within 1wk of screen
    Treat with any IMP within 1mth of screen,or 5halflives,if known Abnormal lab tests at screen:
    ALT or AST>2 times upper limit of ULN
    Hgb<11 g/100mL(male) &<10 g/100mL(female)
    Neutrophils<1500/mm^3(except<1000/mm3 for those of African descent)
    PLT<100 000/mm^3
    Lab parameters may be repeated once during the screen period if felt to be spurious or due to technical error in order to det eligibility
    Current history of substance and/or alcohol abuse
    Sanofi current employee/has an immediate family member(eg spouse,parents,child,sibling)Sanofi current employee
    Currently incarcerated or anticipated/scheduled to be incarcerated during the course of the study
    Any condition or circumstance that will preclude the pt from following and completing protocol requirements in the opinion of the PI
    Pregnant or breastfeeding woman
    Women who are of childbearing potential not protected by highlyeffective contraceptive method(s) of birth control as(defined in the ICF form and/or Appendix G for UK)and/or who are unwilling or unable to be tested for pregnancy
    Women of childbearing potential must have a confirmed neg pregtest at screen and randomiz visits.They must use effective contraceptive method throughout the entire duration of the study treatment,and for at least 12wk after the last admin of IMP
    Postmenopausal women must be amenorrheic for at least 12mth
    Per una migliore comprensione, fare riferimento alla sinossi ITA
    Durata malat>36 mesi dalla 1°manifestaz del fen di nonRaynaud
    mRSS<10 o>35 allo screen e vis basali
    Anamnesi di vasculite, att o in remis
    Diagn malat del tess connett (diversa da SSc) o di PM/Scl
    PosHIV o nota anamnesi di inf HIV, att o in remiss
    Test anorm per Epa B e/o epa C indicativi di un'inf att o cronica
    Test anorm per Epa B: HBsAG pos all'epa B o Ab core tot pos all'epa B (HBcAb) con Ab di superf neg all'epa B o Ab core tot pos con Ag di superf pos e presenza DNA epa B
    Test anorm epa C Ab pos antiHCV e RNA pos HCV
    Test QTF pos o 2 conf allo screen (indip prec stato di tratt)
    Inf grave (es polmonite, pielonefrite) entro 4 sett dallo screen, inf con ospedaliz o somm antibiotici x via EV entro 4 sett dallo screen o infez batt cronica (es osteomielite)
    Anamnesi anafilassi a qualsiasi terapia biol
    Prova di qualunque inf o condiz medica CS, grave o instabile, acuta o cronic progr, non controll (es cerebrale, cardiaca, polmonare, renale, epatica, gastrointest o neurol, diversa da SSc o SScILD) o malat chirurg prec att o in atto o qualunque condiz che possa impattare la sicur del pz a giudiz del PI
    Allo screen FVC predetta <=75% e capacità diffusione polmonare di CO predetta corretta in base valore di Hb <=40%.
    Storia di insuff cardiaca EF del ventricolo sx <=45%, coronaropatia angina infarto miocardio e cardiomiopatia ischemica/ipertrofica. Ogni precedente anamnesi neoplasia o K maligno att,incluse le malat linfoproliferative nei 5aa precedenti il basale (a eccez del K della cervice in situ trattato con successo, del K della pelle a cell squamose non metastatizzato o a cell basali)
    modifiche ischem all’ECG e/o altre modifiche CS all’ECG allo screen
    Alta dose steroidi (>10 mg/die prednisolone equiv); o variaz dose steroidi entro 4 sett prec lo screen o durante il periodo di screen o cambiamenti attesi durante il corso dello studio
    Precedente tratt con rituximab nei 12 mesi precedenti la visita di screen
    Precedente tratt con trapianto di midollo osseo, irradiaz tot linfoide o ablaz ad alte dosi di ciclofosfamide
    Tratt con alta dose di farmaci immunosoppressivi (es. ciclofosfamide>1 mg/kg orale/die o>750 mg IV/mese; azatioprina>100mg/die; metrotrexato>15mg/sett; micofenolato mofetil>2g/die) nei 3 mesi prec lo screen o variaz dose nelle 4 sett precedenti al basale
    Tratt con etanercept, ciclosporina A, IVIG, rapamicina, Penicillamina D, inibitori della TK entro 4 sett dallo screen o ATG entro 6 mesi dallo screen
    Tratt con infliximab, certolizumab, golimumab, abatacept o adalimumab, tocilizumab entro 8 sett dallo screen o anakinra nella sett precedente lo screen
    Tratt con qualunque IMP nel mese precedente lo screen, o nelle 5 emivite, se noto (quello fra i 2che risulta + lungo)
    Test di lab anormali allo screen
    ALT o AST>2 volte ULN
    Hb<11g/100 ml per i maschi e <10 mg/100 ml per le donne
    NE <1500mm^3 (ad eccez <1000/mm3 per pop Africane)
    PLT <100000 mm^3
    Creatinina = 150 µmol/L nota: i valori di lab possono essere ripetuti 1volta durante il periodo di screen se ritenuti non attendibili o in caso di err tecnico x determ eleggibilità
    Anamnesi attuale abuso alcol e/o sostanze
    Attuale impiego c/o Sanofi o parente impiegato Sanofi (coniuge, genitori, figlio, fratello)
    Attuale condizione/previsione di carcerazione durante il corso dello studio
    Qualunque condiz o circostanza che può precludere al pz la possibilità di seguire e completare il protocollo a giudizio del PI
    Donne in gravid o in allat
    Donne in età fertile che non fanno uso di contrac ad elevata efficacia (vedi modulo di CI) e/o che non vogliono o non sono in grado di eseguire un test di gravidanza. Le donne in età fertile devono avere un test di grav neg allo screen e alle visite di randomiz. Devono utilizzare un metodo contracc
    efficace durante l'intera durata dello studio e x almeno 12 sett dopo l'ultima somm dell’IMP
    Donne in menop devono essere in amenorrea da almeno 12 mesi
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline in mRSS
    Variazione nel mRSS dal basale
    E.5.1.1Timepoint(s) of evaluation of this end point
    From Baseline to Week 24
    Dal basale alla settimana 24
    E.5.2Secondary end point(s)
    Change from Baseline in Health Assessment Questionnaire Disability Index (HAD-QI), assessed with SHAQ

    Change from baseline in respiratory function as measured by observed Forced Vital Capacity (FVC)
    Change from baseline in observed Carbon Monoxide Diffusing Lung Capacity (DLco 8corrected for hemoglobin))
    Variazione dal basale nell¿Indice di disabilit¿ del Questionario per la valutazione della salute (HAQ-DI), valutato mediante questionario per la valutazione della salute nella sclerodermia (SHAQ)
    Variazione dal basale nella funzione respiratoria, misurata in base ai valori osservati di capacit¿ vitale forzata (CVF)

    Variazione dal basale della capacit¿
    polmonare di diffusione del monossido di carbonio (DLco[corretta per l¿emoglobina])
    E.5.2.1Timepoint(s) of evaluation of this end point
    From Baseline to Week 24
    Dal basale alla settimana 24
    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 Yes
    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 trial2
    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 Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA24
    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
    Russian Federation
    United Kingdom
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months3
    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: 70
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 24
    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 state7
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 55
    F.4.2.2In the whole clinical trial 94
    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 Decision2016-12-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-07-19
    P. End of Trial
    P.End of Trial StatusCompleted
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