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    Summary
    EudraCT Number:2011-001326-26
    Sponsor's Protocol Code Number:NI-0801-03
    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:2012-01-19
    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 number2011-001326-26
    A.3Full title of the trial
    Primary Biliary Cirrhosis: Investigating A New Treatment Option using NI 0801, a fully human anti-CXCL10 monoclonal antibody. An open label single arm study to investigate the safety and efficacy of multiple administrations of NI-0801, a fully human anti-CXCL10 monoclonal antibody in primary biliary cirrhosis patients with an incomplete response to ursodeoxycholic acid.
    La Cirrosi Biliare Primitiva: valutare una nuova opzione terapeutica usando NI-0801, un anticorpo monoclonale anti-CXCL10 completamente umano. Studio in aperto a braccio singolo per valutare la sicurezza e l'efficacia di somministrazioni multiple di NI-0801, un anticorpo monoclonale completamente umano anti-CXCL10 in pazienti con cirrosi biliare primitiva con risposta incompleta all'acido ursodesossicolico.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Primary Biliary Cirrhosis: Investigating A New Treatment Option using NI 0801, a fully human anti-CXCL10 monoclonal antibody.
    La Cirrosi Biliare Primitiva: valutare una nuova opzione terapeutica usando NI-0801, un anticorpo monoclonale anti-CXCL10 completamente umano.
    A.3.2Name or abbreviated title of the trial where available
    PIANO
    PIANO
    A.4.1Sponsor's protocol code numberNI-0801-03
    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 SponsorNOVIMMUNE BV
    B.1.3.4CountryNetherlands
    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 supportNOVIMMUNE BV
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIRCCS Istituto Clinico Humanitas
    B.5.2Functional name of contact pointCentro Malattie Autoimmuni Fegato
    B.5.3 Address:
    B.5.3.1Street AddressVia Manzoni 113
    B.5.3.2Town/ cityRozzano (MI)
    B.5.3.3Post code20089
    B.5.3.4CountryItaly
    B.5.4Telephone number'+39 02 8224 5128
    B.5.5Fax number'+39 02 8224 5191
    B.5.6E-mailpietro.invernizzi@humanitas.it
    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 nameNI-0801
    D.3.2Product code NI-0801
    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.3.9.2Current sponsor codeNI-0801
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Proven PBC, as demonstrated by the presence of at least 2 of the following 3 diagnostic factors: - History of increased ALP levels for at least 6 months - Positive serum AMA titer (>1:40) - Liver biopsy consistent with PBC Patient should be on incomplete response to UDCA
    Diagnosi di Cirrosi Biliare Primitiva (PBC), confermata dalla presenza di almeno 2 dei seguenti 3 criteri diagnostici: - Storia di aumento del livello delle ALP per almeno 6 mesi - Positività sierica per AMA (>1:40) - Biopsia epatica diagnostica per la diagnosi di PBC I pazienti devono presentare una risposta incompleta al trattamneto standar Acido Ursodesossicolico
    E.1.1.1Medical condition in easily understood language
    Patients with a diagnosis of Primary Biliary Cirrhosis (PBC) that do not respond to UDCA
    Pazienti con diagnosi di Cirrosi Biliare Primitiva (PBC) che non rispondono in modo soddisfacente al farmaco Acido Ursodesossicolico
    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 14.1
    E.1.2Level SOC
    E.1.2Classification code 10021428
    E.1.2Term Immune system disorders
    E.1.2System Organ Class 10021428 - Immune system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level SOC
    E.1.2Classification code 10019805
    E.1.2Term Hepatobiliary disorders
    E.1.2System Organ Class 10019805 - Hepatobiliary 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 investigate the efficacy of multiple doses of NI 0801 on hepatic function
    •Valutare l’efficacia di dosi multiple di NI 0801 sulle funzioni epatiche
    E.2.2Secondary objectives of the trial
    •To evaluate the safety and tolerability of multiple doses of NI-0801 in PBC patients •To characterize the pharmacokinetic and pharmacodynamic profile of multiple doses of NI 0801
    •Valutare la sicurezza e tollerabilità di dosi multiple di NI-0801 in pazienti affetti da PBC •Caratterizzare il profilo farmacocinetico e farmacodinamico di dosi multiple di NI 0801
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female, 18 years or older 2. Proven PBC, as demonstrated by the presence of at least 2 of the following 3 diagnostic factors: - History of increased ALP levels for at least 6 months - Positive serum AMA titer (>1:40) - Liver biopsy consistent with PBC 3. Incomplete response to UDCA defined as failure to normalise ALP levels or to reduce ALP levels by more than 40% after 1 year of therapy with UDCA according to the local recommended dose. 4. Stable dose of UDCA for at least 6 months prior to screening 5. Screening ALP > 1.5 ULN 6. Screening ALT or AST > 1.5 ULN 7. Female patients must be postmenopausal, surgically sterile, or willing to use 2 methods of effective contraception with all sexual partners until 3 months after having received the last dose of study medication 8. Male patients who agree to take the appropriate precautions to avoid fathering a child until 3 months after having received the last dose of study medication 9. Have given written informed consent 10. Patients must be able to adhere to the study visits and protocol requirements
    1. Uomini o donne, dai 18 anni in su 2. Diagnosi di Cirrosi Biliare Primitiva (PBC), confermata dalla presenza di almeno 2 dei seguenti 3 criteri diagnostici: - Storia di aumento del livello delle ALP per almeno 6 mesi - Positività sierica per AMA (&gt;1:40) - Biopsia epatica diagnostica per la diagnosi di PBC 3. Risposta incompleta all'UDCA definita come definita come il fallimento nella normalizzazione dei livelli di ALP o nella riduzione dei livelli di ALP di almeno il 40% dopo 1 anno di terapia con UDCA, secondo le linee guida locali. 4. Dose stabile di UDCA per almeno sei mesi rpiam dello screening 5. Allo screening ALP &gt; 1.5 ULN 6. Allo screening ALT or AST &gt; 1.5 ULN 7. Patienti donne devono essere in postmenopausa, sterili o devono accettare di fare uso di 2 metodi contraccettivi con tutti i partner fino a 3 mesi dopo aver ricevuto l'ultima dose di farmaco 8. Pazienti uomini che acconsentono a prendere adeguate precauzioni contraccettive fino a 3 mnesi dopo aver ricevuto l'ultima dose di farmaco 9. Firma del Consenso Informato 10. I pazienti devono essere in grado di seguire le procedure dello studio
    E.4Principal exclusion criteria
    • Screening bilirubin > 2.9 mg/dL (50 μmol/L) • Screening creatinine clearance < 80 ml/min • History or presence of hepatic decompensation (e.g., esophageal variceal bleeding, hepatic encephalopathy, or ascites) • Positive serology result for Human Immunodeficiency Virus (HIV), Hepatitis B or C • Known or previous diagnosis of malignancy • Presence of any active infection • Previous history of active TB within 12 months of screening
    • Allo screening bilirubina &gt; 2.9 mg/dL (50 μmol/L) • Allo screening clearance della creatinina &lt; 80 ml/min • Presenza o storia di disfunzione epatica (e.g., sanguinamento delle varici-esofagee, encefalopatia epatica, o ascite) • Positività al Virus da Immunodeficiencenza Acquisita (HIV), Epatite B o C • Precedente diagnosi di un tumore • Presenza di qualsiasi infezione attiva • Storia di TB attiva nei 12 mesi precedenti lo screening
    E.5 End points
    E.5.1Primary end point(s)
    Change in liver enzyme measurements from baseline to Week 12 (SD85)
    Cambiamento dei valori degli enzimi epatici dalla visita di baseline (screening) alla settimana 12 (giorno di studio 85)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 12 (SD85)
    settimana 12 (giorno di studio 85)
    E.5.2Secondary end point(s)
    PBC-related exploratory assessments • Biochemical markers of liver function • Mayo risk score (MRS) • Liver stiffness measurement (LSM) with Transient Elastography (Fibroscan™) • Enhanced Liver Fibrosis (ELF) test • Quality-of-Life assessment: PBC-40 Pharmacokinetic characterisation Pharmacodynamic measurements • The evolution over time of total circulating CXCL10 levels will be measured Extent of the NI 0801 inhibition on CXCL10 chemotactic activity by in vitro chemotaxis • Serum cytokines and chemokines • FACS analysis for the quantification of leukocytes and subsets and CXCR3 expression on these subsets Safety and tolerability • All emerging AEs • The number of subjects withdrawing from the study for safety relatedreasons • Vital signs and any changes revealed by medical examination. • Laboratory abnormalities in Safety Hematology parameters, Safety Blood Chemistry parameters and Urinalysis parameters Immunogenicity • The presence of anti-drug antibodies
    Indagini esploratorie correlate con la PBC: • Marcatori biochimici delle funzioni epatiche • Valore di rischio Mayo (MRS) • Misura dell'inspessimento epatico (LMS) con l'uso dell'Elastografia Transiente (Fibroscan) • test dell'Aumento della fibrosi epatica (ELF) • Questionario sulla qualità di vita: PBC-40 Caratterizzazione Farmacicinetica Misurazioni Farmacodinamiche • Valutazione nel tempo dei livelli totali circolanti di CXCL10 Inibizione di NI 0801 sull'attività chemotattica di CXCL10 atrtaverso la chemotassi in vitro • Citochine e chemochine sieriche • Analisi FACS per la quantizzazione dei leucociti e sottopopolazioni, espressione di CXCR3 su quetsa sottopopolazione Sicurezza e tollerabilità • tutti gli EA segnalati • Il numero dei soggetti che interrompono lo studio per motivi di sicurezza • Segni vitali e qualsiasi cambiamneto rilevato dall'esame medico • Valori di laboratorio anomali nei parametri ematologici, chimici e nei parametri urinari di immunogenicità • Presenza di anticorpi anti-farmaco
    E.5.2.1Timepoint(s) of evaluation of this end point
    ELF, Fibroscan, PBC-40: SD1, SD85, W18 PK, blood samples: SD1, SD5, SD15, SD29, SD43, SD57, SD71, SD75, SD85, W18, W24 Chemokines/cytokines: SD1, SD5, SD15, SD29, SD43, SD57, SD71, SD75, SD85, W18, W24 FACS: SD1, SD85 Immunogenicity: SD1, SD15, SD29, SD43, SD57, SD71, W24 Vital signs, hematology and biochemistry blood samples, AEs recording & concomittant medications: SD1, SD5, SD15, SD29, SD43, SD57, SD71, SD75, SD85, W18, W24
    ELF, Fibroscan, PBC-40: SD1, SD85, W18 PK, campioni di sangue: SD1, SD5, SD15, SD29, SD43, SD57, SD71, SD75, SD85, W18, W24 Chemochine/citochine: SD1, SD5, SD15, SD29, SD43, SD57, SD71, SD75,SD85,W18, W24 FACS: SD1, SD85 Immunogenicità: SD1, SD15, SD29, SD43, SD57, SD71, W24 Segni vitali, campioni di sangue per ematologia e biochimica, segnalazione AEs e farmaci concomitanti: SD1, SD5, SD15, SD29, SD43, SD57, SD71, SD75, SD85, W18, W24
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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.7.1.3.1Other 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 No
    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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial1
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA10
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    The end of the study is defined as the last patient completes the last follow-up visit at the week 24 post-dose.
    La fine dello studio è definita quando l'ultimo paziente completa l'ultima visita di follow-up 24 settimane dopo l'ultima infusione.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    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 years0
    E.8.9.2In all countries concerned by the trial months12
    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.1.1Number of subjects for this age range: 0
    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: 80
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 40
    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)
    standard therapy for PBC
    terapia standard per la PBC
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation NIHR-CRN
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2011-06-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-06-21
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2012-10-10
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