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    EudraCT Number:2011-001326-26
    Sponsor's Protocol Code Number:NI-0801-03
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2011-03-24
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2011-001326-26
    A.3Full title of the trial
    PIANO. 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.
    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.
    A.3.2Name or abbreviated title of the trial where available
    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 S.A.
    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 support
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    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.3.9.3Other descriptive namefully human monoclonal antibody directed against CXCL10
    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. 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Primary biliary cirrhosis
    E.1.1.1Medical condition in easily understood language
    Disease of the liver marked by the slow progressive destruction of the small bile ducts within the liver
    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 LLT
    E.1.2Classification code 10036680
    E.1.2Term Primary biliary cirrhosis
    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

    E.2.2Secondary objectives of the trial
    To evaluate the safety and tolerability of multiple doses of NI-0801 in primary biliary cirrhosis patients
    To characterize the pharmacokinetic and pharmacodynamic profile of multiple doses of NI 0801
    To assess immunogenicity of 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
    E.4Principal exclusion criteria
    1. Screening bilirubin > 2.9 mg/dL (50 ╬╝mol/L)
    2. Screening creatinine clearance < 80 ml/min
    3. History or presence of hepatic decompensation
    4. History or presence of other concomitant liver diseases including hepatitis due to hepatitis B or C virus (HCV, HBV), primary sclerosing cholangitis (PSC), alcoholic liver disease, definite autoimmune liver disease (AIH) and biopsy proven nonalcoholic steatohepatitis (NASH)
    5. Positive serology result for Human Immunodeficiency Virus (HIV), Hepatitis B or C
    6. Average alcohol ingestion >21 units/week (male) / >14 units/week (female)
    7. Have received any immunosuppressants, steroids or peroxisome proliferator activation receptor (PPAR) activators at any time during the 3 months prior to screening for the study
    8. Known or previous diagnosis of malignancy
    9. Presence of any active infection
    10. Presence or history of multiple allergic reactions to drugs
    11. Previous history of active TB within 12 months of screening
    12. Pregnant or breastfeeding women
    13. Have received or plan to receive any vaccines within 6 weeks prior to receiving study drug and throughout the study period
    E.5 End points
    E.5.1Primary end point(s)
    Change in liver enzyme measurements from baseline to Week 12 (SD85)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 12 (SD85)
    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 related reasons
    • Vital signs and any changes revealed by medical examination.
    • Laboratory abnormalities in Safety Hematology parameters, Safety Blood Chemistry parameters and Urinalysis parameters

    • The presence of anti-drug antibodies

    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

    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 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. 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) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    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 concerned12
    E.8.5The trial involves multiple Member States No
    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 date of the last visit of the last patient undergoing the study.
    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 months1
    E.8.9.1In the Member State concerned days30
    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: 35
    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 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 state40
    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)
    Continue with standard treatment of care
    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 Decision2011-05-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-05-27
    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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