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    Summary
    EudraCT Number:2014-002393-37
    Sponsor's Protocol Code Number:RG_13-027
    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:2014-11-19
    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 number2014-002393-37
    A.3Full title of the trial
    A single arm, two-stage, multi-centre, phase II clinical trial investigating the safety and activity of the use of BTT1023, a human monoclonal antibody targeting vascular adhesion protein (VAP-1), in the treatment of patients with primary sclerosing cholangitis (PSC)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical trial investigating the use of BTT1023 in patients with PSC
    A.3.2Name or abbreviated title of the trial where available
    BUTEO: A clinical trial of BTT1023 in patients with PSC
    A.4.1Sponsor's protocol code numberRG_13-027
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02239211
    A.5.4Other Identifiers
    Name:CRCTU CAS numberNumber:HE2022
    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 SponsorUniversity of Birmingham
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationThe University of Birmingham
    B.5.2Functional name of contact pointDr Gideon Hirschfield
    B.5.3 Address:
    B.5.3.1Street AddressFloor 5. IBR Building, The Univeristy of Birmingham
    B.5.3.2Town/ cityBirmingham
    B.5.3.3Post codeB15 2TT
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number01214143823
    B.5.5Fax number01214148286
    B.5.6E-mailg.hirschfield@bham.ac.uk
    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 nameBTT1023 IV Infusion 20 mg/mL, 5 mL Drug Product
    D.3.2Product code BTT1023
    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.3.11 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 No
    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
    Primary Sclerosing Cholangitis (PSC)
    E.1.1.1Medical condition in easily understood language
    Primary Sclerosing Cholangitis (PSC) - Liver autoimmune disease
    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 20.1
    E.1.2Level LLT
    E.1.2Classification code 10036732
    E.1.2Term Primary sclerosing cholangitis
    E.1.2System Organ Class 100000004871
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    We wish to see if the antibody, BTT1023, against VAP-1, is both safe and effective in the setting of primary sclerosing cholangitis, an autoimmune liver disease. We will determine this by safety assessments and measurement of the liver enzyme alkaline phosphatase, expecting that if the antibody therapy works, the level of alkaline phosphatase will fall by the end of the study.
    E.2.2Secondary objectives of the trial
    The secondary objectives of the study are to understand more about how the antibody therapy works in patients, as well as to look for other markers that the therapy works (including other blood tests of liver fibrosis, MRI imaging, patient symptoms and quality of life).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1) Males and females 18 - 75 years of age who are willing and able to provide informed, written consent and comply with all trial requirements
    2) Clinical diagnosis of PSC as evident by chronic cholestasis of more than six months duration with either a consistent MRI showing sclerosing cholangitis or a liver biopsy consistent with PSC in the absence of a documented alternative aetiology for sclerosing cholangitis
    3) In those with concomitant Inflammatory Bowel Disease, clinical and colonoscopic evidence (in line with the patient’s standard of care; within 18 months) of stable disease without findings of high grade dysplasia
    4) In those on treatment with ursodeoxycholic acid (UDCA), therapy must be stable for at least 8 weeks and at a dose not greater than 20 mg/kg/day. In those not on treatment with UDCA at the time of screening, a minimum of 8 weeks since the last dose of UDCA should be recorded
    5) Serum ALP greater than 1.5 x upper limit of normal (ULN)
    6) Stable serum ALP levels (levels must not change by more than 25% from Screening Visit 1 and Screening Visit 2)
    7) Female subjects of childbearing potential must have a negative pregnancy test prior to starting trial treatment. For the purposes of this trial, a female subject of childbearing potential is a woman who has not had a hysterectomy, bilateral oophorectomy, or medically-documented ovarian failure. Women ≤ 50 years of age with amenorrhea of any duration will be considered to be of childbearing potential
    8) All sexually active women of childbearing potential must agree to use two forms of highly effective method of contraception from the Screening Visit throughout the trial period and for 99 days following the last dose of trial drug. If using hormonal agents the same method must have been used for at least 1 month before trial dosing and subjects must use a barrier method as the other form of contraception. Lactating women must agree to discontinue breast feeding before trial investigational medicinal product administration
    9) Men, if not vasectomised, must agree to use barrier contraception (condom plus spermicide) during heterosexual intercourse from screening through to trial completion and for 99 days from the last dose of trial investigational medicinal product
    10) Patients must weigh ≥ 40 kg
    E.4Principal exclusion criteria
    1) Presence of documented secondary sclerosing cholangitis on prior clinical investigations
    2) Presence of alternative causes of liver disease, that are considered by the Investigator to be the predominant active liver injury at the time of screening, including viral hepatitis, alcoholic liver disease, non-alcoholic steatohepatitis, primary biliary cirrhosis. Patients with possible overlap syndrome with autoimmune hepatitis are excluded if the Investigator considers autoimmune hepatitis as the predominant liver injury
    3) AST and ALT >10 x ULN or bilirubin >3 x ULN or INR >1.3 in the absence of anti-coagulants
    4) Serum creatinine >130μmol/L or platelet count <50 x 109/L
    5) Any evidence of hepatic decompensation past or present, including ascites, episodes of hepatic encephalopathy or variceal bleeding
    6) Recent cholangitis within last 90 days or ongoing need for prophylactic antibiotics
    7) Pregnancy or breast feeding
    8) Harmful alcohol consumption as evaluated by the Investigator
    9) Flare in colitis activity within last 90 days requiring intensification of therapy beyond baseline maintenance treatment; use of oral prednisolone >10 mg/day, biologics (i.e. monoclonal antibodies) and or hospitalisation for colitis within 90 days. Prior use of biologics is not a contraindication to screening
    10) Diagnosed cholangiocarcinoma or high clinical suspicion of cholangiocarcinoma either clinically or by imaging
    11) Concurrent malignancies or invasive cancers diagnosed within past 3 years except for adequately treated basal cell and squamous cell carcinoma of the skin and in situ carcinoma of the uterine cervix
    12) Presence of a percutaneous drain or bile duct stent
    13) Major surgical procedure within 30 days of screening
    14) Prior organ transplantation
    15) Known hypersensitivity to the investigational product or any of its formulation excipients
    16) Unavailable for follow-up assessment or concern for subject’s compliance
    17) Participation in an investigational trial of a drug or device within 60 days of screening or 5 half-lives of the last dose of investigational drug, where the trial drug half-life is greater than 12 days
    E.5 End points
    E.5.1Primary end point(s)
    1) Response at Day 99: a reduction in serum alkaline phosphatase (ALP) by 25% or more from baseline to Day 99.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 99 (after start of treatment=Day 1)
    E.5.2Secondary end point(s)
    1) To determine the mechanisms of action of BTT1023 through in vivo assessment of VAP-1/semicarbazide-sensitive amine oxidase (SSAO) enzyme activity and immune cell function.

    2) To evaluate the potential of a novel MRI based assessment of liver fibrosis and biliary strictures for assessing therapeutic response in PSC.

    3) Assess the use of sVAP-1/SSAO as a biomarker to monitor disease progression in PSC.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary:
    1) Safety and tolerability
    • Treatment Compliance (including patient withdrawal) and Adverse Event frequency.
    2) An improvement from baseline to day 99 in:
    • Quality of life questionnaires: EQ-5D, Fatigue Severity Scale, Pruritus Visual Analogue Score (VAS), IBD Diaries (if applicable).
    • Tests of liver fibrosis: ELF and Fibroscan.
    • Individual markers of liver biochemistry and function (AST, ALT, ALP, GGT, Bilirubin, Albumin, INR) and composite risk scores (Mayo PSC risk score and MELD).
    • Change (improvement) in LiverMultiscan® MRI imaging – Liver MRI is an emerging method for monitoring liver disease and its treatment. We will evaluate changes in MRI imaging pre and post therapy using the LiverMultiscan® protocol.
    3) Evaluate changes in sVAP-1/SSAO as a biomarker
    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.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 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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.3 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 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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days30
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months10
    E.8.9.2In all countries concerned by the trial days30
    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: 38
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 3
    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 state59
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 0
    F.4.2.2In the whole clinical trial 0
    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)
    As this clinical trial involves an Investigational Medicinal Product (IMP) currently there is no current provision for compassionate use of the IMP outside this or other clinical trials. Patients care will revert back to standard NHS practice and licensed therapies. This study will not prevent patients from receiving any NHS approved therapies in the future. Patients will be made aware of this situation.
    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 Biotie Therapies Corp.
    G.4.3.4Network Country Finland
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2014-12-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-01-06
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-10-23
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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