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    Summary
    EudraCT Number:2015-000906-20
    Sponsor's Protocol Code Number:LUM001-601
    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:2015-04-01
    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 number2015-000906-20
    A.3Full title of the trial
    An Open-label, Multicenter Extension Study to Evaluate the Long-term Safety of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients with Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Open-label, Multicenter Extension Study to Evaluate the Long-term Safety of LUM001, an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients with Alagille Syndrome (ALGS) or Progressive Familial Intrahepatic Cholestasis (PFIC)
    A.4.1Sponsor's protocol code numberLUM001-601
    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 SponsorShire Human Genetic Therapies Inc
    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 supportShire Human Genetic Therapies Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationShire Pharmaceuticals Development Ltd
    B.5.2Functional name of contact pointEU Regulatory Strategy Group Lead
    B.5.3 Address:
    B.5.3.1Street AddressHampshire International Business Park
    B.5.3.2Town/ cityBasingstoke, Hampshire
    B.5.3.3Post codeRG24 8EP
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+441256894569
    B.5.6E-mailrmorgan@shire.com
    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/3/13/1214
    D.3 Description of the IMP
    D.3.1Product nameLUM001
    D.3.2Product code LUM001
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNLUM001
    D.3.9.1CAS number 228113-66-4
    D.3.9.2Current sponsor codeLUM001
    D.3.10 Strength
    D.3.10.1Concentration unit µg/kg microgram(s)/kilogram
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number14 to 400
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC)
    E.1.1.1Medical condition in easily understood language
    Cholestatic liver diseases associated with either ALGS or PFIC. Long term extension study allowing extended treatment for patients from both the ALGS and PFIC study programmes
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level SOC
    E.1.2Classification code 10010331
    E.1.2Term Congenital, familial and genetic disorders
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level PT
    E.1.2Classification code 10053870
    E.1.2Term Alagille syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic 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 evaluate the long-term safety of LUM001 in patients with ALGS or PFIC
    E.2.2Secondary objectives of the trial
    To evaluate the long-term durability of treatment effect of LUM001 on serum laboratory markers of cholestasis (i.e., serum bilirubin, aminotransferases, cholesterol).
    To evaluate the long-term durability of treatment effect of LUM001 on serum bile acids.
    To evaluate the long-term durability of treatment effect of LUM001on pruritus.
    To evaluate the long-term durability of treatment effect of LUM001 on xanthomas associated with ALGS.
    To evaluate the long-term durability of treatment effect of LUM001 on quality of life in patients with ALGS or PFIC.
    To evaluate the long-term durability of treatment effect of LUM001 on quality of life of caregivers of patients with ALGS or PFIC.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Competent to provide informed consent and assent, as age appropriate, (per IRB/EC).
    2.Completed a treatment protocol of LUM001 in the treatment of cholestatic liver disease in patients with ALGS or PFIC, which includes Study LUM001-303, LUM001-304, LUM001 305, or LUM001-501.
    3.Females of childbearing potential must have a negative urine pregnancy test [β human chorionic gonadotropin (β-hCG)] at the Week 0 Visit.
    4.Sexually active females must be prepared to use an effective method (≤ 1% failure rate) of contraception during the trial. Effective methods of contraception are considered to be:
    a.Hormonal (e.g., contraceptive pill, patch, intramuscular implant or injection); or
    b.Barrier method, e.g., (a) condom with spermicide, or (b) diaphragm, with spermicide; or
    c.Intrauterine device (IUD).
    5.Access to phone for scheduled calls from study site.
    E.4Principal exclusion criteria
    1.Experienced an adverse event or serious adverse event (SAE) related to the study drug during a core LUM001 treatment protocol that led to the discontinuation of the subject from the core LUM001 treatment study.
    2.Any conditions or abnormalities (including laboratory abnormalities) which, in the opinion of the Investigator or Medical Monitor, may compromise the safety of the subject, or interfere with the subject participating in or completing the study.
    3.History of non-adherence during the subject’s participation in the core LUM001 treatment protocol. Non-adherence is defined by dosing compliance of less than 80% in the core LUM001 treatment protocol.
    4.Unlikely to comply with the study protocol, or unsuitable for any other reason, as judged by the investigator.
    E.5 End points
    E.5.1Primary end point(s)
    The primary evaluation for the durability of the therapeutic effect will be the change from Week 0 to Week 24, 48, 72, and 104/early termination in:
    •Fasting serum bile acid level.
    •Biochemical markers of cholestasis and liver disease [ALT, GGT and total bilirubin].
    •Pruritus as measured by the Caregiver Impression of Change to assess itch and Clinician Scratch Scale.
    •Xanthomas as measured by Clinician Xanthoma Scale.
    •To evaluate the long-term durability of treatment effect of LUM001 on quality of life in patients with ALGS or PFIC, using the PedsQL core module.
    •To evaluate the long-term durability of treatment effect of LUM001 on quality of life of caregivers of patients with ALGS or PFIC, using the PedsQL family impact module
    E.5.1.1Timepoint(s) of evaluation of this end point
    Serum bile acids and cholestasis markers are measured at baseline and weeks 24, 48, 72 and 104. Patient questionnaires to measure pruritis, xanthomas and quality of life are assessed at baseline and weeks 24, 48, 72 and 104
    E.5.2Secondary end point(s)
    Additional exploration of evaluations of durability of therapeutic effect, including behavior.
    E.5.2.1Timepoint(s) of evaluation of this end point
    At weeks 24, 48, 72 and 104
    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 No
    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.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) 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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA12
    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
    Australia
    Canada
    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
    LPLV
    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 months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months6
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 120
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 2
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 76
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 37
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 5
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children from 1-18 yrs are to be recruited. Assent and parent/caregiver consent will be obtained for those children not able to provide assent personally.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state40
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 65
    F.4.2.2In the whole clinical trial 120
    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)
    The trial will continue until the study drug (LUM001) has been approved for treatment of ALGS and PFIC. Patients will then return to the care of their own physician at the end of the trial.
    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 Medicines for Children Research Network (MCRN)
    G.4.3.4Network Country United Kingdom
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2015-05-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-05-08
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2015-07-24
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