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    Summary
    EudraCT Number:2013-005373-43
    Sponsor's Protocol Code Number:LUM001-304
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-07-10
    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 ConcernedFrance - ANSM
    A.2EudraCT number2013-005373-43
    A.3Full title of the trial
    Long-Term, Open-Label Study with a Double-Blind, Placebo Controlled, Randomized Drug Withdrawal Period of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients with Alagille Syndrome
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    The purpose of this study is to evaluate a drug (LUM001) that may help treat the liver and control itching in Alagille Syndrome. In this study, all children who are eligible to enrol will take study drug for 18 weeks, followed by a 4 week period where they will either take LUM001 or placebo. After this 4 week period, all patients will go back on active study drug treatment for the remaining 26 weeks.
    A.3.2Name or abbreviated title of the trial where available
    ICONIC
    A.4.1Sponsor's protocol code numberLUM001-304
    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 SponsorLumena Pharmaceuticals 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 supportLumena Pharmaceuticals Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationLumena Pharmaceuticals Inc
    B.5.2Functional name of contact pointChief Operating Officer
    B.5.3 Address:
    B.5.3.1Street Address12531 High Bluff Drive, Suite 110
    B.5.3.2Town/ citySan Diego
    B.5.3.3Post codeCA 92130
    B.5.3.4CountryUnited States
    B.5.4Telephone number++1844744 5347
    B.5.5Fax number++1858461 4888
    B.5.6E-mailinfo@lumenapharma.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 Powder and solvent for 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 mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number0.05 to 60
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboOral solution
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Alagille Syndrome
    E.1.1.1Medical condition in easily understood language
    Alagille Syndrome
    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 18.0
    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
    The objectives of this study are:
    •To evaluate the long-term safety and tolerability of LUM001 in children with ALGS.
    •To evaluate the effect of LUM001 on serum bile acid levels in children with ALGS.
    •To evaluate the effect of LUM001 on biochemical markers of cholestasis and liver disease in children with ALGS.
    •To evaluate the effect of LUM001 on pruritus in children with ALGS.
    •To evaluate the long-term durability of effect of LUM001 in children with ALGS during 48-weeks of treatment.
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    To participate in this study subjects must meet all of the following criteria:
    1.Male or female between the ages of 12 months and 18 years inclusive.
    2.Diagnosis of ALGS .
    3.Evidence of cholestasis (one or more of the following):
    a.Total serum bile acid > 3x ULN for age.
    b.Conjugated bilirubin > 1 mg/dL.
    c.Fat soluble vitamin deficiency otherwise unexplainable.
    d.GGT > 3x ULN for age.
    e.Intractable pruritus explainable only by liver disease.
    4.Females of childbearing potential must have a negative serum pregnancy test during Screening.
    5.Sexually active females must be prepared to use an effective method of contraception during the trial, such as:
    a.Hormonal (e.g., contraceptive pill, patch, intramuscular implant or injection).
    b.Barrier method, i.e., (a) condom (male or female) or (b) diaphragm, with spermicide.
    c.Intrauterine device (IUD).
    6.Subject is expected to have a consistent caregiver(s) for the duration of the study.
    7.Informed consent and assent (per IRB/IEC) as appropriate.
    8.Access to phone for scheduled calls from study site.
    9.Caregivers (and age appropriate subjects) must be willing and able to use an eDiary device during the study.
    10.Caregivers (and age appropriate subjects) must digitally accept the licensing agreement in the eDiary software.
    11.Caregivers (and age appropriate subjects) must complete at least 10 eDiary reports (morning or evening) during each of two consecutive weeks of the screening period (maximum possible reports = 14 per week).
    12.Average daily score >2 on the Itch Reported Outcome (ItchRO™) questionnaire (maximum possible daily score of 4) for two consecutive weeks in the screening period, prior to dosing. A daily score is the higher of the scores for the morning and evening ItchRO. The average daily score is the sum of all daily scores divided by the number of days the ItchRO was completed.
    E.4Principal exclusion criteria
    Subjects will be excluded from the study if they meet any of the following criteria:
    1.Chronic diarrhea requiring ongoing intravenous fluid or nutritional intervention.
    2.Surgical interruption of the enterohepatic circulation.
    3.Previous liver transplant.
    4.Decompensated cirrhosis [ALT >15 x ULN, INR >1.5 (unresponsive to vitamin K therapy), albumin < 3.0 gm/dL, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy].
    5.History or presence of other concomitant liver disease.
    6.History or presence of any other disease or condition known to interfere with the absorption, distribution, metabolism or excretion of drugs, including bile salt metabolism in the intestine (e.g., inflammatory bowel disease).
    7.Known diagnosis of human immunodeficiency virus (HIV) infection.
    8.Cancers except for in situ carcinoma, or cancers treated at least 5 years prior to Screening with no evidence of recurrence.
    9.Recent medical history, or current status that suggests that the subject may be unable to complete the study.
    10.Any female who is pregnant or lactating or who is planning to become pregnant during the study period.
    11.Known history of alcohol or substance abuse.
    12.Administration of bile acid or lipid binding resins within 28 days prior to screening and throughout the trial.
    13.Receipt of investigational drug, biologic, or medical device within 28 days prior to Screening, or 5 half-lives of the study agent, whichever is longer.
    14.History of non-adherence to medical regimens, unreliability, mental instability or incompetence that could compromise the validity of informed consent or lead to non-adherence with the study protocol based upon Investigator judgment.
    15.Any other conditions or 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.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint will be the mean change in serum bile acid levels from Week 18 to Week 22 for those who responded to LUM001 treatment, which is defined as subjects who had a >50% reduction in serum bile acid levels from baseline to Week 18.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Serum bile acid levels are measured at baseline, 12, 18, 22 and 48 weeks
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints include mean change from Week 18 to Week 22 in liver enzymes (ALP, ALT, and bilirubin (total and direct)) and pruritus as measured by ItchRO (Observer ItchRO/patient ItchRO). Secondary efficacy endpoints also include mean change from baseline to Week 18 in serum bile acid level, liver enzymes (ALP, ALT, and bilirubin (total and direct)) and pruritus as measured by ItchRO (Observer ItchRO/Patient ItchRO)
    E.5.2.1Timepoint(s) of evaluation of this end point
    These are measured throughout the trial by means of the subject eDiary and clinician scales and questionnaires.
    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 Yes
    E.8.1.1Randomised Yes
    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 Yes
    E.8.1.7.1Other trial design description
    This trial also contains a double blind drug withdrawal period
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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
    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 years1
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    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 Yes
    F.1.1Number of subjects for this age range: 30
    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: 8
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 15
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 7
    F.1.2Adults (18-64 years) No
    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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children from 1-18 yrs are to be recruited. Assent and parent/cargiver 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 state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 18
    F.4.2.2In the whole clinical trial 30
    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 patients will return to their standard care with their own physician following completion of the trial
    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 Decision2014-07-02
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-05-28
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