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    Summary
    EudraCT Number:2012-005346-38
    Sponsor's Protocol Code Number:LUM001-302
    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:2013-05-07
    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 number2012-005346-38
    A.3Full title of the trial
    A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF LUM001, AN APICAL SODIUM-DEPENDENT BILE ACID TRANSPORTER INHIBITOR (ASBTi), IN THE TREATMENT OF CHOLESTATIC LIVER DISEASE IN PAEDIATRIC PATIENTS WITH ALAGILLE SYNDROME
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED CLINICAL STUDY TO EVALUATE THE SAFETY AND EFFICACY OF LUM001, AN AGENT THAT INHIBITS BILE ACID REUPTAKE FROM THE INTESTINE, IN THE TREATMENT OF CHOLESTATIC LIVER DISEASE IN PAEDIATRIC PATIENTS WITH ALAGILLE SYNDROME
    A.3.2Name or abbreviated title of the trial where available
    IMAGO
    A.4.1Sponsor's protocol code numberLUM001-302
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01903460
    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 pointVice President, Operations
    B.5.3 Address:
    B.5.3.1Street Address12531 High Bluff Drive, Suite 110
    B.5.3.2Town/ citySan Diego, CA
    B.5.3.3Post code92130
    B.5.3.4CountryUnited States
    B.5.4Telephone number448447745347
    B.5.6E-mailckennedy@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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameLUM001
    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.3.9.2Current 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 280
    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 (ALGS). This is an example of cholestatic liver disease in children. In patients with Alagille syndrome, impairment of the egress of bile acids from the liver leads to cholestasis, hepatocellular injury and damage, and progressive liver disease
    that may ultimately lead to the need for liver transplantation. Itch is the archetypal symptom of cholestasis, occurring at all stages of cholestatic liver disease, with or without jaundice.
    E.1.1.1Medical condition in easily understood language
    Alagille syndrome is a long-term debilitating and life-threatening disease due to liver and heart problems.
    E.1.1.2Therapeutic area Body processes [G] - Metabolic Phenomena [G03]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    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 safety and tolerability of LUM001 in paediatric subjects with ALGS.

    To evaluate the effect of LUM001 versus placebo on serum bile acids associated with ALGS.

    To evaluate the effect of LUM001 versus placebo on liver enzymes associated with ALGS.

    To evaluate the effect of LUM001 versus placebo on pruritus associated with ALGS.

    To explore the effect of LUM001 versus placebo on other biochemical markers associated with ALGS.
    E.2.2Secondary objectives of the trial
    Not applicable.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female, 2 -18 years of age.
    2. Diagnosis of ALGS based on the diagnostic criteria outlined in Section 16.3 of the study protocol.
    3. Cholestasis as evidenced by total serum bile acid > 3x upper limit of normal (ULN) for age.
    4. Intractable pruritus explainable only by liver disease.
    5. 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 randomization. A daily score is the highest score between the
    morning and evening reports. The average daily score is the sum of all daily scores divided by the number of days the ItchRO was completed.
    6. Native liver.
    7. Females of childbearing potential must have a negative urine or serum pregnancy test [β human chorionic gonadotropin (β-hCG)] during screening and a negative urine pregnancy test at the baseline visit.
    8. 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, i.e., (a) condom (male or female) or (b)
    diaphragm, with spermicide; or
    c. Intrauterine device (IUD).
    9. The ability to read and understand English (caregivers and children above the age of assent).
    10. Subjects expected to have a consistent caregiver(s) for the duration of the study.
    11. Informed consent and assent (per EC) as appropriate.
    12. Access to phone for scheduled calls from study site
    13. Caregivers (and age appropriate subjects) must be willing and able to use an eDiary device daily for a minimum of 20 weeks.
    14. Caregivers (and age appropriate subjects) must digitally accept the licensing agreement in the eDiary software at the outset of the study.
    15. 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, prior to randomization (maximum possible reports = 14 per week).
    E.4Principal exclusion criteria
    Subjects will be excluded from the study if they meet any of the following criteria:

    1. Chronic diarrhoea requiring specific intravenous fluid or nutritional intervention for the diarrhoea and/or its sequelae.
    2. Surgical disruption of the enterohepatic circulation.
    3. Liver transplant.
    4. Decompensated cirrhosis [international normalized ratio (INR) > 1.5 albumen < 30g/L, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy].
    5. ALT or AST > 15 x ULN at screening.
    6. History or presence of other concomitant liver disease.
    7. 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).
    8. Known diagnosis of human immunodeficiency virus (HIV) infection.
    9. Cancers except for in situ carcinoma, or cancers treated at least 5 years prior to screening with no evidence of recurrence.
    10. Any female who is pregnant or lactating or who is planning to become pregnant within 20 weeks of randomization.
    11. Any known history of alcohol or substance abuse.
    12. Administration of bile acid or lipid binding resins within 30 days prior to randomization and throughout the trial.
    13. Investigational drug, biologic, or medical device within 30 days prior to randomization, 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 on 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 is change from baseline to Week 13 in fasting serum bile acids.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 0 and week 13.
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints include change from baseline to Week 13 (compared to placebo) in liver enzymes (ALP, AST, ALT) and pruritus as measured by the average daily score of the ItchRO instrument. The average daily score will be calculated using the 7 days pre-treatment for baseline, and the last 7 days of treatment for Week 13

    Exploratory efficacy endpoints include:

    Change from baseline in fasting serum bile acid level at Weeks 5 and 9.

    Change from baseline for liver enzymes (ALT, AST, ALP) at Weeks 5 and 9.

    Change from baseline in pruritus as measured by the average daily ItchRO (Observer ItchRO/patient ItchRO) at Weeks 5 and 9. The average daily score will be calculated using the 7 days prior to each visit.

    Change from baseline for other biochemical markers of cholestasis (total and conjugated bilirubin, total cholesterol, LDL-C) at Weeks 5, 9 and 13.

    Responder analysis: pruritus response rates as measured by ItchRO (Observer ItchRO/patient ItchRO) at Weeks 5, 9 and 13.

    Change from baseline in the clinician administered pruritus scale, at Weeks 2, 5, 9 and 13.

    Change from baseline in bile acid synthesis [serum 7α-hydroxy-4-cholesten-3-one (7αC4)] at Weeks 5, 9 and 13.

    Change from baseline for PedsQL at Week 13.

    Other efficacy endpoints include:

    Patient Impression of Change (PIC) at Week 13.

    Caregiver Impression of Change (CIC) at Week 13.

    Caregiver Global Therapeutic Benefit (CGTB) assessment at Week 13.

    In addition, the following exploratory evaluations will be conducted if the results of the primary and secondary analyses illustrate a difference between drug and placebo treated subjects:

    Change from baseline for other biochemical markers [autotaxin and
    lysophosphatidic acid (LPA)] at Weeks 5, 9 and 13.

    Change from baseline for urine measures of bile acid at Week 13.

    Change from baseline for measures of bile acid synthesis (FGF-19 and FGF-21) at Weeks 5, 9 and 13.
    E.5.2.1Timepoint(s) of evaluation of this end point
    For secondary endpoints change from baseline to Week 13.

    Timepoints for evaluation of each exploratory endpoint are reported in Section 5.2.
    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 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 No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial4
    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 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 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
    LVLS
    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 months2
    E.8.9.1In the Member State concerned days
    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: 18
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 12
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    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 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
    Paediatric Study.
    Subjects and/or their legally acceptable representative(s) will be required to read, sign, and date an IEC approved informed consent/ascent form (ICF/IAF) summarizing the discussion at screening.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state18
    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)
    Following the end of the study subjects will have the option to return to their previous therapeutic regimen or opt to enroll into an extension study. A separate extension study protocol has been submitted by the sponsor to the MHRA (LUM001-303, EudraCT No. 2013-003832-54, September 13, 2013) and patients who complete the study may be eligible for participation in that study.
    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
    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 Decision2013-06-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-06-10
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-02-23
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