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    Summary
    EudraCT Number:2013-005373-43
    Sponsor's Protocol Code Number:LUM001-304
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-07-09
    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 ConcernedPoland - Office for Medicinal Products
    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 also known as SHP625) that may help treat the liver and control itching in Alagille Syndrome. In the Optional Follow-up Treatment Period (after Week 48), all eligible children treated in the LUM001-304 study will be offered to continue the study drug treatment until the subjects are eligible to enter another LUM001 study or LUM001 is available commercially, or the sponsor
    stops the program or development in this indication.
    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 SponsorMirum 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 supportMirum Pharmaceuticals,Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMirumPharmaceuticals, Inc.
    B.5.2Functional name of contact pointChief Scientific Officer
    B.5.3 Address:
    B.5.3.1Street Address950 Tower Lane, Suite 1050
    B.5.3.2Town/ cityFoster City, California
    B.5.3.3Post code94404
    B.5.3.4CountryUnited States
    B.5.4Telephone number0016503255156
    B.5.5Fax number0016503255157
    B.5.6E-mailmedinfo@mirumpharma.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 INNMaralixibat chloride
    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 typeup to
    D.3.10.3Concentration number800
    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) is an autosomal dominant with variable penetration genetic multisystem disorder. The clinical diagnosis is based on the presence of intrahepatic bile duct paucity on liver biopsy in association with at least three of the major clinical features: chronic cholestasis, cardiac disease, skeletal abnormalities, ocular abnormalities and characteristic facial features.
    E.1.1.1Medical condition in easily understood language
    Alagille syndrome is a genetic disorder that can affect the liver, heart, and other parts of the body. One of the major features of ALGS is liver damage caused by abnormalities in the bile ducts.
    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 20.0
    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 are to evaluate:
    1. The long-term safety and tolerability of LUM001 in children with ALGS.
    2. The effect of LUM001 on serum bile acid levels in children with ALGS.
    3. The effect of LUM001 on biochemical markers of cholestasis and liver
    disease in children with ALGS.
    4. The effect of LUM001 on pruritus in children with ALGS.
    5. The long-term effect of LUM001 in children with ALGS 48 weeks of treatment.

    Additional objectives for the Optional Follow-up Treatment Period are:
    1. To explore twice a day (BID) dosing regimen and higher daily dosing of LUM001;
    2. Assessment of alpha-fetoprotein (AFP) levels, a marker of hepatocellular carcinoma;
    3. Assessment of the palatability of the LUM001 formulation in all subjects, by-proxy in subjects <4 years old and by patient questionnaire in children ≥4 years old.
    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 based on the diagnostic criteria.
    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. Males and females of child-bearing potential who are sexually active, or are not currently sexually active during the study, but become sexually active during the period of the study and for 30 days following the last dose of study drug, must agree to use acceptable contraception during the trial.
    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.

    Inclusion Criteria for subjects with LUM001 dosing interruption <7 days, or ≥7 days are:
    Subjects will be considered eligible for the long-term optional follow-up treatment period if they meet the following criteria:
    1. The subject has either:
    a. completed the protocol through the Week 48 visit with no major safety concerns
    OR
    b. discontinued due to safety reasons judged unrelated to the study drug, and
    laboratory results have returned to levels acceptable for this patient population or individual and subject does not meet any of the protocol’s stopping rules at the time of entry into the follow-up period. The decision will be made by the investigator in consultation with the sponsor medical monitor. [Subjects who were discontinued for other reasons will be considered on an individual basis.]
    2. Females of childbearing potential must have a negative urine or serum pregnancy test (β-human chorionic gonadotropin [β-hCG]) at the time of entry into the long term optional follow-up treatment period.
    3. Males and females of child-bearing potential who are sexually active, or are not currently sexually active during the study, but become sexually active during the period of the study and 30 days following the last dose of study drug, must agree and use acceptable contraception during the trial.
    4. Informed consent and assent (per IRB/EC) as appropriate.
    5. Access to phone for scheduled calls from study site.
    6. Caregivers (and age appropriate subjects) must be willing and able to use an eDiary device during the study.
    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 g/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 (eg, inflammatory bowel disease).
    7. History or presence of gallstones or kidney stones.
    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. Recent medical history or current status that suggests that the subject may be unable to complete the study.
    11. Any female who is pregnant or lactating or who is planning to become pregnant during the study period.
    12. Known history of alcohol or substance abuse.
    13. Administration of bile acid or lipid binding resins within 28 days prior to screening and throughout the trial.
    14. Known hypersensitivity to LUM001or any of its components.
    15. 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.
    16. History of non-adherence to medical regimens, unreliability, mental instability or incompetence that could compromise the validity of informed consent or lead to nonadherence with the study protocol based upon investigator judgment.
    17. 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.
    18. Subjects weighing over 50 kg at screening

    Exclusion Criteria for Subjects with LUM001 dosing interruption≥7 days are:
    All exclusion criteria mentioned for the core study apply upon entry into the long-term optional follow-up period, with the exception of exclusion criterion #18 (subjects weighing over 50 kg at screening).
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoints will be the mean change from Week 18 to 22 of fasting serum bile acid levels in subjects who previously responded to LUM001 treatment, as defined by a reduction in sBA ≥50% from baseline to Week 12 or Week 18. A sensitivity analysis will also be conducted using subjects who experienced a reduction from baseline in serum bile acids of ≥50% at the Week 48 measurement.
    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), in subjects who previously responded to LUM001 treatment, as defined by a reduction in ItchRO scale >1 point from baseline to Week 12 or Week 18.
    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, placebo-controlled, randomized 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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA9
    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
    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 years5
    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 years5
    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) Yes
    F.1.2.1Number of subjects for this age range: 1
    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/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 state8
    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)
    Upon completion of protocol LUM001-304 treatment options for patients will be evaluated by the study doctor
    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 Decision2014-11-03
    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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