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    Summary
    EudraCT Number:2013-003832-54
    Sponsor's Protocol Code Number:LUM001-303
    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:2013-12-09
    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 ConcernedUK - MHRA
    A.2EudraCT number2013-003832-54
    A.3Full title of the trial
    A MULTICENTRE EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY AND DURABILITY OF THE THERAPEUTIC EFFECT OF LUM001, AN APICAL SODIUM-DEPENDENT BILE ACID TRANSPORTER INHIBITOR (ASBTI), IN THE TREATMENT OF CHOLESTATIC LIVER DISEASE IN PEDIATRIC SUBJECTS WITH ALAGILLE SYNDROME
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A MULTICENTRE 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
    IMAGINE STUDY
    A.4.1Sponsor's protocol code numberLUM001-303
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02047318
    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 organisationMirum Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointChief Scientific Officer
    B.5.3 Address:
    B.5.3.1Street Address 70 Willow Road, Suite 200
    B.5.3.2Town/ cityMenlo Park, California
    B.5.3.3Post code94025
    B.5.3.4CountryUnited States
    B.5.4Telephone number1650325 5156
    B.5.5Fax number1650325 5157
    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.3.4Pharmaceutical 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 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 number280
    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). 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 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 primary objective of the study (up to and including week 72) is to:

    1. Evaluate the long-term safety and tolerability of LUM001 in pediatric subjects with
    ALGS.
    E.2.2Secondary objectives of the trial
    Secondary objectives of the study (up to and including week 72) are to:
    •Evaluate the long-term effect of LUM001 on serum bile acid levels associated with ALGS.
    •Evaluate the long-term effect of LUM001 on pruritus associated with ALGS.
    •Explore the long-term effect of LUM001 on other biochemical markers of cholestasis and liver disease.
    •Evaluate the long-term effect of LUM001 on xanthomas associated with ALGS.
    • Explore an expanded dosing range to identify the doses necessary to achieve the optimal benefit-to-risk ratio for this patient population.

    Objectives of the long-term optional follow-up treatment period for subjects who are eligible for PA5:
    •To explore twice a day (BID) dosing regimen and higher daily dosing of LUM001.
    •To obtain safety and efficacy data in subjects treated long term on LUM001.
    •To assess the level of alpha-fetoprotein (AFP), a marker of hepatocellular carcinoma.
    •To assess palatability of the LUM001 formulation.
    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, 12 months to 18 years of age.
    2. Competent to provide informed consent and assent (per IRB/EC), as appropriate.
    3. Completed participation in protocol LUM001-302.
    4. Females of childbearing potential must have a negative urine pregnancy test [β human chorionic gonadotropin (β-hCG)] at the Baseline Visit.
    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 study. 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).
    d. A sexual partner who is surgically sterilized.
    6. Subjects are expected to have consistent caregiver(s) for the duration of the study.
    7.Caregiver (and age appropriate subjects) must be able to read and understand English.
    8. Caregivers (and age appropriate subjects) must have access to phone for scheduled calls from study site.
    9. Caregivers (and age appropriate subjects) must be willing and able to complete a daily electronic diary (ItchRO) during the first consecutive 12 weeks of the study and then for 4 consecutive weeks following the Week 24 and Week 44 visits.
    10. Caregivers (and age appropriate subjects) must digitally accept the licensing agreement in the ItchRO electronic diary software at the outset of the study.

    Eligible subjects for the 52-week optional follow-up treatment period:
    Subjects will be considered eligible for the 52-week optional follow-up treatment period if they have completed the protocol through the Week 72 visit with no safety concerns. Subjects who were discontinued due to safety reasons can be re-challenged if blood tests are back to relatively normal values for this patient population and subject does not meet any of the protocol’s stopping rules; the decision will be made by the investigator in consultation with the sponsor medical monitor. Subjects who have undergone a surgical disruption of the enterohepatic circulation will not be eligible to enter into the follow-up treatment period. Subjects who were discontinued for other reasons will be considered on an individual basis.

    Protocol Amendment 5: Eligible subjects for the long-term optional follow-up treatment period
    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 either the Week 124 or the ET 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 study.
    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. Experienced an adverse event or serious adverse event (SAE) related to the study drug during the LUM001-302 study that led to the discontinuation of the subject from the LUM001-302 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 or presence of gallstones or kidney stones.
    4. History of non-adherence during the subject’s participation in the LUM001-302 study. Non-adherence is defined by dosing compliance of less than 80% in the LUM001-302 study, or earlier in the LUM001-303 study.
    5. Unlikely to comply with the study protocol, or unsuitable for any other reason, as judged by the investigator.

    Protocol Amendment 5: Eligible subjects for the long-term optional follow-up treatment period.

    All exclusion criteria mentioned for the original study LUM001-303 apply upon re-entry into the long-term optional follow-up treatment period.
    E.5 End points
    E.5.1Primary end point(s)
    The primary evaluation will be the mean change from Baseline (Day 0) to Week 48 in:

    Fasting serum bile acid level.
    E.5.1.1Timepoint(s) of evaluation of this end point
    0, 2, 4, 8, 12, 16, 24, 36 and 48 weeks.
    E.5.2Secondary end point(s)
    Secondary evaluations will be the mean change from Baseline (Day 0) to Week 48 and the change from Week 12 to Week 48 in:

    1. Biochemical markers of cholestasis and liver disease [ALT, GGT and total bilirubin].
    2. Pruritus as measured by the ItchRO instruments (ItchRO(Obs)TM, caregiver instrument/ItchRO(Pt) TM patient instrument).
    -During the first 12 weeks of the study, the electronic diary (ItchRO) will be completed twice daily (AM & PM). During the stable dosing period (Weeks 13-
    48), twice daily completion of the electronic diary (ItchRO) for 4 consecutive weeks will be required following the Week 24 and Week 44 clinic visits. For
    subjects who continue in the follow-up treatment period, twice daily completion of the electronic diary (ItchRO) for 2 consecutive weeks will be required following the Week 84, 96, 108, and 120 clinic visits.
    3. Xanthomas as measured by clinician xanthoma scale.

    Change from Week 12 to Week 48 in fasting serum bile acid will also be examined.
    Efficacy variables will be presented for each time point at which they are measured. Additional exploration, including behavior during the dose escalation and optimization phases, will be specified in the SAP.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Time points for evaluation of each exploratory endpoint are reported in the Schedule of Procedures (Section 16.1)
    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 No
    E.8.1.1Randomised Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    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 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
    Telephone Contact Visits: Subjects/caregivers will receive follow-up phone calls at Weeks 76, 80, 88, 92, 100, 104, 112, 116, and at 30 days following the last dose of study drug. Concomitant medications and any AEs will be evaluated and recorded at all clinic visits and at scheduled telephone contacts.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    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 years6
    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: 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) Yes
    F.1.1.4.1Number of subjects for this age range: 6
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 6
    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 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
    Pediatric 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 prior to enrollment.
    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)
    Upon completion of protocol LUM001-303, 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 Decision2013-11-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-11-28
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-06-17
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