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    Summary
    EudraCT Number:2013-003833-14
    Sponsor's Protocol Code Number:LUM001-501
    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:2014-08-19
    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 number2013-003833-14
    A.3Full title of the trial
    OPEN LABEL STUDY OF THE EFFICACY AND LONG TERM SAFETY OF LUM001, AN APICAL
    SODIUM-DEPENDENT BILE ACID TRANSPORTER INHIBITOR (ASBTi), IN THE TREATMENT OF CHOLESTATIC LIVER DISEASE IN PEDIATRIC PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    AN OPEN LABEL STUDY OF THE EFFICACY AND LONG TERM SAFETY OF LUM001 IN THE TREATMENT OF CHOLESTATIC LIVER DISEASE IN PEDIATRIC PATIENTS WITH PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS
    A.3.2Name or abbreviated title of the trial where available
    INDIGO STUDY
    A.4.1Sponsor's protocol code numberLUM001-501
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02057718
    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, LLC.
    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 Address70 Willow Road, Suite 200
    B.5.3.2Town/ cityMenlo Park, California
    B.5.3.3Post code94025
    B.5.3.4CountryUnited States
    B.5.4Telephone number0016503255156
    B.5.5Fax number001650 3255157
    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 numberEMA/OD/123/13
    D.3 Description of the IMP
    D.3.1Product nameLUM001
    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 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
    In patients with progressive familial intrahepatic cholestasis (PFIC), 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 a common symptom associated with cholestasis, it can occur at all stages of cholestatic liver disease, with or without jaundice.
    E.1.1.1Medical condition in easily understood language
    PFIC 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 20.0
    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.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Objectives up to/including Wk 72:
     -To evaluate the long-term safety/tolerability of LUM001 in pediatric subjects (PS) with PFIC
     -To evaluate the effect of LUM001 on serum bile acids in PS with PFIC at 13 wks of treatment
     -To evaluate the effect of LUM001 on biochem. markers of cholestasis and liver disease at 13 wks of treatment
     -To evaluate the effect of LUM001 on pruritus in PS with PFIC at 13 wks of treatment

    Objectives of Optional Follow-up Treatm. Period (Post Wk 72):
     -To offer eligible subjects in the LUM001-501 continued study treatment beyond Week 72 until: (i) the subjects are eligible to enter another LUM001 study or (ii) LUM001 is available commercially
     -To obtain safety/efficacy data in subjects treated long-term on LUM001
     -To explore a BID and higher daily dosing regimen of LUM001
     -To identify genetic indicators of treatment response, incl. exome sequencing
     -To assess alpha-fetoprotein levels
     -To assess LUM001 formulation palatability
    E.2.2Secondary objectives of the trial
    To allow the possibility of analysis of serum markers of treatment response using
    metabolomic and proteomic analysis on previously collected serum samples.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    To participate in this study subjects must meet the following criteria:
    1. Male or female subjects between the ages of 12 months and 18 years inclusive.
    2. Diagnosis of PFIC based on:
    a. Intrahepatic cholestasis manifest by total serum bile acid >3x upper limit of normal (ULN) for age.
    and, b or c:
    b. Two documented mutant alleles in ATP8B1, or ABCB11.
    c. Evidence of chronic liver disease, excluding those listed in (see Section 16.3), with one or more of the following criteria:
    1. Duration of biochemical or clinical abnormalities of >6 months, or
    2. Pathologic evidence of progressive liver disease, or
    3. Sibling of known individual affected by PFIC (predicted to be chronic).
    3. GGTP <100 IU/L at screening.
    4. 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 (Day 0) 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 30 days following the last dose of study drug, must agree and use acceptable contraception during the trial, as described in Section 8.7.1.
    6. Informed consent and assent (per IRB/EC) as appropriate.
    7. Access to phone for scheduled calls from study site.
    8. Caregivers and children above the age of assent must have the ability to read and understand one of the following languages: English, Spanish, US Spanish, French, German or Polish.
    9. Subjects expected to have a consistent caregiver(s) for the duration of the first 13 weeks of the study.
    10. Caregivers (and age appropriate subjects) must be willing and able to use an eDiary device as required by the study. To accommodate potential cultural restrictions within the FIC1 affected population a paper version of the ItchRO diary will be made available.
    11. Caregivers (and age appropriate subjects) using the eDiary must digitally accept the licensing agreement in the eDiary software at the outset of the study.
    12. 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 toassignment (maximum possible reports = 14 per week). Subjects using a paper diary must complete the same number of reports within the same timeframe.

    To participate in optional follow-up treatment period subjects must meet the following criteria:
    1. The subject has either:
     completed the protocol through the Week 72 visit with no major safety concerns .
    OR
     discontinued due to safety reasons judged unrelated to the study drug, and blood tests
    have returned to levels acceptable for this patient population/individual and subject does not meet any of the protocol’s stopping rules at re-entry. 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-hCG) at the time of entry into the optional follow-up treatment period.
    3. Informed consent and assent (per IRB/EC) as appropriate.
    E.4Principal exclusion criteria
    Subjects will be excluded from the study if they meet any of the following criteria:
    1. Chronic diarrhea requiring specific intravenous fluid or nutritional intervention for the diarrhea and/or its sequelae.
    2. Surgical disruption of the enterohepatic circulation at the time at screening. Subjects who have undergone reversal of a prior surgical procedure intended to disrupt enterohepatic circulation and who and have a permanently restored flow of bile acids from the liver to the terminal ileum may be eligible for the study upon consultation with the Sponsor Medical Monitor.
    3. Liver transplant.
    4. Decompensated cirrhosis [international normalized ratio (INR) > 1.5, albumin < 30 g/L, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy].
    5. ALT >15×ULN at screening.
    6. History or presence of other liver disease (see Section 16.3).
    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. Liver mass on imaging.
    9. Known diagnosis of human immunodeficiency virus (HIV) infection.
    10. Cancers except for in situ carcinoma, or cancers treated at least 5 years prior to screening with no evidence of recurrence.
    11. Any female who is pregnant or lactating or who is planning to become pregnant within 20 weeks of assignment.
    12. Any known history of alcohol or substance abuse.
    13. Administration of bile acid or lipid binding resins within 30 days prior to
    Baseline / Day 0 and throughout the trial.
    14. Administration of sodium phenylbutyrate within 30 days prior to
    Baseline / Day 0 and throughout the trial.
    15. Investigational drug, biologic, or medical device within 30 days prior to screening, or 5 halflives 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 non-adherence with the study protocol based on 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.

    Subjects will be excluded from the optional follow-up treatment period if they meet the following criteria:
    1. Surgical disruption of the enterohepatic circulation.
    2. Investigational drug other than LUM001, biologic, or medical device within 30
    days prior to re-entry, or 5 half-lives of the study agent, whichever is longer.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is mean change from baseline to Week 13 in fasting serum bile acids.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day: 0, 28, 56, 91, 168, 196, 252, 336, 420, 504, 588, 602, 672, 756, 840, every three months thereafter, and at the EOT visit.
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints include change from baseline to Week 48 in biochemical markers of cholestasis and liver disease (ALT, total and direct bilirubin) and change from baseline to Week 48 in pruritus as measured by the average daily score of the ItchRO instrument.

    The safety and long-term durability of effect of LUM001 in patients
    will be assessed during 124-weeks of treatment.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Continuous variables will be summarized using descriptive statistics including n, mean, median, standard deviation, and range (i.e., minimum and maximum). Qualitative variables will be summarized using counts and percentages. Summaries will be provided by study phase (Weeks 0-13, 14-72, 73-124) and over the entire study duration (Weeks 0-124), by visit and by stable dosing dose group (if appropriate). All data will be reported in data listings. All statistical tests will be conducted at the 0.05 significance level using two-tailed tests and p-values will be reported. Given the rare nature of PFIC, the statistical power of any comparison is limited. As such the analysis will be largely descriptive in nature.
    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 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA8
    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
    Poland
    United Kingdom
    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
    The end of study for the purposes of regulatory reporting is the point at which the last contact with the last subject during the protocol-specified scheduled follow-up treatment period is made.
    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 months11
    E.8.9.1In the Member State concerned days15
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months0
    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) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 16
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 2
    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
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 7
    F.4.2.2In the whole clinical trial 18
    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 LUM001-501 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 Decision2015-01-14
    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 trial2020-05-08
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