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    Summary
    EudraCT Number:2013-003833-14
    Sponsor's Protocol Code Number:LUM001-501
    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:2015-06-22
    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 number2013-003833-14
    A.3Full title of the trial
    AN 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.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, CA
    B.5.3.3Post code92130
    B.5.3.4CountryUnited States
    B.5.4Telephone number448447745347
    B.5.5Fax number++18584614888
    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 numberEMA/OD/123/13
    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.3.9.1CAS 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 18.0
    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
    The objectives of this study are:
    To evaluate the safety and tolerability of LUM001 in paediatric subjects with PFIC

    To evaluate the effect of LUM001 on serum bile acids associated with PFIC at 13 weeks of treatment

    To evaluate the effect of LUM001 on biochemical markers of cholestasis and liver disease at 13 weeks of treatment

    To evaluate the effect of LUM001 on pruritus associated with PFIC at 13 weeks of treatment

    To evaluate the safety and long-term durability of effect of LUM001 in patients 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 the following criteria:
    1. Male or female subjects between the ages of 12 months and 18 years, inclusive
    3. Diagnosis of PFIC based on:
    a. 2. Intrahepatic cholestasis manifest by fasting total serum bile acid >100 micromol/L and b or c
    b.Two documented mutant alleles in ATP8B1, or ABCB11, or other genes confirmed to cause PFIC
    or
    b. Evidence of chronic liver disease, excluding those listed in Protocol Appendix 16.3 with one or more of the following criteria:
    i. Duration of biochemical or clinical abnormalities of >6 months, or
    ii. Pathologic evidence of progressive liver disease, or
    iii. Sibling of known individual affected by PFIC (predicted to be chronic)
    4. GGTP <100 IU/L at screening
    5. 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.
    6. 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, e.g., (a) condom (male or female) or (b) diaphragm, with spermicide; or
    c. Intrauterine device (IUD)
    7. Informed consent and assent.
    8. Access to phone for scheduled calls from study site.
    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 first 13 weeks of the study
    11. 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.
    12. Caregivers (and age appropriate subjects) using the eDiary must digitally accept the licensing agreement in the eDiary software at the outset of the study.
    13. 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 assignment (maximum possible reports = 14 per week). Subjects using a paper diary must complete the same number of reports within the same timeframe.
    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 liver disease [international normalized ratio (INR) > 1.5, albumin <30 g/L, history or presence of clinically significant ascites, variceal haemorrhage and/or encephalopathy].
    5. ALT > 15 x ULN at screening.
    6. History or presence of other liver disease (see Protocol Appendix 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 assignment and throughout the trial.
    14. Investigational drug, biologic, or medical device within 30 days prior to screening, or 5 half-lives of the study agent, whichever is longer.
    15. 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.
    16. 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 serum bile acids.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day: 0, 14, 28, 56 and 91.
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints include change from baseline to Week 13 in biochemical markers of cholestasis and liver disease (ALP, AST, ALT, total and direct bilirubin) and change from baseline to Week 13 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 48-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, range (e.g., minimum and maximum). Qualitative variables will be summarized using counts and percentages. Summaries will be provided by study phase (Weeks 0-13 and 14-48) and over the entire study duration (Weeks 0-48), by visit and by stable dosing dose group (if appropriate). All data will be reported in data listings. Where appropriate, 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 comparisons 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 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 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
    United States
    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 Visit 30 Days after the Week 48 Visit for the last
    trial subject.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    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: 12
    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: 2
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 4
    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
    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 state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 7
    F.4.2.2In the whole clinical trial 12
    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. It is also the intent that the drug will remain available as appropriate for continued treatment of subjects completing LUM001-501 as long as Lumena Pharmaceuticals, Inc. is developing the drug for the treatment of PFIC.
    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 Decision2014-08-26
    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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