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    Summary
    EudraCT Number:2020-001104-41
    Sponsor's Protocol Code Number:ALXN1840-WD-204
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2020-06-11
    Trial 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 number2020-001104-41
    A.3Full title of the trial
    A Phase 2, open-label study to assess copper and molybdenum balance in participants with Wilson disease treated with ALXN1840
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Copper Balance in Participants with Wilson Disease Treated with ALXN1840
    A.3.2Name or abbreviated title of the trial where available
    Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840
    A.4.1Sponsor's protocol code numberALXN1840-WD-204
    A.5.4Other Identifiers
    Name:RPL codeNumber:C19048
    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 SponsorAlexion 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 supportAlexion Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAlexion Europe S.A.S.
    B.5.2Functional name of contact pointNathalie Boeglin
    B.5.3 Address:
    B.5.3.1Street Address103-105 rue Anatole France
    B.5.3.2Town/ cityLevallois-Perret
    B.5.3.3Post code92300
    B.5.3.4CountryFrance
    B.5.4Telephone number33147 10 0615
    B.5.6E-mailNathalie.Boeglin@alexion.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/3/12/1089
    D.3 Description of the IMP
    D.3.2Product code ALXN1840
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    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 INNNot applied
    D.3.9.1CAS number 649749-10-0
    D.3.9.2Current sponsor codeALXN1840
    D.3.9.3Other descriptive nameBIS-CHOLINE TETRATHIOMOLYBDATE
    D.3.9.4EV Substance CodeSUB168578
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number15
    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
    Wilson Disease (WD)
    E.1.1.1Medical condition in easily understood language
    WD is a genetic disorder which leads to people having higher levels of copper in their body than normal.
    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 LLT
    E.1.2Classification code 10047988
    E.1.2Term Wilson's disease
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Assess net copper (Cu) balance with daily repeat-dose ALXN1840 treatment (15 mg and 30 mg) in participants with Wilson disease (WD)
    E.2.2Secondary objectives of the trial
    • Assess change in Cu balance in response to ALXN1840 15 mg/day and 30 mg/day during ALXN1840 accumulation and at steady state periods versus the pretreatment baseline in participants with WD

    • Investigate the effect of ALXN1840 (15 mg/day and 30 mg/day) on the disposition of Cu in participants with WD

    • Investigate the effect of ALXN1840 on the disposition of molybdenum (Mo) at steady state at 15 mg/day and 30 mg/day in participants with WD

    • Assess steady-state total Mo balance as a measure of ALXN1840 15 mg/day and 30 mg/day in participants with WD

    • Assess accumulation of Mo with ALXN1840 treatment at 15 mg/day and 30 mg/day in participants with WD

    • Determine the steady-state plasma pharmacokinetic (PK) of total Mo and plasma ultrafiltrate (PUF) Mo (as surrogate measures of ALXN1840, 15 mg and 30 mg) in participants with WD.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Participants aged ≥18 at the time of signing the ICF.

    2. Diagnosis of WD by Leipzig Criteria > 4 documented by testing as outlined in the 2012 European Association for the Study of Liver WD Clinical Practice Guidelines

    3. Participants who in the opinion of the referring Investigator may benefit from decoppering therapy.

    4. Participants must be able to comply with all study-related procedures.

    5. Participants must be able to reside in the CRU for intensive metabolic monitoring of Cu and Mo.

    6. Participants willing to discontinue chelator therapy for approximately 4 days prior to initiation of ALXN1840 to allow a baseline assessment of Cu balance.

    7. Participants with adequate venous access to allow collection of required blood samples.

    8. Participants must be able to swallow intact ALXN1840 tablets.

    9. Participants willing to avoid use of minerals containing Cu, Zn, or Mo throughout the study duration.

    10. Participants willing to adhere to Cu/Mo-controlled diet during inpatient periods and willing to comply with a low Cu dietary requirement during the Outpatient Period.

    11. Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

    12. Participants capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
    E.4Principal exclusion criteria
    1. Decompensated cirrhosis or model for end-stage liver disease (MELD) score > 13.

    2. Modified Nazer score > 7.

    3. Clinically significant gastrointestinal bleed within past 3 months.

    4. Alanine aminotransferase (ALT) > 2 × upper limit of normal (ULN).

    5. Marked neurological disease requiring assistance with self-care or activities of daily life.

    6. Hemoglobin less than lower limit of the reference range for age and sex.

    7. Active infection with hepatitis B virus (positive hepatitis B surface antigen) or C virus (participants with positive hepatitis C antibody result would require confirmation of active disease with a positive hepatitis C polymerase chain reaction test), or seropositivity for human immunodeficiency virus (HIV).

    8. History or presence of/significant history of or current cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrinological, hematological, or neurological disorders, or psychiatric disorder that in the opinion of the Investigator may constitute a risk when taking the study intervention; or may interfere with the interpretation of data.

    9. Previous treatment with ALXN1840 or other form of tetrathiomolybdate within 1 year prior to dosing.

    10. Previous treatment with zinc within 30 days prior to the Screening Visit.

    11. Participation in a clinical study of an experimental or unapproved/unlicensed therapy at the same time or within 90 days or 5 half-lives, whichever is longer, prior to the Screening Visit.

    12. Participants in renal failure, defined as in end-stage renal disease on dialysis (chronic kidney disease [CKD] stage 5) or creatinine clearance < 30 mL/min.

    13. Pregnant (or females who are planning to become pregnant) or breastfeeding females (women of childbearing potential must have a negative serum pregnancy test result at screening).

    14. Known sensitivity to ALXN1840, ALXN1840 excipients (anhydrous dicalcium phosphate, anhydrous sodium carbonate), or any of the ingredients contained in ALXN1840 or related compounds.

    15. In the opinion of the Investigator, the participant and/or their legal guardian is likely to be non-compliant or uncooperative during the study.

    16. History of illicit drug abuse, history of significant alcohol abuse within 1 year prior to the Screening Visit, or clinical evidence of substance and/or alcohol abuse within the 2 years before screening. Alcohol abuse is defined as regular weekly intake of more than 14 units (for both males and females), using the following National Health Service (NHS) alcohol tracker http://www.nhs.uk/Tools/Pages/drinks-tracker.aspx.

    17. Positive urine drug toxicology screen at Screening or on Day -8.

    18. Alcohol consumption within 48 hours prior to study intervention administration or positive alcohol breath test at screening or on Day -8.

    19. Participants unwilling to consistently complete every meal and tolerate a controlled, limited menu for the duration of the study.
    E.5 End points
    E.5.1Primary end point(s)
    Mean daily Cu balance where Cu balance is measured by the calculated difference between Cu intake (in food and drink) and Cu output (in feces and urine) during ALXN1840 accumulation and steady-state periods for each dose.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 1-8, Day 25-28, Day 31-35 and Day 36-39
    E.5.2Secondary end point(s)
    • Change in mean daily Cu balance as measured by the calculated difference between Cu intake (in food and drink) and Cu output (in feces and urine) from pretreatment baseline (Days -4 through -1) and ALXN1840 accumulation and steady-state periods for each dose

    • Copper quantified in food, drink, feces, and urine, including plasma total and labile bound Cu (LBC) during ALXN1840 accumulation and steady-state periods for each dose

    • Molybdenum quantified in food, drink, feces, and urine, plasma total Mo at ALXN1840 steady state

    • Mean daily Mo balance as demonstrated through measurement of Mo intake (in food, drink, and ALXN1840), and Mo output (feces and urine) representing ALXN1840 steady state

    • Accumulation of Mo as determined by Mo balance

    • PK parameters for plasma total and PUF-Mo
    E.5.2.1Timepoint(s) of evaluation of this end point
    Day 1, Day 4-7, Day 14, Day 25-40
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy No
    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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 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 No
    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 years0
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 10
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    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 completion of both inpatient periods of the study (ie, Day 40), participants will either:
    • transition to therapy that was discontinued before enrollment, or
    • at the discretion of their treating physician, consider and request continuation of ALXN1840 treatment permissible under local regulations for preapproval access.
    All participants should return to the CRU for the EOS Visit on Day 54 (+/- 2 days).
    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 Decision2020-07-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-07-22
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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