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    Summary
    EudraCT Number:2019-003711-60
    Sponsor's Protocol Code Number:ALXN1840-WD-205
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-06-02
    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 ConcernedGermany - BfArM
    A.2EudraCT number2019-003711-60
    A.3Full title of the trial
    A Phase 2, single-arm, pathologist-blinded study using liver biopsy specimens to assess copper concentration and histopathologic changes in patients with Wilson disease who are treated with ALXN1840 for 48 weeks followed by an extension treatment period with ALXN1840 for up to an additional 48 weeks
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2 clinical study to assess copper levels and liver changes in patients with Wilson disease who are treated with ALXN1840
    A.4.1Sponsor's protocol code numberALXN1840-WD-205
    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 SAS
    B.5.2Functional name of contact pointMartine Zimmerman
    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 number33787148158
    B.5.6E-mailmartine.zimmerman@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 numberEU3/12/1089
    D.3 Description of the IMP
    D.3.1Product nameALXN1840
    D.3.2Product code ALXN1840
    D.3.4Pharmaceutical form Gastro-resistant 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's Disease
    E.1.1.1Medical condition in easily understood language
    Wilson's Disease
    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
    To evaluate change in liver copper (Cu) concentration following treatment with ALXN1840 at Week 48 in patients with Wilson disease (WD)
    E.2.2Secondary objectives of the trial
    The secondary objectives are to:
    - To assess change in liver histopathology following treatment with ALXN1840
    - To evaluate the safety of ALXN1840 in patients with WD
    - To evaluate pharmacokinetics (PK) of ALXN1840
    - To evaluate the effects of ALXN1840 on clinical symptoms
    please see study protocol for more details.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patients must be ≥ 18 years of age at the time of signing the informed consent.
    2. Diagnosis of WD by Leipzig Criteria >4 documented by testing as outlined in the 2012 European Association for the Study of Liver (EASL) WD Clinical Practice Guidelines (Ferenci, 2003; EASL, 2012)
    3. Continuous treatment for WD with penicillamine, trientine or zinc for at least 1 year in duration prior to screening
    4. Adequate venous access to allow collection of required blood samples
    5. Able to swallow intact ALXN1840 tablets
    6. Body mass index < 30 kg/m2
    7. Able to cooperate a percutaneous liver biopsy, including having the ability to lie flat and still throughout the procedure, and tolerate mild sedation, if required
    8. Adequately visualized landmarks on screening ultrasound without evidence of significant ascites, hemangiomas, or other findings that would put the patient at unnecessarily high risk of complications
    9. Male and female patients of reproductive potential must agree to remain abstinent or use an effective method of contraception throughout the duration of the study
    10. Capable of giving signed informed consent
    E.4Principal exclusion criteria
    1. Decompensated cirrhosis or MELD score >13
    2. Modified Nazer score > 7 (Dhawan, 2005)
    3. Clinically significant gastrointestinal bleed within past 3 months
    4. Alanine aminotransferase > 2 × upper limit of normal (ULN)
    5. History of bleeding abnormality or known coagulopathy, including platelet count < 100,000, and international normalized ratio for prothrombin time (PT-INR) ≥ 1.5; coagulopathy or bleeding risk due to medication is acceptable if medication can be safety discontinued for biopsy
    6. Patient unwilling to accept blood products
    7. Marked neurological disease requiring either nasogastric feeding tube or intensive inpatient medical care
    8. Hemoglobin less than lower limit of the reference range for age and sex
    9. Systemic disease or other illness, any disability acquired from trauma or another illness, or any deviation in laboratory values that are confirmed on re-examination to be clinically significant by the Investigator
    10. Patients in renal failure, defined as in end-stage renal disease on dialysis (chronic kidney disease [CKD] 5) or creatinine clearance < 30 mL/min
    11. Known sensitivity to ALXN1840, ALXN1840 excipients or any of the ingredients contained in ALXN1840
    12. History or presence of/significant history of or current cardiovascular, respiratory, renal, gastrointestinal, endocrinological, hematological, or neurological disorders capable of significantly altering the absorption, metabolism, or elimination of drugs
    13. Lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years
    14. Current or chronic history of liver disease not associated with WD, or known hepatic or biliary abnormalities (with the exception of asymptomatic gallstones)
    15. Use of nonprescription/ over-the-counter medications, including herbal remedies, nutritional supplements, or mineral supplements containing Cu, zinc, iron or Mo after dosing on Day 1 through the end of the study.
    16. In the opinion of the Investigator, the patient and/or their legal guardian is likely to be non-compliant or uncooperative during the study
    17. Participation in any other interventional study during the study or for 3 months (or 5 half-lives of the administered investigational medicinal product, whichever is longer) prior to study start.
    18. Presence of hepatitis B surface antigen or positive hepatitis C antibody or RNA test result at screening or within 3 months prior to first dose of study drug. NOTE: Patients with positive Hepatitis C antibody due to prior resolved disease can be enrolled if a confirmatory negative Hepatitis C ribonucleic acid (RNA) test is obtained. NOTE: The RNA test is optional and patients with negative Hepatitis C antibody test are not required to also undergo Hepatitis C RNA testing
    19. Positive human immunodeficiency virus (HIV) antibody test
    20. Regular alcohol consumption within 6 months prior to the study defined as > 14 units for males or > 7 units for females per week. One unit is equivalent to 8 g of alcohol: a half pint (approx. 240 mL) of beer, 1 glass (125 mL) of wine or 1 (25 mL) measure of spirits.
    21. Abuse of illicit or prescribed drugs
    22. Sensitivity to any drug or other allergy that, in the opinion of the Investigator or Alexion Medical Monitor, contraindicates participation in the study
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline to Week 48 in liver Cu concentration
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 48
    E.5.2Secondary end point(s)
    The secondary endpoints include the following:
    - Change from baseline to Week 48 in steatosis, inflammation, and fibrosis
    - Adverse events (AEs), vital signs, ECGs, clinical laboratory data, and physical examination data
    - ALXN1840 PK profiles in plasma at Week 6 (Day 43) and Week 36 (Day 253), of which ALXN1840 PK are measured as total molybdenum (Mo) and plasma ultrafiltrate (PUF) Mo
    - Pre-dose trough ALXN1840 concentrations in plasma at each study site visit
    - ALXN1840 concentrations in the liver biopsy specimen.
    - Change from baseline in Clinical Global Impression-Improvement (CGI-I) Scale and the Clinical Global Impression-Severity (CGI-S) Scale
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 48
    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 No
    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 Yes
    E.8.1.7.1Other trial design description
    Pathologist Blinded
    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.8.2.4Number of treatment arms in the trial1
    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 EEA35
    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
    Canada
    Korea, Republic of
    New Zealand
    Russian Federation
    Serbia
    Singapore
    Turkey
    United States
    Austria
    Belgium
    Denmark
    France
    Germany
    Poland
    Spain
    Sweden
    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
    LPLV of the 48-week extension period
    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 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: 20
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 8
    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 state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 16
    F.4.2.2In the whole clinical trial 28
    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)
    Subjects who complete the 48-week treatment period will be offered the opportunity to participate in an Extension Phase of the study to evaluate the long-term safety and durability of treatment effect of ALXN1840. If the subject does not choose to participate in this Extension Phase, he/she will be assisted in their transition to SoC for WD under the guidance of their local physician.
    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 Decision2020-09-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-01-15
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-05-17
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