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Early Access Program for ALXN1840 in Patients With Wilson Disease

Primary Purpose

Wilson Disease

Status
No longer available
Phase
Locations
Study Type
Expanded Access
Intervention
ALXN1840
Sponsored by
Alexion
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Wilson Disease focused on measuring Wilson Disease, ALXN1840

Eligibility Criteria

3 Years - undefined (Child, Adult, Older Adult)All Sexes

Inclusion Criteria: ≥ 3 years of age or older at the time of providing informed consent/assent and expressed desire to continue treatment with ALXN1840. Confirmed diagnosis of WD. Has completed one of the following ALXN1840 clinical studies: ALXN1840-WD-205 WTX101-301 ALXN1840-WD-302 Other ALXN1840 studies In the Treating Physician's medical opinion, the potential benefits of treatment with ALXN1840 outweigh the potential risks for the participant. Participant or legal representative provided informed consent/assent to be treated with ALXN1840 through this EAP. Female participants of childbearing potential and male participants must follow protocol-specified-contraception guidance. Exclusion Criteria: Eligible for and able to participate in an Alexion-sponsored study of ALXN1840. Unwilling or unable to comply with the Treating Physician's treatment plan related to this EAP for any reason. Has decompensated hepatic cirrhosis. Model for End-Stage Liver Disease (MELD) score > 13. Modified Nazer Score > 7. End-stage renal disease on dialysis (chronic kidney disease stage 5 [CKD 5]) or creatinine clearance < 30 mL/min. Known hypersensitivity to ALXN1840, ALXN1840 excipients, or any of the ingredients contained in ALXN1840. Women who are nursing or pregnant (or women who are planning to become pregnant during treatment with ALXN1840).

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    January 6, 2023
    Last Updated
    May 15, 2023
    Sponsor
    Alexion
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    1. Study Identification

    Unique Protocol Identification Number
    NCT05686564
    Brief Title
    Early Access Program for ALXN1840 in Patients With Wilson Disease
    Official Title
    Early Access Program for ALXN1840 in Patients With Wilson Disease
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    May 2023
    Overall Recruitment Status
    No longer available
    Study Start Date
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Alexion

    4. Oversight

    5. Study Description

    Brief Summary
    This is an open-label, single-arm, multi-center EAP, designed to provide early access to ALXN1840 for eligible patients with WD.
    Detailed Description
    To provide access to ALXN1840 treatment, an investigational medicinal product (IMP) that has not yet been granted marketing authorization, to participants who complete Studies ALXN1840-WD-205, WTX101-301, or ALXN1840-WD-302, or other studies with ALXN1840 who, in the opinion of the Treating Physician, may benefit from continued treatment with ALXN1840, and who meet the eligibility criteria described in this protocol.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Wilson Disease
    Keywords
    Wilson Disease, ALXN1840

    7. Study Design

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    ALXN1840
    Other Intervention Name(s)
    INN: tiomolibdic acid; USAN/JAN: tiomolibdate choline (WTX101)
    Intervention Description
    bis-choline tetrathiomolybdate

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    3 Years
    Eligibility Criteria
    Inclusion Criteria: ≥ 3 years of age or older at the time of providing informed consent/assent and expressed desire to continue treatment with ALXN1840. Confirmed diagnosis of WD. Has completed one of the following ALXN1840 clinical studies: ALXN1840-WD-205 WTX101-301 ALXN1840-WD-302 Other ALXN1840 studies In the Treating Physician's medical opinion, the potential benefits of treatment with ALXN1840 outweigh the potential risks for the participant. Participant or legal representative provided informed consent/assent to be treated with ALXN1840 through this EAP. Female participants of childbearing potential and male participants must follow protocol-specified-contraception guidance. Exclusion Criteria: Eligible for and able to participate in an Alexion-sponsored study of ALXN1840. Unwilling or unable to comply with the Treating Physician's treatment plan related to this EAP for any reason. Has decompensated hepatic cirrhosis. Model for End-Stage Liver Disease (MELD) score > 13. Modified Nazer Score > 7. End-stage renal disease on dialysis (chronic kidney disease stage 5 [CKD 5]) or creatinine clearance < 30 mL/min. Known hypersensitivity to ALXN1840, ALXN1840 excipients, or any of the ingredients contained in ALXN1840. Women who are nursing or pregnant (or women who are planning to become pregnant during treatment with ALXN1840).

    12. IPD Sharing Statement

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    Early Access Program for ALXN1840 in Patients With Wilson Disease

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