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Early Access Program for ALXN1840 in Patients With Wilson Disease

Sponsor
Alexion Pharmaceuticals (Industry)
Overall Status
Available
CT.gov ID
NCT05686564
Collaborator
(none)

Study Details

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.

Condition or Disease Intervention/Treatment Phase

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.

Study Design

Study Type:
Expanded Access
Official Title:
Early Access Program for ALXN1840 in Patients With Wilson Disease

Outcome Measures

Primary Outcome Measures

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    3 Years and Older
    Sexes Eligible for Study:
    All
    Inclusion Criteria:

    1. ≥ 3 years of age or older at the time of providing informed consent/assent and expressed desire to continue treatment with ALXN1840.

    2. Confirmed diagnosis of WD.

    3. Has completed one of the following ALXN1840 clinical studies:

    4. ALXN1840-WD-205

    5. WTX101-301

    6. ALXN1840-WD-302

    7. Other ALXN1840 studies

    8. In the Treating Physician's medical opinion, the potential benefits of treatment with ALXN1840 outweigh the potential risks for the participant.

    9. Participant or legal representative provided informed consent/assent to be treated with ALXN1840 through this EAP.

    10. Female participants of childbearing potential and male participants must follow protocol-specified-contraception guidance.

    Exclusion Criteria:

    1. Eligible for and able to participate in an Alexion-sponsored study of ALXN1840.

    2. Unwilling or unable to comply with the Treating Physician's treatment plan related to this EAP for any reason.

    3. Has decompensated hepatic cirrhosis.

    4. Model for End-Stage Liver Disease (MELD) score > 13.

    5. Modified Nazer Score > 7.

    6. End-stage renal disease on dialysis (chronic kidney disease stage 5 [CKD 5]) or creatinine clearance < 30 mL/min.

    7. Known hypersensitivity to ALXN1840, ALXN1840 excipients, or any of the ingredients contained in ALXN1840.

    8. Women who are nursing or pregnant (or women who are planning to become pregnant during treatment with ALXN1840).

    Contacts and Locations

    Locations

    No locations specified.

    Sponsors and Collaborators

    • Alexion Pharmaceuticals

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Alexion Pharmaceuticals
    ClinicalTrials.gov Identifier:
    NCT05686564
    Other Study ID Numbers:
    • D9430R00001
    First Posted:
    Jan 17, 2023
    Last Update Posted:
    Jan 17, 2023
    Last Verified:
    Jan 1, 2023
    Keywords provided by Alexion Pharmaceuticals
    Wilson Disease
    ALXN1840
    Additional relevant MeSH terms:
    Hepatolenticular Degeneration
    Choline

    Study Results

    No Results Posted as of Jan 17, 2023