ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy

Last updated: June 12, 2026
Sponsor: Alexion Pharmaceuticals, Inc.
Overall Status: Active - Recruiting

Phase

1/2

Condition

N/A

Treatment

ALXN2350

Clinical Study ID

NCT07218887
D7030C00001
2024-519674-40-00
  • Ages 18-70
  • All Genders

Study Summary

This Phase 1/2 study is an open-label, dose finding and dose expansion study investigating the safety, tolerability, and efficacy of a single IV infusion of ALXN2350 in adult participants with BAG3 associated DCM.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Pathogenic or likely pathogenic mutation in BAG3

  • Medical history of diagnosis of DCM

  • Stable combination of HF SoC medications

  • Adequate acoustic windows for echocardiography

Exclusion

Exclusion Criteria:

  • Presence of antibodies to AAV9

  • Presence of a pathogenic or likely pathogenic variant in another gene where thatother gene is authoritatively recognized as causal for DCM.

  • Decompensated HF

Note: Other inclusion and exclusion criteria may apply.

Study Design

Total Participants: 6
Treatment Group(s): 1
Primary Treatment: ALXN2350
Phase: 1/2
Study Start date:
October 24, 2025
Estimated Completion Date:
January 27, 2032

Connect with a study center

  • Research Site

    Massa, 54100
    Italy

    Active - Recruiting

  • Research Site

    Barcelona, 08035
    Spain

    Active - Recruiting

  • Research Site

    Majadahonda, 28222
    Spain

    Active - Recruiting

  • Research Site

    London, SW3 6NP
    United Kingdom

    Active - Recruiting

  • Research Site

    Birmingham, Alabama 35233
    United States

    Active - Recruiting

  • Research Site

    Boston, Massachusetts 02115
    United States

    Active - Recruiting

  • Research Site

    Cincinnati, Ohio 45219
    United States

    Active - Recruiting

  • Research Site

    Portland, Oregon 97239-3098
    United States

    Active - Recruiting

  • Research Site

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

  • Research Site

    Houston, Texas 77030
    United States

    Active - Recruiting

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