ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
Alexion Pharmaceuticals, Inc.
Study ID
NCT07218887
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • BAG3 Mutation Associated Dilated Cardiomyopathy

Eligibility Criteria

Sex
ALL
Age
18 Years - 70 Years
Healthy Volunteers
Not accepted

Interventions

  • ALXN2350 — DRUG
    ALXN2350 is a gene therapy product consisting of an AAV9 capsid containing BAG3 transgene. It is administered as a single intravenous (IV) infusion.

Study Details

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.

Key Dates

Start date
Oct 24, 2025
Status verified
Mar 2026
Primary completion
Jan 27, 2032
Completion
Jan 27, 2032

Study Design

Enrollment
6 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: ALXN2350
    Participants will receive one of three dose levels of ALXN2350 depending on the cohort.

Primary Outcome Measure

Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [ Time Frame: Baseline up to Week 78 ]

Central Contacts

Locations (5)

FacilityCityStateZIPSite coordinators
Research SiteBirminghamAlabama35233-
Research SiteBostonMassachusetts02115-
Research SiteCincinnatiOhio45219-
Research SitePortlandOregon97239-3098-
Research SiteHoustonTexas77030-

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Research Site· Birmingham, ALResearch Site· Boston, MAResearch Site· Cincinnati, OHResearch Site· Portland, ORResearch Site· Houston, TX

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