ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy

Purpose

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.

Condition

  • BAG3 Mutation Associated Dilated Cardiomyopathy

Eligibility

Eligible Ages
Between 18 Years and 70 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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 Criteria

  • Presence of antibodies to AAV9 - Presence of a pathogenic or likely pathogenic variant in another gene where that other gene is authoritatively recognized as causal for DCM. - Decompensated HF Note: Other inclusion and exclusion criteria may apply.

Study Design

Phase
Phase 1/Phase 2
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
ALXN2350 		Participants will receive one of three dose levels of ALXN2350 depending on the cohort.
  • Drug: ALXN2350
    ALXN2350 is a gene therapy product consisting of an AAV9 capsid containing BAG3 transgene. It is administered as a single intravenous (IV) infusion.

Recruiting Locations

Research Site
Birmingham, Alabama 35233

Research Site
Boston, Massachusetts 02115

Research Site
Cincinnati, Ohio 45219

Research Site
Portland, Oregon 97239-3098

Research Site
Houston, Texas 77030

More Details

Status
Recruiting
Sponsor
Alexion Pharmaceuticals, Inc.

Study Contact

Alexion Pharmaceuticals, Inc. (Sponsor)
1-855-752-2356
clinicaltrials@alexion.com