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
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental ALXN2350 |
Participants will receive one of three dose levels of ALXN2350 depending on the cohort. |
|
Recruiting Locations
Research Site
Birmingham 4049979, Alabama 4829764 35233
Birmingham 4049979, Alabama 4829764 35233
More Details
- Status
- Recruiting
- Sponsor
- Alexion Pharmaceuticals, Inc.