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ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy
Sponsor: Alexion Pharmaceuticals, Inc.
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.
Official title: A Phase 1/2, Open-Label, Multicenter, Dose Finding and Dose Expansion Study to Investigate the Safety, Tolerability, and Efficacy of ALXN2350 Gene Therapy in Adult Participants With BAG3 Mutation Associated Dilated Cardiomyopathy
Key Details
Gender
All
Age Range
18 Years - 70 Years
Study Type
INTERVENTIONAL
Enrollment
6
Start Date
2025-10-24
Completion Date
2032-01-27
Last Updated
2026-03-04
Healthy Volunteers
No
Interventions
ALXN2350
ALXN2350 is a gene therapy product consisting of an AAV9 capsid containing BAG3 transgene. It is administered as a single intravenous (IV) infusion.
Locations (7)
Research Site
Birmingham, Alabama, United States
Research Site
Boston, Massachusetts, United States
Research Site
Cincinnati, Ohio, United States
Research Site
Portland, Oregon, United States
Research Site
Houston, Texas, United States
Research Site
Barcelona, Spain
Research Site
Majadahonda, Spain