NCT07218887

ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 6 (estimated)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

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.

Conditions

• BAG3 Mutation Associated Dilated Cardiomyopathy

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2025-10-24
• Primary completion: 2032-01-27
• Completion: 2032-01-27
• First posted: 2025-10-20
• Last updated: 2026-03-04

Locations

7 sites across 2 countries: United States, Spain

Regulatory

• FDA-regulated drug study