RecruitingPhase 1Phase 2NCT07218887

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


Sponsor

Alexion Pharmaceuticals, Inc.

Enrollment

6 participants

Start Date

Oct 24, 2025

Study Type

INTERVENTIONAL

Conditions

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.


Eligibility

Min Age: 18 YearsMax Age: 70 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called ALXN2350 for people with bag3 mutation associated dilated cardiomyopathy. The study is currently recruiting participants at 7 locations. People eligible for this study include aged 18 Years to 70 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGALXN2350

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

View Full Details on ClinicalTrials.gov

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NCT07218887