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Clinical Trials in the UK / NCT07218887
Recruiting Phase 1/2

ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy

NCT07218887 · tracked via the Priya Life Science UK tracker
Sponsor
Alexion Pharmaceuticals, Inc.
Phase
Phase 1/2
Started
2025-10-24
Last updated
2026-08-28

Condition(s) studied

BAG3 Mutation Associated Dilated Cardiomyopathy

Investigational drug(s) / intervention(s)

ALXN2350

ALXN2350: ALXN2350 is a gene therapy product consisting of an AAV9 capsid containing BAG3 transgene. It is administered as a single intravenous (IV) infusion.

Study 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

Sex
ALL
Min age
18 Years
Max age
70 Years
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 * LVEF \> 15% and ≤ 45% at Screening by echocardiography * Diagnosis of chronic HF for at least 3 months * NYHA Class I/II/III at Screening 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 * Cardiac ventricular arrhythmia that requires treatment * History of untreated clinically significant valve disease or a Screening confirmation of severe aortic stenosis, severe mitral stenosis, moderate or severe aortic insufficiency or severe mitral insufficiency * NYHA Class IV * History of substance abuse that could contribute to cardiac dysfunction Note: Other inclusion and exclusion criteria may apply.

Primary outcome measure(s)

Trial sites (11)

FacilityCityRegionStatus
Research Site Birmingham Alabama Recruiting
Research Site Palo Alto California Recruiting
Research Site Boston Massachusetts Recruiting
Research Site Cincinnati Ohio Recruiting
Research Site Portland Oregon Recruiting
Research Site Philadelphia Pennsylvania Recruiting
Research Site Houston Texas Recruiting
Research Site Massa Italy Recruiting
Research Site Barcelona Spain Recruiting
Research Site Majadahonda Spain Recruiting
Research Site London United Kingdom Recruiting

More Alexion Pharmaceuticals, Inc. trials in the UK

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07218887 on ClinicalTrials.gov ↗ ← All trials in the UK