ALXN2350 for adults with BAG3-related 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

This study will test whether a single IV infusion of ALXN2350 is safe and helps adults who have dilated cardiomyopathy caused by a BAG3 mutation.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 6 (estimated)
• Ages: 18 Years to 70 Years
• Sex: All
• Sponsor: Alexion Pharmaceuticals, Inc. (Industry-sponsored)
• Locations: 7 sites (Birmingham, Alabama and 6 other locations)
• Trial ID: NCT07218887 on ClinicalTrials.gov

What this trial studies

This is an open-label Phase 1/2 dose-finding and dose-expansion study that gives a single intravenous infusion of ALXN2350 to adults with genetically confirmed BAG3-associated DCM. The primary focus is safety and tolerability across escalating doses, with expansion cohorts used to look for preliminary signs of clinical benefit using cardiac imaging and heart-failure measures. Key eligibility includes a pathogenic or likely pathogenic BAG3 variant, stable guideline-directed heart failure therapy, and adequate echocardiographic windows, while participants with pre-existing AAV9 antibodies or another known genetic cause of DCM are excluded. Study procedures include baseline and follow-up clinical assessments and imaging at U.S. sites to monitor efficacy signals and adverse events after the infusion.

Who should consider this trial

Good fit: Adults with a pathogenic or likely pathogenic BAG3 mutation, a clinical diagnosis of dilated cardiomyopathy, stable heart-failure medications, and adequate echocardiographic windows are ideal candidates.

Not a fit: People with pre-existing antibodies to AAV9, a different authoritative genetic cause of DCM, decompensated heart failure, or other exclusion criteria are unlikely to receive benefit from this treatment.

Why it matters

Potential benefit: If successful, ALXN2350 could offer a one-time, gene-targeted treatment that stabilizes or improves heart function in adults with BAG3-associated DCM.

How similar studies have performed: AAV9-based gene therapies have shown success in other genetic disorders, but gene therapy for BAG3-associated dilated cardiomyopathy is largely novel and unproven in humans.

Eligibility criteria

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.

Where this trial is running

Birmingham, Alabama and 6 other locations

• Research Site — Birmingham, Alabama, United States (Recruiting)
• Research Site — Boston, Massachusetts, United States (Recruiting)
• Research Site — Cincinnati, Ohio, United States (Recruiting)
• Research Site — Portland, Oregon, United States (Recruiting)
• Research Site — Houston, Texas, United States (Recruiting)
• Research Site — Barcelona, Spain (Recruiting)
• Research Site — Majadahonda, Spain (Recruiting)

Study contacts

• Study coordinator: Alexion Pharmaceuticals, Inc. (Sponsor)

Conditions BAG3 Mutation Associated Dilated Cardiomyopathy; ALXN2350; Gene Therapy