ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy

Conditions

• BAG3 Mutation Associated Dilated Cardiomyopathy

Eligibility Criteria

Sex

ALL

Age

18 Years - 70 Years

Healthy Volunteers

Not accepted

Interventions

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

Study Details

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.

Key Dates

First listed

Oct 20, 2025

Start date

Oct 24, 2025

Status verified

Jun 2026

Primary completion

Jan 27, 2032

Completion

Jan 27, 2032

Study Design

Enrollment

6 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: ALXN2350
  Participants will receive one of three dose levels of ALXN2350 depending on the cohort.

Primary Outcome Measure

Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [ Time Frame: Baseline up to Week 78 ]

Central Contacts

• Alexion Pharmaceuticals, Inc. (Sponsor)
  1-855-752-2356
  [email protected]

Locations (6)

Find similar trials in Birmingham, AL

Related Studies

• An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)
  PHASE1/PHASE2 · Recruiting · Affinia Therapeutics · Miami, Florida