ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy

NCT07218887 | Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: D7030C000012024-519674-40-00
• Study Type: interventional
• Target: 6
• Locations: 2 countries
• Sites: 7

Brief 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

Keywords

ALXN2350; Gene Therapy; BAG3 Mutation Associated Dilated Cardiomyopathy

Outcome Measures

Primary Outcomes (1)

• Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

  Baseline up to Week 78

Secondary Outcomes (3)

• Part B: Number of Participants With TEAEs and SAEs

  Baseline up to Week 78

• Parts A and B: Number of Participants With Cardiac Events at Weeks 52 and 78

  Weeks 52 and 78

• Parts A and B: Time to the First Event of Death, Heart Transplant, Mechanical Circulating Support, or Aborted Sudden Cardiac Death

  Baseline up to Week 78

Study Arms (1)

ALXN2350

EXPERIMENTAL

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

Drug: ALXN2350

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.

ALXN2350

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Pathogenic or likely pathogenic mutation in BAG3
• Medical history of diagnosis of DCM
• Stable combination of HF SoC medications
• Adequate acoustic windows for echocardiography

You may not qualify if:

• 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

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (7)

Research Site

Birmingham, Alabama, 35233, United States

RECRUITING

Research Site

Boston, Massachusetts, 02115, United States

RECRUITING

Research Site

Cincinnati, Ohio, 45219, United States

RECRUITING

Research Site

Portland, Oregon, 97239-3098, United States

RECRUITING

Research Site

Houston, Texas, 77030, United States

RECRUITING

Research Site

Barcelona, 08035, Spain

RECRUITING

Research Site

Majadahonda, 28222, Spain

RECRUITING

Central Study Contacts

Alexion Pharmaceuticals, Inc. (Sponsor)

CONTACT

1-855-752-2356; clinicaltrials@alexion.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 17, 2025
• First Posted: October 20, 2025
• Study Start: October 24, 2025
• Primary Completion (Estimated): January 27, 2032
• Study Completion (Estimated): January 27, 2032
• Last Updated: March 4, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR

Locations

US (5); ES (2)