Recruiting

Phase 1

Phase 2

ALXN2350ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy

Sponsor:

Alexion Pharmaceuticals, Inc.

Code:

NCT07218887

Conditions

BAG3 Mutation Associated Dilated Cardiomyopathy

Eligibility Criteria

Sex: All

Age: 18 - 70

Healthy Volunteers: Not accepted

Interventions

ALXN2350

Study Details

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

Study ID

NCT07218887

Start date

Oct 24, 2025

Status verified date

Aug, 2026

Completion date

Jan 27, 2032

Anticipated

Primary completion date

Jan 27, 2032

Anticipated

Eligibility Criteria

Sex: All

Age: 18 - 70

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

6 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: ALXN2350

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

Interventions

ALXN2350

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

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 and Locations

Central contacts

Alexion Pharmaceuticals, Inc. (Sponsor)

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

Locations

Research Site

Recruiting

Birmingham, Alabama, United States, 35233

Contacts

Nehal Vekariya

205-934-7173 nvekariya@uabmc.edu

Research Site

Recruiting

Palo Alto, California, United States, 94304

Contacts

Shannon Schultz

650-498-9617 sks01@stanford.edu

Research Site

Recruiting

Boston, Massachusetts, United States, 02115

Contacts

Alyx Vogle

617-732-6320 avogle@bwh.harvard.edu

Research Site

Recruiting

Cincinnati, Ohio, United States, 45219

Contacts

Sara Black

513-585-1777 sara.black@thechristhospital.com

Research Site

Recruiting

Portland, Oregon, United States, 97239-3098

Contacts

Hailey Volk

503-418-2246 volkh@ohsu.edu

Research Site

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Joseph Puhl

267-399-3049 joseph.puhl@pennmedicine.upenn.edu

Research Site

Recruiting

Houston, Texas, United States, 77030

Contacts

Jennifer Garrett

346-238-4516

More Information

Sponsor

Alexion Pharmaceuticals, Inc.

Last update posted

Aug 28, 2026

Last verified

Aug, 2026

Keywords

ALXN2350
Gene Therapy
BAG3 Mutation Associated Dilated Cardiomyopathy

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-22. This information was provided to ClinicalTrials.gov by Alexion Pharmaceuticals, Inc. on 2026-08-28.