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ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy

RecruitingPhase 1, Phase 2

Sponsor: Alexion Pharmaceuticals, Inc.

Conditions: BAG3 Mutation Associated Dilated Cardiomyopathy

Interventions: ALXN2350

Countries: United States, Italy, Spain, United Kingdom

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 overview

Sex: ALL

Age: 18 Years to 70 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

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
* 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.

Locations (11)

Birmingham, Alabama, United States
Palo Alto, California, United States
Boston, Massachusetts, United States
Cincinnati, Ohio, United States
Portland, Oregon, United States
Philadelphia, Pennsylvania, United States
Houston, Texas, United States
Massa, Italy
Barcelona, Spain
Majadahonda, Spain
London, United Kingdom