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Recruiting
NCT07218887
ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy
Conditions: BAG3 Mutation Associated Dilated Cardiomyopathy
Sex: All
Ages: 18 Years – 70 Years
Healthy volunteers: No
Phase: PHASE1, PHASE2
Enrollment: 6
Sponsor: Alexion Pharmaceuticals, Inc.
Location: Research Site Birmingham Alabama
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.
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.
Source: ClinicalTrials.gov (NCT07218887). StuddyBuddy aggregates publicly available trial information.