ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy

Study Identifier:
D7030C00001
ClinicalTrials.gov Identifier:
NCT07218887
EudraCT Identifier:
N/A
EU CT ID:
2024-519674-40-00
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
1-855-752-2356 or [email protected]
Recruiting

Study Details

Medical Condition
  • Cardiomyopathy
Study Drug
  • Drug: ALXN2350
Date
Oct 2025 - Jan 2032
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18 - 70 Years
Requirements Information
Inclusion and Exclusion 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.
Sex
Female & Male
Age
18 - 70 Years

Study Details

Medical Condition
  • Cardiomyopathy
Study Drug
  • Drug: ALXN2350
Date
Oct 2025 - Jan 2032
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18 - 70 Years
Requirements Information
Inclusion and Exclusion 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.

Protocol 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.

Trial Locations

Location
Status
Location
Research Site
Birmingham, Alabama, United States, 35233
Status
Recruiting
Location
Research Site
Palo Alto, California, United States, 94304
Status
Recruiting
Location
Research Site
Boston, Massachusetts, United States, 02115
Status
Recruiting
Location
Research Site
Cincinnati, Ohio, United States, 45219
Status
Recruiting
Location
Research Site
Portland, Oregon, United States, 97239-3098
Status
Recruiting
Location
Research Site
Philadelphia, Pennsylvania, United States, 19104
Status
Recruiting
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