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
  • Unmapped
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
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
  • Note: Other inclusion and exclusion criteria may apply.
Sex
Female & Male
Age
18 - 70 Years

Study Details

Medical Condition
  • Unmapped
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 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
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
  • 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
Portland, Oregon, United States, 97239-3098
Status
Recruiting