A Phase 1/2, Open-Label, Multicenter, Dose Finding and Dose Expansion Study to Investigate the Safety, Tolerability, and Efficacy of ALXN2350 Gene Therapy in Adult Participants With BAG3 Mutation Associated Dilated Cardiomyopathy
Status: Recruiting
Location: See all (2) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 70
Healthy Volunteers: f
View:
• Pathogenic or likely pathogenic mutation in BAG3
• Medical history of diagnosis of DCM
• Stable combination of HF SoC medications
• Adequate acoustic windows for echocardiography
Locations
United States
Alabama
Research Site
RECRUITING
Birmingham
Oregon
Research Site
RECRUITING
Portland
Contact Information
Primary
Alexion Pharmaceuticals, Inc. (Sponsor)
clinicaltrials@alexion.com
1-855-752-2356
Time Frame
Start Date: 2025-10-24
Estimated Completion Date: 2032-01-27
Participants
Target number of participants: 18
Treatments
Experimental: ALXN2350
Participants will receive one of three dose levels of ALXN2350 depending on the cohort.
Related Therapeutic Areas
Sponsors
Leads: Alexion Pharmaceuticals, Inc.