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 (4) 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
Massachusetts
Research Site
RECRUITING
Boston
Oregon
Research Site
RECRUITING
Portland
Texas
Research Site
RECRUITING
Houston
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: 6
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.