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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
6
ALXN2350 is a gene therapy product consisting of an AAV9 capsid containing BAG3 transgene. It is administered as a single intravenous (IV) infusion.
Research Site
Birmingham, Alabama, United States
RECRUITINGResearch Site
Boston, Massachusetts, United States
RECRUITINGResearch Site
Cincinnati, Ohio, United States
Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: Baseline up to Week 78
Part B: Number of Participants With TEAEs and SAEs
Time frame: Baseline up to Week 78
Parts A and B: Number of Participants With Cardiac Events at Weeks 52 and 78
Time frame: Weeks 52 and 78
Parts A and B: Time to the First Event of Death, Heart Transplant, Mechanical Circulating Support, or Aborted Sudden Cardiac Death
Time frame: Baseline up to Week 78
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Research Site
Portland, Oregon, United States
RECRUITINGResearch Site
Houston, Texas, United States
RECRUITINGResearch Site
Barcelona, Spain
RECRUITINGResearch Site
Majadahonda, Spain
RECRUITING