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
Palo Alto, California, United States
RECRUITINGPart 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
CFB in cardiac serum biomarker NT-pro-BNP
Time frame: Weeks 52 and 78
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Research Site
Boston, Massachusetts, United States
Research Site
Cincinnati, Ohio, United States
RECRUITINGResearch Site
Portland, Oregon, United States
RECRUITINGResearch Site
Philadelphia, Pennsylvania, United States
RECRUITINGResearch Site
Houston, Texas, United States
RECRUITINGResearch Site
Massa, Italy
RECRUITINGResearch Site
Barcelona, Spain
RECRUITINGResearch Site
Majadahonda, Spain
RECRUITING...and 1 more locations