A Phase 1/2 interventional study of ALXN2350 in BAG3 Mutation Associated Dilated Cardiomyopathy, sponsored by Alexion Pharmaceuticals, Inc.. Recruiting at 11 sites in 4 countries. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2026-08-28.
Sponsored by Alexion Pharmaceuticals, Inc. (part of AstraZeneca) · Phase 1/2, Interventional, and Treatment
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.
255 studies on the registry are indexed under Cardiomyopathy, Dilated; 66 are open to participants now.
This study's planned enrollment of 6 is below the median of 43 across 145 interventional studies indexed under Cardiomyopathy, Dilated.
Browse Cardiomyopathy, Dilated studies →Alexion Pharmaceuticals, Inc. is the lead sponsor of 255 studies on the registry; 23 are open to participants now.
Of its 101 completed or terminated interventional studies of FDA-regulated products, 75 (74%) have results posted.
First submitted after Alexion Pharmaceuticals, Inc. became part of AstraZeneca.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Note: Other inclusion and exclusion criteria may apply.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants will receive one of three dose levels of ALXN2350 depending on the cohort.
Drug: ALXN2350
ALXN2350 is a gene therapy product consisting of an AAV9 capsid containing BAG3 transgene. It is administered as a single intravenous (IV) infusion.
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
CFB in cardiac serum biomarker NT-pro-BNP
Time frame: Weeks 52 and 78
Plan to share: Yes — Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
Supporting information: Study protocol, Statistical analysis plan, Clinical study report
No publications or documents are linked to this record.
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