ALXN2350-DCM-201- ALXN2350 in adult participants with BAG3-associated dilated cardiomyopathy
Research type
Research Study
Full title
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
IRAS ID
1011568
Contact name
Jonathan Lu
Contact email
Sponsor organisation
Alexion Pharmaceuticals, Inc.
Research summary
The purpose of this study are to find the appropriate dose of gene therapy (ALXN2350) and see whether ALXN350 is safe and works well to treat BAG-3 associated dilated cardiomyopathy in adult participants. This study aims to assess and evaluate, but not limited to, the following changes after ALXN2350 administration: the heart structure and how the heart works; how many new cases of heart related events occur; participant's quality of life; the function of the body's natural defence system.
Dilated cardiomyopathy is a chronic and progressive condition that affects the normal functioning of the heart muscle and can lead to heart failure. This condition results in the heart muscle walls becoming stretched and thinned, thus resulting in the heart not being able to squeeze and pump sufficient blood around the body.
ALXN2350 is a gene therapy that works by transferring a normal BAG3 gene into the heart to either replace a missing or abnormal BAG3 gene. This transfer is done using a virus called AAV9, which acts as a ‘transporter’ carrying the correct BAG3 gene into the heart.
In the first part of this study (Part A), participants will be dosed across separate cohorts with differing ALXN2350 dose levels. This is done to find the appropriate, target ALXN2350 dose to give to participants in the second part of the study (Part B). Both Part A & B consist of 4 phases: Screening to determine if a patient is eligible to take part in the study, single ALXN2350 dose intravenous infusion & Impatient Visits, Short-term and Long-term Follow-up Visits.
The total study duration is about 5 years.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
25/SC/0164
Date of REC Opinion
25 Jul 2025
REC opinion
Further Information Favourable Opinion