ALXN2350: ALXN2350 is a gene therapy product consisting of an AAV9 capsid containing BAG3 transgene. It is administered as a single intravenous (IV) infusion.
Study 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
Sex
ALL
Min age
18 Years
Max age
70 Years
Healthy volunteers
No
Inclusion Criteria:
* Pathogenic or likely pathogenic mutation in BAG3
* Medical history of diagnosis of DCM
* Stable combination of HF SoC medications
* Adequate acoustic windows for echocardiography
* LVEF \> 15% and ≤ 45% at Screening by echocardiography
* Diagnosis of chronic HF for at least 3 months
* NYHA Class I/II/III at Screening
Exclusion Criteria:
* Presence of antibodies to AAV9
* Presence of a pathogenic or likely pathogenic variant in another gene where that other gene is authoritatively recognized as causal for DCM
* Decompensated HF
* Cardiac ventricular arrhythmia that requires treatment
* History of untreated clinically significant valve disease or a Screening confirmation of severe aortic stenosis, severe mitral stenosis, moderate or severe aortic insufficiency or severe mitral insufficiency
* NYHA Class IV
* History of substance abuse that could contribute to cardiac dysfunction
Note: Other inclusion and exclusion criteria may apply.
Primary outcome measure(s)
Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — Baseline up to Week 78
Trial sites (11)
Facility
City
Region
Status
Research Site
Birmingham
Alabama
Recruiting
Research Site
Palo Alto
California
Recruiting
Research Site
Boston
Massachusetts
Recruiting
Research Site
Cincinnati
Ohio
Recruiting
Research Site
Portland
Oregon
Recruiting
Research Site
Philadelphia
Pennsylvania
Recruiting
Research Site
Houston
Texas
Recruiting
Research Site
Massa
Italy
Recruiting
Research Site
Barcelona
Spain
Recruiting
Research Site
Majadahonda
Spain
Recruiting
Research Site
London
United Kingdom
Recruiting
More Alexion Pharmaceuticals, Inc. trials in the UK
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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