BAG3 Mutation Associated Dilated Cardiomyopathy
Conditions
Keywords
ALXN2350, Gene Therapy, BAG3 Mutation Associated Dilated Cardiomyopathy
Brief 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.
Interventions
DRUGALXN2350
ALXN2350 is a gene therapy product consisting of an AAV9 capsid containing BAG3 transgene. It is administered as a single intravenous (IV) infusion.
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Baseline up to Week 78 |
Secondary
| Measure | Time frame |
|---|---|
| Part B: Number of Participants With TEAEs and SAEs | Baseline up to Week 78 |
| Parts A and B: Number of Participants With Cardiac Events at Weeks 52 and 78 | 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 | Baseline up to Week 78 |
| CFB in cardiac serum biomarker NT-pro-BNP | Weeks 52 and 78 |
Countries
Italy, Spain, United Kingdom, United States
Contacts
CONTACTAlexion Pharmaceuticals, Inc. (Sponsor)
Outcome results
None listed