ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy
Key facts
- Where
- Birmingham, Boston, Cincinnati, Houston, Palo Alto, Philadelphia, and Portland, United StatesSee the 7 sites
- Phase
- Phase 1/2
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 70 years old?
Source: NCT07218887, who can take part
Where in United States
- Birmingham, AlabamaHospital not named in the registryContact: Nehal VekariyaTaking people
- Boston, MassachusettsHospital not named in the registryContact: Alyx VogleTaking people
- Cincinnati, OhioHospital not named in the registryContact: Sara BlackTaking people
- Houston, TexasHospital not named in the registryContact: Jennifer GarrettTaking people
- Palo Alto, CaliforniaHospital not named in the registryContact: Shannon SchultzTaking people
- Philadelphia, PennsylvaniaHospital not named in the registryContact: Joseph PuhlTaking people
- Portland, OregonHospital not named in the registryContact: Hailey VolkTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- Alexion Pharmaceuticals, Inc.
- Trial code
- NCT07218887EU 2024-519674-40-00
- Record updated
- 28 August 2026
- Original description
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.Read full original descriptionShow less
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
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.
Inclusion: 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: Presence of antibodies to AAV9; 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; History of substance abuse that could contribute to cardiac dysfunction; Note: Other inclusion and exclusion criteria may apply; Presence of a pathogenic or likely pathogenic variant in another gene where that other gene is authoritatively recognized as causal for DCM; Decompensated HF; NYHA Class IV.