Taking people in ItalyPhase 1/2
ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy
Key facts
- Who runs it
- Alexion Pharmaceuticals, Inc.
- Where
- Massa, ItalySee the siteAlso in 4 other countries, 11 sites
- Trial code
- Status
- Recruiting
- Phase
- Phase 1/2
- People taking part
- 6 people, planned
- Started
- 24 Oct 2025
- Expected to end
- 27 Jan 2032
- Type of study
- Interventional
- Treatments tested
- ALXN2350
- Record updated
- 28 August 2026
Who can take part
- Age
- 18 years to 70 years
- Sex
- Women and men
- Healthy volunteers
- Cannot take part
The registry criteria, word for word
Show all 14 criteriaShow less
Who can take part
- 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
Who cannot take part
- 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
Check if you might fit
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?
Where in Italy
Also in 4 other countries, 11 sites
- Fondazione Toscana Gabriele MonasterioMassa, ItalyTaking people
About the trial
What the trial wants to find out
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.
The group in the trial
- ALXN2350Treatment being tested
Participants will receive one of three dose levels of ALXN2350 depending on the cohort.
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 doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
- Official 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
- Fit questions
- Built from NCT07218887, who can take part
- Record updated
- 28 August 2026