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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
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

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data 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