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NCT Number: NCT07218887

ALXN2350 in Adult Participants With BAG3-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.

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Key information

Conditions

BAG3 Mutation Associated Dilated Cardiomyopathy

Age range

18 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Research Site, Massa, Italy

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

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

Note: Other inclusion and exclusion criteria may apply.

Treatment and study plan

ALXN2350

Drug

ALXN2350 is a gene therapy product consisting of an AAV9 capsid containing BAG3 transgene. It is administered as a single intravenous (IV) infusion.

Primary outcomes

  1. Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

    Time frame: Baseline up to Week 78

Secondary outcomes

  1. Part B: Number of Participants With TEAEs and SAEs

    Time frame: Baseline up to Week 78

  2. Parts A and B: Number of Participants With Cardiac Events at Weeks 52 and 78

    Time frame: Weeks 52 and 78

  3. Parts A and B: Time to the First Event of Death, Heart Transplant, Mechanical Circulating Support, or Aborted Sudden Cardiac Death

    Time frame: Baseline up to Week 78

Study contacts

Contact information is provided by the study sponsor or research team.

Alexion Pharmaceuticals, Inc. (Sponsor)

CONTACT

[email protected]

1-855-752-2356

Sponsors and collaborators

Lead sponsor

Alexion Pharmaceuticals, Inc.

Industry

Registry information

Official study 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

Acronym: DCMRestore

Important dates

Study start
2025
Primary completion
2032
Study completion
2032
First posted
Oct 20, 2025
Registry last updated
Jun 15, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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