Recruiting

DCMRestoreALXN2350 Gene Therapy for BAG3 Mutation Associated Dilated Cardiomyopathy

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Study Aim

This study aims to treat dilated cardiomyopathy caused by BAG3 mutations using ALXN2350 gene therapy in adults. It will assess any treatment emergent and serious adverse events in participants.

What is being tested

ALXN2350

Drug
Who is being recruted

From 18 to 70 Years
+7 Eligibility Criteria
See all eligibility criteria
How is the trial designed

Treatment Study

Phase 1 & 2
Interventional
Study Start: October 2025
See protocol details

Summary

Principal SponsorAlexion Pharmaceuticals, Inc.
Study ContactAlexion Pharmaceuticals, Inc. (Sponsor)
Last updated: February 14, 2026
Sourced from a government-validated database.Claim as a partner

Study start date: October 24, 2025

Actual date on which the first participant was enrolled.

This study is exploring a new treatment option for adults who have a specific heart condition called dilated cardiomyopathy (DCM) due to a BAG3 gene mutation. The main aim is to find out how safe and effective a new gene therapy, named ALXN2350, can be for these patients. This is important as it could lead to better treatment options for individuals affected by this genetic heart issue, potentially improving their quality of life and health outcomes. Participants in the study will receive a single dose of ALXN2350 through an intravenous (IV) infusion, which means the treatment is given directly into the bloodstream. The study focuses on evaluating how well patients can tolerate the treatment and how safe it is for them. By monitoring these aspects, researchers hope to determine the most effective dose with the least side effects, paving the way for future studies and potential treatments.

Official TitleA 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
NCT07218887
Principal SponsorAlexion Pharmaceuticals, Inc.
Study ContactAlexion Pharmaceuticals, Inc. (Sponsor)
Last updated: February 14, 2026
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

6 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 18 to 70 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Criteria

4 inclusion criteria required to participate
Pathogenic or likely pathogenic mutation in BAG3
Medical history of diagnosis of DCM
Stable combination of HF SoC medications
Adequate acoustic windows for echocardiography
3 exclusion criteria prevent from participating
Decompensated HF
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.

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives

One single intervention group is designated in this study

This study does not include a placebo group 

Treatment Groups

Group I

Experimental
Participants will receive one of three dose levels of ALXN2350 depending on the cohort.

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 4 locations

Recruiting

Research Site

Birmingham, United StatesOpen Research Site in Google Maps
Recruiting

Research Site

Boston, United States
Recruiting

Research Site

Portland, United States
Recruiting

Research Site

Houston, United States
Recruiting
4 Study Centers