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ALXN2350 Gene Therapy for BAG3 Mutation Associated Dilated Cardiomyopathy

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Study AimThis 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

BAG3 Mutation Associated Dilated Cardiomyopathy

From 18 to 70 Years
+7 Eligibility Criteria

See all eligibility criteria

How is the trial designed

Treatment Study

Phase 1
Interventional
Study Start: December 2025

See protocol details

Summary

Principal SponsorAlexion Pharmaceuticals, Inc.
Study ContactAlexion Pharmaceuticals, Inc. (Sponsor)
Last updated: October 20, 2025
Sourced from a government-validated database.Claim as a partner
Study start date: December 31, 2025Actual 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 
Principal SponsorAlexion Pharmaceuticals, Inc.
Study ContactAlexion Pharmaceuticals, Inc. (Sponsor)
Last updated: October 20, 2025
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
18 patients to be enrolledTotal 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.

How participants are assigned to different groups/arms
In this clinical study, all participants receive the same treatment. Since there is only one group, there is no need for randomization or assignment to different arms. This type of study is often used to test a new treatment without comparing it to another.

Other Ways to Assign Participants
Randomized allocation: Participants are assigned randomly, like flipping a coin, to ensure fairness and reduce bias.
Non-randomized allocation: Participants are assigned based on specific factors, such as their medical condition or a doctor's decision.

How treatments are given to participants
In this study, all participants receive the same treatment. This approach is often used to evaluate the effects of a single intervention without comparing it to another.

Other Ways to Assign Treatments
Parallel assignment: Participants are split into separate groups, each receiving a different treatment.
Cross-over assignment: Participants switch between treatments during the study.
Factorial assignment: Participants receive different combinations of treatments.
Sequential assignment: Participants receive treatments one after another in a specific order, possibly based on individual responses.
Other assignment: Treatment assignment does not follow a standard or predefined design.

How the effectiveness of the treatment is controlled
In a non placebo-controlled study, no participants receive an inert substance (placebo) to compare outcomes. Instead, all participants receive either the experimental treatment or an alternative treatment (often the Standard of Care). This method allows researchers to compare the effects of the experimental treatment with those of a different active intervention, rather than a placebo.

Other Options
Placebo-Controlled: A placebo is used to compare the effects of the experimental treatment with those of an inert substance, isolating the true treatment effect.

How the interventions assigned to participants is kept confidential
Everyone involved in the study knows which treatment is being given. This is typically used when it's not possible or necessary to hide the treatment details from participants or researchers.

Other Ways to Mask Information
Single-blind: Participants do not know which treatment they are receiving, but researchers do.
Double-blind: Neither participants nor researchers know which treatment is given.
Triple-blind: Participants, researchers, and outcome assessors do not know which treatment is given.
Quadruple-blind: Participants, researchers, outcome assessors, and care providers all do not know which treatment is given.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria
Any sexBiological sex of participants that are eligible to enroll.
From 18 to 70 YearsRange of ages for which participants are eligible to join.
Healthy volunteers not allowedIf individuals who are healthy and do not have the condition being studied can participate.
Conditions
Pathology
BAG3 Mutation Associated Dilated Cardiomyopathy
Criteria
4 inclusion criteria required to participate
Medical history of diagnosis of DCM
Stable combination of HF SoC medications
Pathogenic or likely pathogenic mutation in BAG3
Adequate acoustic windows for echocardiography
3 exclusion criteria prevent from participating
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

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 no location dataSave this study to your profile to know when the location data is available. 
Recruiting soonNo study centers