ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-10-24

Study Completion Date

2032-01-27

Brief Summary

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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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Detailed Description

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Conditions

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BAG3 Mutation Associated Dilated Cardiomyopathy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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ALXN2350

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

Group Type EXPERIMENTAL

ALXN2350

Intervention Type DRUG

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

Interventions

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ALXN2350

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

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

18 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No