ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy (DCMRestore)

Alexion Pharmaceuticals

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: BAG3 Mutation Associated Dilated Cardiomyopathy

Treatments: Drug: ALXN2350

Study type: Interventional

Funder types: Industry

Identifiers

NCT07218887

D7030C00001

2024-519674-40-00 (Other Identifier)

Details and patient eligibility

About

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.

Enrollment: 18 estimated patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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18 participants in 1 patient group

ALXN2350

Experimental group

Description:

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

Treatment:

Drug: ALXN2350