Menu
Recruiting NCT07218887

ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy

Phase I / Phase II Interventional BAG3 Mutation Associated Dilated Cardiomyopathy

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: ALXN2350.
Who it may be relevant to
Registry conditions: BAG3 Mutation Associated Dilated Cardiomyopathy. Basic parameters: 18 years — 70 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Italy, Spain, United Kingdom
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official 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

Overview

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.

Interventions

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

Primary outcome measures

  • Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [Time frame: Baseline up to Week 78]
Secondary outcome measures (3)
  • Part B: Number of Participants With TEAEs and SAEs [Time frame: Baseline up to Week 78]
  • Parts A and B: Number of Participants With Cardiac Events at Weeks 52 and 78 [Time frame: Weeks 52 and 78]
  • 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]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 6 centers
  • Research Site — Birmingham
  • Research Site — Boston
  • Research Site — Cincinnati
  • Research Site — Portland
  • Research Site — Philadelphia
  • Research Site — Houston
Spain · 2 centers
  • Research Site — Barcelona
  • Research Site — Majadahonda
Italy · 1 center
  • Research Site — Massa
United Kingdom · 1 center
  • Research Site — London

Identifiers

NCT: NCT07218887 · D7030C00001 · 2024-519674-40-00

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗