ALXN2350 for Dilated Cardiomyopathy
(DCMRestore Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial examines a new treatment, ALXN2350, for individuals with dilated cardiomyopathy (DCM) linked to a BAG3 gene mutation. The goal is to determine if a single IV infusion of ALXN2350 is safe and effective. Participants must have this genetic mutation, a history of DCM, and be on a stable heart failure treatment plan. The trial will test different doses to identify the most effective one. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the chance to be among the first to receive it.
Is there any evidence suggesting that ALXN2350 is likely to be safe for humans?
Research shows that ALXN2350 is under investigation to determine its safety and tolerability. This early-phase trial (Phase 1/2) primarily focuses on these aspects. While earlier studies have not provided much solid information about its safety, early-phase trials typically involve careful monitoring to identify any potential side effects.
Early-phase studies like this one generally aim to find the right dose and check for unwanted side effects. Reaching this stage indicates some initial belief in the treatment's potential safety for human testing. Participants in these trials often act as pioneers, aiding researchers in understanding how a new treatment functions in the body.12345Why do researchers think this study treatment might be promising?
Unlike the standard treatments for dilated cardiomyopathy, which often include medications like beta-blockers and ACE inhibitors, ALXN2350 is unique because it introduces a novel mechanism of action. This experimental treatment is designed to target the underlying causes of the disease at a molecular level, which could potentially address issues that current treatments do not. Researchers are excited about ALXN2350 because it promises a more targeted approach, possibly leading to better outcomes and fewer side effects for patients.
What evidence suggests that ALXN2350 might be an effective treatment for dilated cardiomyopathy?
Research shows that ALXN2350 is a gene therapy designed to help people with dilated cardiomyopathy (DCM), linked to a change in the BAG3 gene. This treatment is currently being tested in this trial, where participants will receive one of three dose levels of ALXN2350, depending on the cohort. Early results are promising. ALXN2350 works by repairing the faulty gene that causes heart problems in these patients. By addressing the root cause, it aims to improve heart function and quality of life. Although detailed data from human trials remains limited, the underlying science suggests that ALXN2350 could effectively treat this specific heart condition.12345
Are You a Good Fit for This Trial?
This trial is for adults with a specific heart condition called BAG3-associated Dilated Cardiomyopathy (DCM). Participants must have a confirmed mutation in the BAG3 gene, a history of DCM diagnosis, and be on stable heart failure medications. They also need to have good enough heart imaging quality for echocardiography.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single IV infusion of ALXN2350 at one of three dose levels
Follow-up
Participants are monitored for safety, tolerability, and efficacy after the infusion
What Are the Treatments Tested in This Trial?
Interventions
- ALXN2350
Find a Clinic Near You
Who Is Running the Clinical Trial?
Alexion Pharmaceuticals, Inc.
Lead Sponsor
Dr. Alberto R. Martinez
Alexion Pharmaceuticals, Inc.
Chief Medical Officer since 2010
MD from University of Sao Paulo
Marc Dunoyer
Alexion Pharmaceuticals, Inc.
Chief Executive Officer since 2021
PhD in Molecular Biology