What is the Purpose of this Study?
This study focuses on people who have lamin A/C protein-related dilated cardiomyopathy (LMNA-related DCM), a condition in which the heart muscle becomes weak and enlarged, making it harder for the heart to pump blood and potentially causing problems with the heart’s rhythm. The purpose of this study is to evaluate an experimental gene therapy medication called NVC-001. When a gene is missing or not working correctly, diseases like LMNA-related DCM can occur. Gene therapy is a way of treating disease by giving cells new instructions by injecting pieces of genes in the body. In this study, researchers will use a modified virus (AAV vector) that carries the genes for making an altered version of a natural protein that is normally part of the structure of the heart cells. The shortened protein helps protect the heart by decreasing the excess mechanical stress that the damaged LMNA gene causes in heart cells as they beat. Researchers aim to determine whether NVC-001 is safe, causes any side effects, and helps improve heart function. All participants will receive NVC-001; it will be given by infusion into a vein in the arm.
Eligibility
Not Available
Where can I participate?
Beverly
What is the full name of this clinical trial?
A Phase 1/2, First-in-Human, Open-Label, Multicenter, Single-Dose Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of NVC-001, a Novel Adeno-Associated Virus Vector Expressing a Dominant Negative SUN1 Transgene, in Patients with LMNA-Related Dilated Cardiomyopathy