This study examines NVC-001 gene therapy at three dose levels in adults with dilated cardiomyopathy caused by mutations in the LMNA gene. Participants are aged 18 and over, have left ventricular systolic dysfunction and carry a pathogenic or likely pathogenic LMNA variant. It is a Phase 1/2 study and is currently recruiting.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Key Inclusion Criteria: 1. Adult ≥18 years old, able to provide informed consent 2. Clinical diagnosis of LMNA-related dilated cardiomyopathy (DCM) with LV systolic dysfunction 3. Pathogenic or likely pathogenic LMNA mutation confirmed by a CLIA-certified/CAP-accredited laboratory 4. NYHA Functional Class I, II, or III 5. LVEF \>25% and ≤45% by echocardiography 6. Implantable cardioverter-defibrillator (ICD) or cardiac resynchronization therapy-defibrillator )CRT-D) implanted \>3 months prior to consent Key Exclusion Criteria: 1. Any chronic medical condition that, in the Investigator's opinion, would compromise participant safety or compliance 2. Hospitalization as an inpatient for the following conditions within 3 months prior to signing the ICF: Heart failure, Persistent/refractory ventricular arrhythmia requiring external defibrillation, or multiple/recurrent cardioversion/defibrillation by ICD Indication for, or history of, LV assist device or cardiac transplantation due to end-stage cardiac dysfunction 3. Current or anticipated need for chronic IV inotropic therapy