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 (Serotype 9) Vector Expressing a Dominant Negative SUN1 Transgene, in Patients With LMNA-Related Dilated Cardiomyopathy (SUNBEAM-LMNA)
Nuevocor Pte. Ltd.
Summary
This study is designed to evaluate the safety and tolerability of NVC-001 in adults with LMNA-related dilated cardiomyopathy (LMNA-DCM). The study will also assess exploratory measures of efficacy.
Description
This is a Phase 1/2, open-label study of NVC-001 in adults with LMNA-related dilated cardiomyopathy (LMNA-DCM). The primary objective is to evaluate the safety and tolerability of NVC-001. Secondary and exploratory objectives include characterization of pharmacodynamic effects and assessment of preliminary efficacy.
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
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 opi…