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 will be conducted to evaluate the safety and tolerability, and assess exploratory efficacy of NVC-001 in adults participants with LMNA-Related Dilated Cardiomyopathy.
Description
This study consists of an open-label single ascending dose part and an expansion phase to evaluate the safety and preliminary efficacy of NVC-001 in participants with LMNA-Related Dilated Cardiomyopathy
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: * Able to provide full informed consent and comply with all requirements of the study protocol * Male or female participants ≥18 years old * Pathogenic or likely pathogenic mutation of LMNA * NYHA Class I, II, or III * Anti-AAV9 neutralizing antibody titer per protocol-specified criteria * Adequate hepatic and renal function per protocol-specified criteria Key Exclusion Criteria: * Any chronic medical condition that in the opinion of the Investigator would compromise the safety and/or compliance of the enrolled participant * Any clinically relevant change in standard…
Interventions
- GeneticNVC-001 - Low Dose
Solution for intravenous infusion
- GeneticNVC-001 - Intermediate Dose
Solution for intravenous infusion
- GeneticNVC-001 - High Dose
Solution for intravenous infusion
Location
- Cedars-Sinai Medical CenterLos Angeles, California