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)
Status: Recruiting
Location: See all (8) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:
• Adult ≥18 years old, able to provide informed consent
• Clinical diagnosis of LMNA-related dilated cardiomyopathy (DCM) with LV systolic dysfunction
• Pathogenic or likely pathogenic LMNA mutation confirmed by a CLIA-certified/CAP-accredited laboratory
• NYHA Functional Class I, II, or III
• LVEF \>25% and ≤45% by echocardiography
• Implantable cardioverter-defibrillator (ICD) or cardiac resynchronization therapy-defibrillator )CRT-D) implanted \>3 months prior to consent
Locations
United States
Alabama
University of Alabama in Birmingham
NOT_YET_RECRUITING
Birmingham
California
Cedars-Sinai Medical Center
RECRUITING
Los Angeles
Massachusetts
Brigham and Women's Hospital
NOT_YET_RECRUITING
Boston
Michigan
University of Michigan
NOT_YET_RECRUITING
Ann Arbor
Minnesota
Mayo Clinic
RECRUITING
Rochester
Ohio
The Christ Hospital
NOT_YET_RECRUITING
Cincinnati
The Ohio State University
NOT_YET_RECRUITING
Columbus
Texas
Houston Methodist Hospital
RECRUITING
Houston
Time Frame
Start Date: 2026-06-16
Estimated Completion Date: 2030-03
Participants
Target number of participants: 21
Treatments
Experimental: SAD low dose
Single Ascending Dose (SAD): participants will receive low dose of NVC-001
Experimental: SAD medium dose
Single Ascending Dose (SAD): participants will receive medium dose of NVC-001
Experimental: SAD high dose
Single Ascending Dose (SAD): participants will receive high dose of NVC-001
Related Therapeutic Areas
Sponsors
Leads: Nuevocor Pte. Ltd.