A Study With NVC-001 in Patients With LMNA-related Dilated Cardiomyopathy (SUNBEAM-LMNA)

ClinicalTrials.gov ID

NCT07606274

Lead Sponsor

Nuevocor Pte. Ltd.INDUSTRY

Overall Status

Recruiting

Study Type

Interventional

Phase

Phase 1Phase 2

Enrollment

21Estimated

Last Update Posted

Aug 7, 2026Actual

Start Date

Jun 16, 2026Actual

Primary Completion Date

Mar 2030Estimated

Completion Date

Mar 2030Estimated

Official Title

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)

Brief 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.

Detailed 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.

• LMNA-Related Dilated Cardiomyopathy

• NVC-001 - Low Dose
• NVC-001 - Intermediate Dose
• NVC-001 - High Dose

Ages Eligible for Study

Adult (18-64)Older Adult (65+)

Sexes Eligible for Study

All

Accepts Healthy Volunteers

No

Eligibility Criteria

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

Primary Purpose

Other

Allocation

Non-Randomized

Interventional Model

Sequential Assignment

Masking

None (Open Label)

Lead Sponsor

Nuevocor Pte. Ltd.INDUSTRY

info@nuevocor.com507-422-5433

info@nuevocor.com281-222-9983

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