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

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
Phases
Phase 1Phase 2
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT07606274
Enrollment
21
Registered
2026-05-26
Start date
2026-06-16
Completion date
2030-03-01
Last updated
2026-08-07

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

LMNA-Related Dilated Cardiomyopathy

Keywords

LMNA-related DCM, Dilated Cardiomyopathy, DCM

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.

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

Sponsors

Nuevocor Pte. Ltd.
Lead SponsorINDUSTRY

Study design

Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
OTHER
Masking
NONE

Eligibility

Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No

Inclusion 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

Design outcomes

Primary

MeasureTime frame
Safety: Frequency, severity, and relatedness of treatment-emergent adverse events (TEAEs)At week 26

Secondary

MeasureTime frame
Change in echocardiographic parametersUp to 104 weeks
Change from baseline in arrhythmiasUp to 104 weeks
Change in exercise capacityUp to 104 weeks
Change from baseline in quality of life (QoL)Up to 104 weeks
Incidence of clinical eventsUp to week 104

Countries

United States

Contacts

STUDY_DIRECTORMonica Shah, MD

Nuevocor Pte. Ltd.

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Aug 8, 2026