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

July 2, 2026 updated by: Nuevocor Pte. Ltd.

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)

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

21

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • Not yet recruiting
        • University of Alabama in Birmingham
    • California
      • Los Angeles, California, United States, 90048
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Not yet recruiting
        • Brigham and Women's Hospital
    • Minnesota
      • Rochester, Minnesota, United States, 55905
    • Ohio
      • Cincinnati, Ohio, United States, 45219
        • Not yet recruiting
        • The Christ Hospital
    • Texas
      • Houston, Texas, United States, 77030

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Other
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: SAD low dose
Single Ascending Dose (SAD): participants will receive low dose of NVC-001
Genetic: NVC-001 - Low Dose
Solution for intravenous infusion
Experimental: SAD medium dose
Single Ascending Dose (SAD): participants will receive medium dose of NVC-001
Genetic: NVC-001 - Intermediate Dose
Solution for intravenous infusion
Experimental: SAD high dose
Single Ascending Dose (SAD): participants will receive high dose of NVC-001
Genetic: NVC-001 - High Dose
Solution for intravenous infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Safety: Frequency, severity, and relatedness of treatment-emergent adverse events (TEAEs)
Time Frame: At week 26
At week 26

Secondary Outcome Measures

Outcome Measure
Time Frame
Change in echocardiographic parameters
Time Frame: Up to 104 weeks
Up to 104 weeks
Change from baseline in arrhythmias
Time Frame: Up to 104 weeks
Up to 104 weeks
Change in exercise capacity
Time Frame: Up to 104 weeks
Up to 104 weeks
Change from baseline in quality of life (QoL)
Time Frame: Up to 104 weeks
Up to 104 weeks
Incidence of clinical events
Time Frame: Up to week 104
Up to week 104

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Nuevocor Pte. Ltd.

Investigators

  • Study Director: Monica Shah, MD, Nuevocor Pte. Ltd.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 16, 2026

Primary Completion (Estimated)

March 1, 2030

Study Completion (Estimated)

March 1, 2030

Study Registration Dates

First Submitted

May 6, 2026

First Submitted That Met QC Criteria

May 18, 2026

First Posted (Actual)

May 26, 2026

Study Record Updates

Last Update Posted (Actual)

July 7, 2026

Last Update Submitted That Met QC Criteria

July 2, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NVC001-PT-CL-001
  • 2025-524828-21-00 (Ctis)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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