An Open-label Study With NVC-001 in Patients With LMNA-related Dilated Cardiomyopathy (SUNBEAM-LMNA)

May 18, 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 will be conducted to evaluate the safety and tolerability, and assess exploratory efficacy of NVC-001 in adults participants with LMNA-Related Dilated Cardiomyopathy.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Detailed 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

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

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:

  • 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 of care heart failure medication prior to the Screening Visit
  • Any unstable cardiac symptoms requiring cardiac surgery, myocardial infarction, or percutaneous coronary intervention within 6 months prior to Screening
  • NYHA class IV or anticipated need for LVAD or transplant in the next 6 months
  • Pregnant or lactating or planning to become pregnant
  • Receiving an investigational intervention or participating in another clinical study within 30 days or within 5 half-lives of the drug prior to Screening
  • Prior participation in any gene therapy or approved gene therapy clinical study.

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 as measured by frequency, severity, and relatedness of treatment emergent adverse events (TEAEs)
Time Frame: At week 26 post dosing
At week 26 post dosing

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety as measured by frequency, severity, and relatedness of TEAEs
Time Frame: Up to 104 weeks post-dosing
Up to 104 weeks post-dosing
Changes in Left Ventricular Ejection Fraction (LEVF)
Time Frame: Up to 104 weeks post-dosing
LEVF will be assessed based on electrocardiogram, echocardiogram, implantable cardioverter defibrillator and ambulatory rhythm monitoring
Up to 104 weeks post-dosing
Changes in participant-reported Quality of Life using the Kansas City Cardiomyopathy Questionnaire (KCCQ)
Time Frame: Up to 104 weeks post-dosing
Up to 104 weeks post-dosing
Changes in participant-reported Quality of Life using the Minnesota Living with Heart Failure Questionnaire (MLHFQ)
Time Frame: Up to 104 weeks post-dosing
Up to 104 weeks post-dosing
Changes in participant-reported Quality of Life using the Florida Shock Anxiety Scale (FSAS)
Time Frame: Up to 104 weeks post-dosing
Up to 104 weeks post-dosing
Changes in participant-reported Quality of Life using Medical Outcomes Study Short Form 36 (SF-36)
Time Frame: Up to 104 weeks post-dosing
Up to 104 weeks post-dosing
Changes in NYHA functional class
Time Frame: Up to 104 weeks post-dosing
Up to 104 weeks post-dosing

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 (Estimated)

May 8, 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)

May 26, 2026

Last Update Submitted That Met QC Criteria

May 18, 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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