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Recruiting NCT07606274

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

Phase I / Phase II Interventional LMNA-Related Dilated Cardiomyopathy

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: NVC-001 - Low Dose, NVC-001 - Intermediate Dose, NVC-001 - High Dose.
Who it may be relevant to
Registry conditions: LMNA-Related Dilated Cardiomyopathy. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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)

Overview

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

  • Genetic NVC-001 - Low Dose
    Solution for intravenous infusion
  • Genetic NVC-001 - Intermediate Dose
    Solution for intravenous infusion
  • Genetic NVC-001 - High Dose
    Solution for intravenous infusion

Primary outcome measures

  • Safety: Frequency, severity, and relatedness of treatment-emergent adverse events (TEAEs) [Time frame: At week 26]
Secondary outcome measures (5)
  • Change in echocardiographic parameters [Time frame: Up to 104 weeks]
  • Change from baseline in arrhythmias [Time frame: Up to 104 weeks]
  • Change in exercise capacity [Time frame: Up to 104 weeks]
  • Change from baseline in quality of life (QoL) [Time frame: Up to 104 weeks]
  • Incidence of clinical events [Time frame: Up to week 104]

Eligibility criteria

Inclusion criteria

  • 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

Exclusion criteria

  • Any chronic medical condition that, in the Investigator's opinion, would compromise participant safety or compliance
  • 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
  • Current or anticipated need for chronic IV inotropic therapy

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Sequential
Masking
Open label
Primary purpose
Other

Study locations

United States · 8 centers
  • University of Alabama in Birmingham — Birmingham
  • Cedars-Sinai Medical Center — Los Angeles
  • Brigham and Women's Hospital — Boston
  • University of Michigan — Ann Arbor
  • Mayo Clinic — Rochester
  • The Christ Hospital — Cincinnati
  • The Ohio State University — Columbus
  • Houston Methodist Hospital — Houston

Identifiers

NCT: NCT07606274 · NVC001-PT-CL-001 · 2025-524828-21-00

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗