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

Purpose

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.

Condition

  • LMNA-Related Dilated Cardiomyopathy

Eligibility

Eligible Ages
Over 18 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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

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 Design

Phase
Phase 1/Phase 2
Study Type
Interventional
Allocation
Non-Randomized
Intervention Model
Sequential Assignment
Primary Purpose
Other
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
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

Recruiting Locations

Cedars-Sinai Medical Center
Los Angeles, California 90048
Contact:
Nuevocor Clinical Trial Team
310-248-8300
info@nuevocor.com

Mayo Clinic
Rochester, Minnesota 55905
Contact:
Nuevocor Clinical Trial Team
507-422-5433
info@nuevocor.com

Houston Methodist Hospital
Houston, Texas 77030
Contact:
Nuevocor Clinical Trial Team
281-222-9983
info@nuevocor.com

More Details

Status
Recruiting
Sponsor
Nuevocor Pte. Ltd.

Study Contact

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.