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

Nuevocor Pte. Ltd.

Status and phase: Not yet enrolling; Phase 2; Phase 1

Conditions: LMNA-Related Dilated Cardiomyopathy

Treatments: Genetic: NVC-001 - High Dose; Genetic: NVC-001 - Intermediate Dose; Genetic: NVC-001 - Low Dose

Study type: Interventional

Funder types: Industry

Identifiers

NCT07606274

NVC001-PT-CL-001

2025-524828-21-00 (EU Trial (CTIS) Number)

Details and patient eligibility

About

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.

Full 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

Enrollment: 21 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Other

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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21 participants in 3 patient groups

SAD low dose

Experimental group

Description:

Single Ascending Dose (SAD): participants will receive low dose of NVC-001

Treatment:

Genetic: NVC-001 - Low Dose

SAD medium dose

Experimental group

Description:

Single Ascending Dose (SAD): participants will receive medium dose of NVC-001

Treatment:

Genetic: NVC-001 - Intermediate Dose

SAD high dose

Experimental group

Description:

Single Ascending Dose (SAD): participants will receive high dose of NVC-001

Treatment:

Genetic: NVC-001 - High Dose