LMNA-Related Dilated CardiomyopathyPhase 1/2NCT07606274
A Study With NVC-001 in Patients With LMNA-related Dilated Cardiomyopathy (SUNBEAM-LMNA)
A Phase 1/2 study testing NVC-001 - Low Dose, NVC-001 - Intermediate Dose and NVC-001 - High Dose in people with LMNA-Related Dilated Cardiomyopathy, run by Nuevocor Pte. Ltd. at 8 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- LMNA-Related Dilated Cardiomyopathy
- What is being tested?
- NVC-001 - Low Dose, NVC-001 - Intermediate Dose and NVC-001 - High Dose
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 8 sitesUnited States
- Study dates
- 16 Jun 2026to Mar 2030
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
8 sites in 1 country. Contact the study team or a site near you to ask about taking part.
8 sites in 1 country
United States8 sites, 8 recruiting
- Not yet recruiting
University of Michigan
Ann Arbor, Michigan, 48109
Nuevocor Clinical Trial Team, info@nuevocor.com
- Not yet recruiting
University of Alabama in Birmingham
Birmingham, Alabama, 35294
Nuevocor Clinical Trial Team, info@nuevocor.com
- Not yet recruiting
Brigham and Women's Hospital
Boston, Massachusetts, 02115
Nuevocor Clinical Trial Team, info@nuevocor.com
- Not yet recruiting
The Christ Hospital
Cincinnati, Ohio, 45219
Nuevocor Clinical Trial Team, info@nuevocor.com
- Not yet recruiting
The Ohio State University
Columbus, Ohio, 43210
Nuevocor Clinical Trial Team, info@nuevocor.com
- Recruiting
Houston Methodist Hospital
Houston, Texas, 77030
Nuevocor Clinical Trial Team, 281-222-9983, info@nuevocor.com
- Recruiting
Cedars-Sinai Medical Center
Los Angeles, California, 90048
Nuevocor Clinical Trial Team, 310-248-8300, info@nuevocor.com
- Recruiting
Mayo Clinic
Rochester, Minnesota, 55905
Nuevocor Clinical Trial Team, 507-422-5433, info@nuevocor.com
About this study
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)
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.
Read the 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.
- Conditions
- LMNA-Related Dilated Cardiomyopathy
- Being tested
- NVC-001 - Low Dose, NVC-001 - Intermediate Dose, NVC-001 - High Dose
- Keywords
- LMNA-related DCM, Dilated Cardiomyopathy, DCM
- Medical subject headings
- Cardiomyopathy, Dilated
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NVC-001 - Low DoseGenetic
Solution for intravenous infusion
NVC-001 - Intermediate DoseGenetic
Solution for intravenous infusion
NVC-001 - High DoseGenetic
Solution for intravenous infusion
| Study group | Role | What they receive |
|---|---|---|
| SAD low dose | Experimental | Single Ascending Dose (SAD): participants will receive low dose of NVC-001 Receives: Genetic: NVC-001 - Low Dose |
| SAD medium dose | Experimental | Single Ascending Dose (SAD): participants will receive medium dose of NVC-001 Receives: Genetic: NVC-001 - Intermediate Dose |
| SAD high dose | Experimental | Single Ascending Dose (SAD): participants will receive high dose of NVC-001 Receives: Genetic: NVC-001 - High Dose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety: Frequency, severity, and relatedness of treatment-emergent adverse events (TEAEs)
Measured: At week 26
Secondary outcomes (5)
Change in echocardiographic parameters
Measured: Up to 104 weeks
Change from baseline in arrhythmias
Measured: Up to 104 weeks
Change in exercise capacity
Measured: Up to 104 weeks
Change from baseline in quality of life (QoL)
Measured: Up to 104 weeks
Incidence of clinical events
Measured: Up to week 104
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 May 2026
- Study start16 Jun 2026
- Primary completionMar 2030 (estimated)
- Study completionMar 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LMNA-Related Dilated CardiomyopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 21 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Nuevocor Pte. Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Monica Shah, MD, Study director, Nuevocor Pte. Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07606274
- Sponsor's protocol ID
- NVC001-PT-CL-001
- Other IDs
- 2025-524828-21-00, 2025
- Status last verified
- Aug 2026
- First submitted
- 6 May 2026
- Last updated
- 7 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT07606274
- Acronym / sponsor ID
- NVC001-PT-CL-001
- Conditions
- LMNA-Related Dilated Cardiomyopathy
- Keywords
- LMNA-related DCM, Dilated Cardiomyopathy, DCM
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 21 participants (estimated)
- Lead sponsor
- Nuevocor Pte. Ltd.Industry (pharma, biotech, devices)
- Study start
- 16 Jun 2026 (actual)
- Primary completion
- Mar 2030 (estimated)
- Study completion
- Mar 2030 (estimated)
- First posted
- 26 May 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| SAD low dose | Experimental | Genetic: NVC-001 - Low DoseSingle Ascending Dose (SAD): participants will receive low dose of NVC-001 |
| SAD medium dose | Experimental | Genetic: NVC-001 - Intermediate DoseSingle Ascending Dose (SAD): participants will receive medium dose of NVC-001 |
| SAD high dose | Experimental | Genetic: NVC-001 - High DoseSingle Ascending Dose (SAD): participants will receive high dose of NVC-001 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NVC-001 - Low DoseSolution for intravenous infusion | Genetic | SAD low dose |
| NVC-001 - Intermediate DoseSolution for intravenous infusion | Genetic | SAD medium dose |
| NVC-001 - High DoseSolution for intravenous infusion | Genetic | SAD high dose |
Primary outcome
Safety: Frequency, severity, and relatedness of treatment-emergent adverse events (TEAEs)
Time frame: At week 26
5 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Monica Shah, MD study director
Nuevocor Pte. Ltd.
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing NVC-001 - Low Dose, NVC-001 - Intermediate Dose and NVC-001 - High Dose in people with LMNA-Related Dilated Cardiomyopathy, run by Nuevocor Pte. Ltd. at 8 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 7 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety: Frequency, severity, and relatedness of treatment-emergent adverse events (TEAEs) (time frame: At week 26). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Aug 2026.