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.
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
Allocation
NON_RANDOMIZED
Purpose
OTHER
Masking
NONE
Enrollment
21
Solution for intravenous infusion
Solution for intravenous infusion
Solution for intravenous infusion
Cedars-Sinai Medical Center
Los Angeles, California, United States
Safety as measured by frequency, severity, and relatedness of treatment emergent adverse events (TEAEs)
Time frame: At week 26 post dosing
Safety as measured by frequency, severity, and relatedness of TEAEs
Time frame: Up to 104 weeks post-dosing
Changes in Left Ventricular Ejection Fraction (LEVF)
LEVF will be assessed based on electrocardiogram, echocardiogram, implantable cardioverter defibrillator and ambulatory rhythm monitoring
Time frame: 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
Changes in participant-reported Quality of Life using the Minnesota Living with Heart Failure Questionnaire (MLHFQ)
Time frame: 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
Changes in participant-reported Quality of Life using Medical Outcomes Study Short Form 36 (SF-36)
Time frame: Up to 104 weeks post-dosing
Changes in NYHA functional class
Time frame: Up to 104 weeks post-dosing
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