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.
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 Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
OTHER
Masking
NONE
Enrollment
21
Solution for intravenous infusion
Solution for intravenous infusion
Solution for intravenous infusion
University of Alabama in Birmingham
Birmingham, Alabama, United States
NOT_YET_RECRUITINGCedars-Sinai Medical Center
Los Angeles, California, United States
RECRUITINGBrigham and Women's Hospital
Boston, Massachusetts, United States
Safety: Frequency, severity, and relatedness of treatment-emergent adverse events (TEAEs)
Time frame: At week 26
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
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University of Michigan
Ann Arbor, Michigan, United States
NOT_YET_RECRUITINGMayo Clinic
Rochester, Minnesota, United States
RECRUITINGThe Christ Hospital
Cincinnati, Ohio, United States
NOT_YET_RECRUITINGThe Ohio State University
Columbus, Ohio, United States
NOT_YET_RECRUITINGHouston Methodist Hospital
Houston, Texas, United States
RECRUITING