About this trial
VT-10201 is an Open-label, Phase 1b, Single-ascending Dose Study That Will Evaluate the Safety of VERVE-102 Administered to Patients With Heterozygous Familial Hypercholesterolemia (HeFH) or Premature Coronary Artery Disease (CAD) Who Require Additional Lowering of LDL-C. VERVE-102 Uses Base-editing Technology Designed to Disrupt the Expression of the PCSK9 Gene in the Liver and Lower Circulating PCSK9 and LDL-C. This Study is Designed to Determine the Safety and Pharmacodynamic Profile of VERVE-102 in This Patient Population.
Eligibility criteria
Qualifiers
Diagnosis of HeFH or premature CAD
Disqualifiers
Homozygous familial hypercholesterolemia
Active or history of chronic liver disease
Current treatment with PCSK9 inhibitor or prior treatment within specified timeframe
Clinically significant or abnormal laboratory values as defined by the protocol
Trial design
Treatments tested in this trial
- VERVE-102
Treatment groups
10
Treatment groupsSee each treatment group below.