A Study of VERVE-101 in Patients With Familial Hypercholesterolemia and Cardiovascular Disease

NCTID	NCT05398029 (View at clinicaltrials.gov)
Description	VT-1001 is an open-label, phase 1b, single-ascending dose study that will evaluate the safety of VERVE-101 administered to patients with heterozygous familial hypercholesterolemia (HeFH), atherosclerotic cardiovascular disease (ASCVD), and uncontrolled hypercholesterolemia. VERVE-101 uses base-editing technology designed to disrupt the expression of the PCSK9 gene in the liver and lower circulating PCSK9 and LDL-C in patients with established ASCVD due to HeFH. This study is designed to determine the safety and pharmacodynamic profile of VERVE-101 in this patient population. (Show More)
Development Status	Inactive
Indication	Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease
Disease Ontology Term	DOID:13810
Compound Name	VERVE-101
Sponsor	Verve Therapeutics, Inc.
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	13 (ACTUAL)
Results Posted	Not Available

Eligible Age	18 Years - 75 Years
Standard Ages	Adult, Older adult
Sexes Eligible for Study	ALL
Eligibility Criteria	Inclusion Criteria: * Male and/or female participants 18 up to 75 years at time of signing of informed consent * Female participants not of child-bearing potential * Diagnosis of HeFH * Established ASCVD Exclusion Criteria: * Active or history of chronic liver disease * Current treatment with PCSK9 monoclonal antibody therapy * Current or past treatment with inclisiran * Clinically significant or abnormal laboratory values as defined by the protocol View Inclusion and Exclusion Criteria at ClinicalTrials.gov

No.of Trial Sites	3
Locations	New Zealand,United Kingdom

Has US IND	True
FDA Designations
Recent Updates	This program is deprioritized in favor of VERVE-102