Long-term Follow-up of Participants Dosed With an Investigational Gene Editing Therapy for Cardiovascular Disease

Long-term Follow-up Study of Investigational Gene-editing Therapies in Participants With or at High Risk for Cardiovascular Disease

LTF-001 is a long-term follow-up study of participants who received an investigational gene-editing therapy developed by the sponsor to evaluate the long-term effects of the investigational therapy. Participants will be followed for a total of 15 years after the first administration of the gene-editing therapy.

Study Overview

• Status: Enrolling by invitation
• Conditions: Hypercholesterolemia; Atherosclerotic Cardiovascular Disease; Heterozygous Familial Hypercholesterolemia

• Study Type: Observational
• Enrollment (Estimated): 116

Contacts and Locations

Study Locations

• New Zealand
  • Auckland, New Zealand
    • Clinical Study Center
  • Christchurch, New Zealand
    • Clinical Study Center
• United Kingdom
  • London, United Kingdom
    • Clinical Study Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Participants who received an investigational gene-editing therapy developed by the sponsor.

Description

Inclusion Criteria

1. A participant has completed or discontinued from a Verve sponsored clinical study in which they received at least one dose of study drug.
2. A participant has provided informed consent for LTF-001.

Exclusion Criteria: N/A

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of treatment-related adverse events (AEs), serious adverse events (SAEs) and other events of interest using CTCAE 5.0 to assess the long-term safety of gene-editing therapies.	To assess the long-term safety of gene-editing therapies, the following will be assessed: Incidence of treatment-related adverse events (AEs), serious adverse events (SAEs) and any CTCAE grade 3 or higher AEs.	up to 15 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percent and absolute change from baseline of lipid concentrations over time after administration of a gene-editing therapy.	Blood samples will be collected to assess percent change from baseline in lipid concentrations (including LDL-C).	up to 15 years
Percent and absolute change from baseline of target biomarkers over time after administration of a gene-editing therapy.	Blood samples will be collected to assess percent change from baseline in target biomarkers.	up to 15 years

Collaborators and Investigators

• Sponsor: Verve Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): June 9, 2024
• Primary Completion (Estimated): October 1, 2039
• Study Completion (Estimated): October 1, 2039

Study Registration Dates

• First Submitted: October 12, 2023
• First Submitted That Met QC Criteria: October 31, 2023
• First Posted (Actual): November 1, 2023

Study Record Updates

• Last Update Posted (Actual): April 8, 2026
• Last Update Submitted That Met QC Criteria: April 2, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: Gene Editing; Cardiovascular Disease; Familial Hypercholesterolemia; Base Editing; LTF-001
• Additional Relevant MeSH Terms: Vascular Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Arteriosclerosis; Arterial Occlusive Diseases; Lipid Metabolism, Inborn Errors; Hyperlipoproteinemias; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Cardiovascular Diseases; Hypercholesterolemia; Hyperlipoproteinemia Type II; Atherosclerosis
• Other Study ID Numbers: LTF-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes