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

NCT06112327 | Enrolling By Invitation

• 1 other identifier: LTF-001
• Study Type: observational
• Target: 116
• Locations: 2 countries
• Sites: 3

Brief Summary

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.

Conditions

Atherosclerotic Cardiovascular Disease; Heterozygous Familial Hypercholesterolemia; Hypercholesterolemia

Keywords

LTF-001; Cardiovascular Disease; Gene Editing; Familial Hypercholesterolemia; Base Editing

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• Percent and absolute change from baseline of lipid concentrations over time after administration of a gene-editing therapy.

  up to 15 years

• Percent and absolute change from baseline of target biomarkers over time after administration of a gene-editing therapy.

  up to 15 years

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

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

Sponsors & Collaborators

• Verve Therapeutics, Inc.: lead

Study Sites (3)

Clinical Study Center

Auckland, New Zealand

Location

Clinical Study Center

Christchurch, New Zealand

Location

Clinical Study Center

London, United Kingdom

Location

MeSH Terms

Conditions

Atherosclerosis; Hypercholesterolemia; Cardiovascular Diseases; Hyperlipoproteinemia Type II

Condition Hierarchy (Ancestors)

Arteriosclerosis; Arterial Occlusive Diseases; Vascular Diseases; Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hyperlipoproteinemias

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 12, 2023
• First Posted: November 1, 2023
• Study Start: June 9, 2024
• Primary Completion (Estimated): October 1, 2039
• Study Completion (Estimated): October 1, 2039
• Last Updated: April 8, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1); NZ (2)