Testing a new gene editing treatment for patients with high cholesterol
A Phase 1b Single Ascending Dose Study to Evaluate the Safety of VERVE-201 in Patients With Refractory Hyperlipidemia
This study is testing a new gene editing treatment to see if it can safely lower cholesterol levels in people with high cholesterol that doesn't respond to other treatments.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 36 (estimated) |
| Ages | 18 Years to 70 Years |
| Sex | All |
| Sponsor | Verve Therapeutics, Inc. Industry-sponsored |
| Locations | 10 sites (Adelaide and 9 other locations) |
| Trial ID | NCT06451770 on ClinicalTrials.gov |
What this trial studies
This Phase 1b study evaluates the safety of VERVE-201, a gene editing therapy designed to inactivate the ANGPTL3 gene in the liver, in patients suffering from refractory hypercholesterolemia. The study employs a single-ascending dose approach to assess the pharmacodynamic profile and safety of the treatment. Participants will receive VERVE-201 to determine its effectiveness in lowering low-density lipoprotein cholesterol (LDL-C) levels. The study aims to provide insights into the potential of gene editing as a therapeutic option for this condition.
Who should consider this trial
Good fit: Ideal candidates include individuals with refractory hypercholesterolemia who are either females of non-childbearing potential or males.
Not a fit: Patients with active or a history of chronic liver disease or those currently receiving treatments targeting ANGPTL3 may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly lower cholesterol levels in patients who have not responded to existing therapies.
How similar studies have performed: While gene editing approaches are emerging, this specific application of base editing for refractory hypercholesterolemia is novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Refractory hypercholesterolemia * Refractory hypertriglyceridemia Exclusion Criteria: * Active or history of chronic liver disease * Current treatment with monoclonal antibody targeting ANGPTL3 or prior treatment within specified timeframe * Clinically significant or abnormal laboratory values as defined by the protocol
Where this trial is running
Adelaide and 9 other locations
- Clinical Study Center — Adelaide, Australia (Recruiting)
- Clinical Study Center — Melbourne, Australia (Recruiting)
- Clinical Study Center — Chicoutimi, Canada (Recruiting)
- Clinical Study Center — Montreal, Canada (Recruiting)
- Clinical Study Center — Toronto, Canada (Recruiting)
- Clinical Study Center — Cape Town, South Africa (Recruiting)
- Clinical Study Center — Johannesburg, South Africa (Recruiting)
- Clinical Study Center — Liverpool, United Kingdom (Recruiting)
- Clinical Study Center — London, United Kingdom (Recruiting)
- Clinical Study Center — Manchester, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Clinical Development
- Email: verve201clinicaltrials@lists.lilly.com
- Phone: 781-970-6833
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.