NCT05398029CompletedPhase 1
A Study of VERVE-101 in Patients With Familial Hypercholesterolemia and Cardiovascular Disease
Open-label, Phase 1b, Single-ascending Dose and Optional re Dosing Study to Evaluate the Safety of VERVE-101 Administered to Patients With Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease, and Uncontrolled Hypercholesterolemia
Source: ClinicalTrials.gov · Last updated 8 May 2025
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 13 (actual)
- First submitted
- 19 May 2022
- Start date
- 5 Jul 2022
- Primary completion date
- 14 Feb 2025
- Lead sponsor
- Verve Therapeutics, Inc.
- Diseases
- Familial hypercholesterolaemia
- NCT number
- NCT05398029
What you commit to
- Study type: Interventional · Phase 1
- Participants enrolled: 13
- From 5 Jul 2022 to the primary completion date, 14 Feb 2025
- 3 sites in 2 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Current treatment with PCSK9 monoclonal antibody therapy
- Current or past treatment with inclisiran
Other illness
- Active or history of chronic liver disease
Lab values
- Clinically significant or abnormal laboratory values as defined by the protocol
Who the study is looking for
- 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
Age in the registry: 18 years to 75 years · Sex: All · Does not accept healthy volunteers
Sites (3)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Clinical Study Center Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
| Clinical Study Center Coordinator of this site? Confirm its status | Auckland | New Zealand | Unknown status |
| Clinical Study Center Coordinator of this site? Confirm its status | Christchurch | New Zealand | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.