HypercholesterolemiaPhase 1NCT06451770
Phase 1b Study of VERVE-201 in Patients With Refractory Hyperlipidemia
A Phase 1 study testing VERVE-201 in people with Hypercholesterolemia and Hypertriglyceridemia, run by Verve Therapeutics, Inc. at 18 sites in 6 countries.
Trial summary
VT-20101 is an Open-label, Phase 1b, Single-ascending dose study that will evaluate the safety of VERVE-201 administered to patients with Refractory Hyperlipidemia. VERVE-201 uses base-editing technology designed to inactivate the expression of the ANGPTL3 gene in the liver and lower circulating low-density… Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Hypercholesterolemia, Hypertriglyceridemia, Refractory Hyperlipidemia
- What is being tested?
- VERVE-201
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 132 participants
- Where
- 18 sitesin 6 countries
- Study dates
- 30 Oct 2024to Dec 2027
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Refractory hypercholesterolemia
- Refractory hypertriglyceridemia
Who cannot join
- 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
Trial locations and contacts
18 sites in 6 countries. Contact the study team or a site near you to ask about taking part.
Trial questions or participation questions: 1-877-CTLILLY (1-877-285-4559) or contact
Physicians interested in becoming principal investigators please contact contact
18 sites in 6 countries
United States5 sites, 5 recruiting
- Recruiting
Clinical Study Center
Clearwater, Florida, 33756
- Recruiting
Clinical Study Center
Garden Grove, California, 92844
- Recruiting
Clinical Study Center
Miami Lakes, Florida, 33016
- Recruiting
Clinical Study Center
New York, New York, 10029
- Recruiting
Clinical Study Center
Port Orange, Florida, 32127
United Kingdom4 sites, 4 recruiting
- Recruiting
Clinical Study Center
Liverpool
- Recruiting
Clinical Study Center
London
- Recruiting
Clinical Study Center 2
Manchester
- Recruiting
Clinical Study Center
Manchester
Canada3 sites, 3 recruiting
- Recruiting
Clinical Study Center
Chicoutimi
- Recruiting
Clinical Study Center
Montreal
- Recruiting
Clinical Study Center
Toronto
Australia2 sites, 2 recruiting
- Recruiting
Clinical Study Center
Adelaide
- Recruiting
Clinical Study Center
Melbourne
Netherlands2 sites, 2 recruiting
- Recruiting
Clinical Study Center
Amsterdam
- Recruiting
Clinical Study Center
Rotterdam
South Africa2 sites, 2 recruiting
- Recruiting
Clinical Study Center
Cape Town
- Recruiting
Clinical Study Center
Johannesburg
About this study
Official title: A Phase 1b Single Ascending Dose Study to Evaluate the Safety of VERVE-201 in Patients With Refractory Hyperlipidemia
VT-20101 is an Open-label, Phase 1b, Single-ascending dose study that will evaluate the safety of VERVE-201 administered to patients with Refractory Hyperlipidemia.
VERVE-201 uses base-editing technology designed to inactivate the expression of the ANGPTL3 gene in the liver and lower circulating low-density lipoprotein cholesterol (LDL-C) and triglycerides. This study is designed to determine the safety and pharmacodynamic profile of VERVE-201 in this patient population.
- Being tested
- VERVE-201
- Keywords
- VERVE-201, Gene Editing, Base Editing, Refractory Hypercholesterolemia, ANGPTL3, ASCVD, Refractory Hypertriglyceridemia, Refractory Hyperlipidemia, Familial Hypercholesterolemia
- Medical subject headings
- Hypercholesterolemia, Hypertriglyceridemia, Hyperlipidemias, Hyperlipoproteinemia Type II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VERVE-201Drug
Intravenous (IV) infusion
| Study group | Role | What they receive |
|---|---|---|
| Part A: Single Ascending Dose Escalation for Participants with Refractory Hypercholesterolemia | Experimental | Participants assigned to a dose-escalation cohort will receive a single dose of VERVE-201. Receives: Drug: VERVE-201 |
| Part B: Re-Dosing for Part A Participants | Experimental | Participants from Part A assigned to the re-dosing cohort will receive a second dose of VERVE-201. Receives: Drug: VERVE-201 |
| Part C: Single Ascending Dose Escalation for Participants with sHTG | Experimental | Participants assigned to a dose-expansion cohort will receive a single dose of VERVE-201. Receives: Drug: VERVE-201 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Measured: Up to Day 365
Secondary outcomes (6)
Evaluation of maximum observed concentration (Cmax)
Measured: Up to Day 365
Evaluation of time to maximum observed concentration (tmax)
Measured: Up to Day 365
Evaluation of terminal elimination half-life (t1/2)
Measured: Up to Day 365
Percent and absolute change from baseline in serum ANGPTL3 concentration
Measured: Up to Day 365
Percent and absolute change from baseline in serum LDL-C
Measured: Up to Day 365
Percent and absolute change from baseline in serum triglycerides
Measured: Up to Day 365
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2024
- Study start30 Oct 2024
- Primary completionDec 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HypercholesterolemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Single ascending dose escalation/adaptive design
- Masking
- None (open label)
- Enrolment
- 132 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Verve Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 8 AM - 8 PM Eastern time (UTC/GMT - 5 hours, EST), Study director, Eli Lilly and Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06451770
- Sponsor's protocol ID
- VT-20101
- Other IDs
- 2025-522617-39-00, 2025
- Status last verified
- Oct 2026
- First submitted
- 4 Jun 2024
- Last updated
- 23 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No