Heterozygous Familial HypercholesterolemiaPhase 1NCT05398029
A Study of VERVE-101 in Patients With Familial Hypercholesterolemia and Cardiovascular Disease
A Phase 1 study testing VERVE-101 in people with Heterozygous Familial Hypercholesterolemia and Atherosclerotic Cardiovascular Disease, run by Verve Therapeutics, Inc. at 3 sites in 2 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease, Hypercholesterolemia
- What is being tested?
- VERVE-101
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 5 Jul 2022to 14 Feb 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- Active or history of chronic liver disease
- Current treatment with PCSK9 monoclonal antibody therapy
- Current or past treatment with inclisiran
- Clinically significant or abnormal laboratory values as defined by the protocol
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
New Zealand2 sites
Clinical Study Center
Auckland
Clinical Study Center
Christchurch
About this study
Official title: 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
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.
- Conditions
- Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease, Hypercholesterolemia
- Being tested
- VERVE-101
- Keywords
- VERVE-101, Familial Hypercholesterolemia, Cardiovascular Disease, Dose Escalation, Gene Editing, Base Editing
- Medical subject headings
- Atherosclerosis, Hypercholesterolemia, Hyperlipoproteinemia Type II, Cardiovascular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VERVE-101Drug
Intravenous (IV) infusion.
| Study group | Role | What they receive |
|---|---|---|
| Part A: Single Ascending Dose Escalation/Adaptive Design | Experimental | Participants will receive a single dose of VERVE-101 in multiple dose-escalation cohorts. Receives: Drug: VERVE-101 |
| Part B: Single Dose Expansion | Experimental | Participants will receive a single dose of VERVE-101 selected based on the doses studied in Part A. Receives: Drug: VERVE-101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events of special interest (AESIs).
Measured: up to Day 365
Secondary outcomes (3)
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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 May 2022
- Study start5 Jul 2022
- Primary completion14 Feb 2025
- Study completion14 Feb 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Heterozygous Familial 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 followed by single dose expansion.
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Verve Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05398029
- Sponsor's protocol ID
- VT-1001
- Status last verified
- May 2025
- First submitted
- 19 May 2022
- Last updated
- 8 May 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05398029
- Acronym / sponsor ID
- VT-1001
- Conditions
- Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease, Hypercholesterolemia
- Keywords
- VERVE-101, Familial Hypercholesterolemia, Cardiovascular Disease, Dose Escalation, Gene Editing, Base Editing
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Verve Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 5 Jul 2022 (actual)
- Primary completion
- 14 Feb 2025 (actual)
- Study completion
- 14 Feb 2025 (actual)
- First posted
- 31 May 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Single ascending dose escalation/adaptive design followed by single dose expansion.
| Arm | Type | Receives |
|---|---|---|
| Part A: Single Ascending Dose Escalation/Adaptive Design | Experimental | Drug: VERVE-101Participants will receive a single dose of VERVE-101 in multiple dose-escalation cohorts. |
| Part B: Single Dose Expansion | Experimental | Drug: VERVE-101Participants will receive a single dose of VERVE-101 selected based on the doses studied in Part A. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (4 listed)
- Active or history of chronic liver disease
- Current treatment with PCSK9 monoclonal antibody therapy
- Current or past treatment with inclisiran
- Clinically significant or abnormal laboratory values as defined by the protocol
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VERVE-101Intravenous (IV) infusion. | Drug | Part A: Single Ascending Dose Escalation/Adaptive Design, Part B: Single Dose Expansion |
Primary outcome
Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events of special interest (AESIs).
Time frame: up to Day 365
3 secondary outcomes
Evaluation of maximum observed concentration (Cmax)
Time frame: up to Day 365
Evaluation of time to maximum observed concentration (tmax)
Time frame: up to Day 365
Evaluation of terminal elimination half-life (t1/2)
Time frame: up to Day 365
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 May 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing VERVE-101 in people with Heterozygous Familial Hypercholesterolemia and Atherosclerotic Cardiovascular Disease, run by Verve Therapeutics, Inc. at 3 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in New Zealand, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events of special interest (AESIs). (time frame: up to Day 365). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 May 2025.