NCT06451770

Brief 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 lipoprotein cholesterol (LDL-C) and triglycerides. This study is designed to determine the safety and pharmacodynamic profile of VERVE-201 in this patient population.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
36

participants targeted

Target at P50-P75 for phase_1

Timeline
19mo left

Started Oct 2024

Typical duration for phase_1

Geographic Reach
4 countries

10 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress49%
Oct 2024Dec 2027

First Submitted

Initial submission to the registry

June 4, 2024

Completed
7 days until next milestone

First Posted

Study publicly available on registry

June 11, 2024

Completed
5 months until next milestone

Study Start

First participant enrolled

October 30, 2024

Completed
3.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2027

Last Updated

April 28, 2026

Status Verified

November 1, 2025

Enrollment Period

3.1 years

First QC Date

June 4, 2024

Last Update Submit

April 22, 2026

Conditions

HypercholesterolemiaHypertriglyceridemiaRefractory Hyperlipidemia

Keywords

VERVE-201Gene EditingBase EditingRefractory HypercholesterolemiaANGPTL3ASCVDRefractory HypertriglyceridemiaRefractory HyperlipidemiaFamilial Hypercholesterolemia

Outcome Measures

Primary Outcomes (1)

  • Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)

    Up to Day 365

Secondary Outcomes (6)

  • Evaluation of maximum observed concentration (Cmax)

    Up to Day 365

  • Evaluation of time to maximum observed concentration (tmax)

    Up to Day 365

  • Evaluation of terminal elimination half-life (t1/2)

    Up to Day 365

  • Percent and absolute change from baseline in serum ANGPTL3 concentration

    Up to Day 365

  • Percent and absolute change from baseline in serum LDL-C

    Up to Day 365

  • +1 more secondary outcomes

Study Arms (6)

Cohort 1: Single Ascending Dose Escalation

EXPERIMENTAL

Participants will receive a single dose of VERVE-201.

Drug: VERVE-201

Cohort 2: Single Ascending Dose Escalation

EXPERIMENTAL

Participants will receive a single dose of VERVE-201.

Drug: VERVE-201

Cohort 3: Single Ascending Dose Escalation

EXPERIMENTAL

Participants will receive a single dose of VERVE-201.

Drug: VERVE-201

Cohort 4: Single Ascending Dose Escalation

EXPERIMENTAL

Participants will receive a single dose of VERVE-201.

Drug: VERVE-201

Cohort 5: Single Ascending Dose Escalation

EXPERIMENTAL

Participants will receive a single dose of VERVE-201.

Drug: VERVE-201

Cohort 6: Single Ascending Dose Escalation

EXPERIMENTAL

Participants will receive a single dose of VERVE-201.

Drug: VERVE-201

Interventions

VERVE-201DRUG

Intravenous (IV) infusion

Cohort 1: Single Ascending Dose EscalationCohort 2: Single Ascending Dose EscalationCohort 3: Single Ascending Dose EscalationCohort 4: Single Ascending Dose EscalationCohort 5: Single Ascending Dose EscalationCohort 6: Single Ascending Dose Escalation

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Refractory hypercholesterolemia
  • Refractory hypertriglyceridemia

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

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

MeSH Terms

Conditions

HypercholesterolemiaHypertriglyceridemiaHyperlipidemiasHyperlipoproteinemia Type II

Condition Hierarchy (Ancestors)

DyslipidemiasLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesHyperlipoproteinemias

Central Study Contacts

Clinical Development

CONTACT

781-970-6833verve201clinicaltrials@lists.lilly.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Model Details: Single ascending dose escalation/adaptive design
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 4, 2024

First Posted

June 11, 2024

Study Start

October 30, 2024

Primary Completion (Estimated)

December 1, 2027

Study Completion (Estimated)

December 1, 2027

Last Updated

April 28, 2026

Record last verified: 2025-11

Locations

ZA(2)AU(2)GB(3)CA(3)