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RecruitingNCT06164730Updated Sep 2, 2026

A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease

A Phase 1 interventional study of VERVE-102 in Heterozygous Familial Hypercholesterolemia and Premature Coronary Heart Disease, sponsored by Verve Therapeutics, Inc.. Recruiting at 27 sites in 6 countries. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2026-09-02.

Sponsored by Verve Therapeutics, Inc. (part of Eli Lilly and Company) · Phase 1, Interventional, and Treatment

From the registry’s dates

  • Started Apr 2024; still recruiting 2 years 6 months later.
Phase
Phase 1
Study type
Interventional
Enrollment
85
Allocation
Non-randomized
Ages
18 Years to 70 Years
Sex
All
01

Study summary

VT-10201 is an Open-label, Phase 1b, Single-ascending Dose Study That Will Evaluate the Safety of VERVE-102 Administered to Patients With Heterozygous Familial Hypercholesterolemia (HeFH) or Premature Coronary Artery Disease (CAD) Who Require Additional Lowering of LDL-C. VERVE-102 Uses Base-editing Technology Designed to Disrupt the Expression of the PCSK9 Gene in the Liver and Lower Circulating PCSK9 and LDL-C. This Study is Designed to Determine the Safety and Pharmacodynamic Profile of VERVE-102 in This Patient Population.

02

Conditions studied

  • Heterozygous Familial Hypercholesterolemia
  • Premature Coronary Heart Disease

Keywords

  • VERVE-102
  • Familial Hypercholesterolemia
  • Coronary Artery Disease
  • Dose Escalation
  • Gene Editing
  • Base Editing
  • heart-2
03

In context

Hyperlipoproteinemia Type II

249 studies on the registry are indexed under Hyperlipoproteinemia Type II; 56 are open to participants now.

This study's planned enrollment of 85 is below the median of 134 across 178 interventional studies indexed under Hyperlipoproteinemia Type II.

Browse Hyperlipoproteinemia Type II studies →

Lead sponsor

Verve Therapeutics, Inc. is the lead sponsor of 5 studies on the registry; 4 are open to participants now.

First submitted before Verve Therapeutics, Inc. became part of Eli Lilly and Company.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 70 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • Diagnosis of HeFH or premature CAD

Exclusion criteria

Exclusion Criteria:

  • Homozygous familial hypercholesterolemia
  • Active or history of chronic liver disease
  • Current treatment with PCSK9 inhibitor or prior treatment within specified timeframe
  • or abnormal laboratory values as defined by the protocol
Terms in these criteria, in plain language(1)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
85 participants (estimated)

Study arms

  • Experimental
    Cohort 1: Single Ascending Dose Escalation

    Participants will receive a single dose of VERVE-102.

    Drug: VERVE-102

  • Experimental
    Cohort 2: Single Ascending Dose Escalation

    Participants will receive a single dose of VERVE-102.

    Drug: VERVE-102

  • Experimental
    Cohort 3: Single Ascending Dose Escalation

    Participants will receive a single dose of VERVE-102.

    Drug: VERVE-102

  • Experimental
    Cohort 4: Single Ascending Dose Escalation

    Participants will receive a single dose of VERVE-102.

    Drug: VERVE-102

  • Experimental
    Cohort 5: Single Ascending Dose Escalation

    Participants will receive a single dose of VERVE-102.

    Drug: VERVE-102

  • Experimental
    Cohort 6: Single Ascending Dose Escalation

    Participants will receive a single dose of VERVE-102.

    Drug: VERVE-102

  • Experimental
    Cohort 7: Single Ascending Dose Escalation

    Participants will receive a single dose of VERVE-102.

    Drug: VERVE-102

  • Experimental
    Cohort 8: Single Ascending Dose Escalation

    Participants will receive a single dose of VERVE-102.

    Drug: VERVE-102

  • Experimental
    Cohort 9: Single Fixed Dose

    Participants will receive a single dose of VERVE-102.

    Drug: VERVE-102

  • Experimental
    Cohort 10: Single Fixed Dose

    Participants will receive a single dose of VERVE-102.

    Drug: VERVE-102

Interventions

  • DrugVERVE-102

    Intravenous (IV) infusion

06

What researchers measure

Primary outcomes

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

    Time frame: up to Day 365

Secondary outcomes

  1. Evaluation of maximum observed concentration (Cmax)

    Time frame: up to Day 365

  2. Evaluation of time to maximum observed concentration (tmax)

    Time frame: up to Day 365

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

    Time frame: up to Day 365

  4. Percent and absolute change from baseline in plasma PCSK9 concentration

    Time frame: up to Day 365

  5. Percent and absolute change from baseline in LDL-C

    Time frame: up to Day 365

07

Study locations

27 of 27 sites recruiting
  • Clinical Study Center
    Dothan, Alabama 36305, United States
    Recruiting
  • Clinical Study Center
    Pomona, California 91768, United States
    Recruiting
  • Clinical Study Center
    Boca Raton, Florida 33434, United States
    Recruiting
  • Clinical Study Center
    Jacksonville, Florida 32216, United States
    Recruiting
  • Clinical Study Center
    Winter Park, Florida 32789, United States
    Recruiting
  • Clinical Study Center
    Indianapolis, Indiana 46237, United States
    Recruiting
  • Clinical Study Center
    High Point, North Carolina 27260, United States
    Recruiting
  • Clinical Study Center
    Philadelphia, Pennsylvania 19104, United States
    Recruiting
  • Clinical Study Center
    DeSoto, Texas 75115, United States
    Recruiting
  • Clinical Study Center
    Renton, Washington 98057, United States
    Recruiting
  • Clinical Study Center
    Adelaide, Australia
    Recruiting
  • Clinical Study Center
    Melbourne, Australia
    Recruiting
  • Clinical Study Center
    Sydney, Australia
    Recruiting
  • Clinical Study Center
    Chicoutimi, Canada
    Recruiting
  • Clinical Study Center
    Hamilton, Canada
    Recruiting
  • Clinical Study Center
    Montreal, Canada
    Recruiting
  • Clinical Study Center
    Québec, Canada
    Recruiting
  • Clinical Study Center
    Toronto, Canada
    Recruiting
  • Clinical Study Center
    Vancouver, Canada
    Recruiting
  • Clinical Study Center
    Rehovot, Israel
    Recruiting
  • Clinical Study Center
    Christchurch, New Zealand
    Recruiting
  • Clinical Study Center
    Birmingham, United Kingdom
    Recruiting
  • Clinical Study Center
    Cambridge, United Kingdom
    Recruiting
  • Clinical Study Center
    Edinburgh, United Kingdom
    Recruiting
  • Clinical Study Center
    London, United Kingdom
    Recruiting
  • Clinical Study Center
    Manchester, United Kingdom
    Recruiting
  • Clinical Study Center
    Nottingham, United Kingdom
    Recruiting
08

References and documents

Publications

  • Vafai SB, Taubel J, Ashdown T, Patel RS, Diamondali S, Cegla J, Soran H, Bashir B, Abitbol A, Gaudet D, Lauziere A, Brunham LR, Newby DE, Nicholls SJ, Scott RS, Kerr J, Tardif JC, Lunken C, Humphries SE, Karsten V, Tyler PD, Zhang X, Huniti N, Flight PA, Jensen CL, Falzone R, Biedenkapp JC, Lister T, Stolz LE, Khera AV, Kathiresan S. In Vivo Base Editing of PCSK9 with VERVE-102 for Hypercholesterolemia. N Engl J Med. 2026 May 25. doi: 10.1056/NEJMoa2601283. Online ahead of print. PubMed 42187087 ↗
09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Sep 2, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT06164730
Responsible party
Sponsor
First posted
Dec 11, 2023
Start date
Apr 30, 2024
Primary completion
Oct 2026 (estimated)
Completion
Oct 2027 (estimated)
Last update
Sep 2, 2026

Study contacts

Trial questions or participation questions: 1-877-CTLILLY (1-877-285-4559) or
Contact
LillyTrials@Lilly.com
317-615-4559
Physicians interested in becoming principal investigators please contact
Contact
clinical_inquiry_hub@lilly.com
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

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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