A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease
For Condition: Heterozygous Familial HypercholesterolemiaFor Condition: Premature Coronary Heart Disease
ID: NCT06164730
Horizon Patient Guidance Summary
Our intelligence engine has synthesized this trial record to answer your most important questions in clear, simple terms:
How it’s taken
Injections / IV Shots
Expected Availability Horizon
Long-term Hope (5+ yrs away)
StageEarly Clinical Stage: Focuses strictly on testing basic safety, toxicity, and dosage in a small group (10–50 people). Represents early-stage scientific hope.
EligibleThis study is looking exclusively for patients diagnosed with the condition.
About This Clinical Trial
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.
Study Timeline & Quick Facts
Trial Status
Open — Looking for Participants
Total Target Enrollment
85 patients (Estimated)
Study Start Date
2024-04-30
Est. Primary Completion
2026-10
Tested Drugs & Treatment Groups
DRUGVERVE-102
Intravenous (IV) infusion
Treatment Arms Description
EXPERIMENTALCohort 1: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-102.
EXPERIMENTALCohort 2: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-102.
EXPERIMENTALCohort 3: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-102.
EXPERIMENTALCohort 4: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-102.
EXPERIMENTALCohort 5: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-102.
EXPERIMENTALCohort 6: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-102.
EXPERIMENTALCohort 7: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-102.
EXPERIMENTALCohort 8: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-102.
EXPERIMENTALCohort 9: Single Fixed Dose
Participants will receive a single dose of VERVE-102.
EXPERIMENTALCohort 10: Single Fixed Dose
Participants will receive a single dose of VERVE-102.
Can I Join This Study?
Suitable For
Everyone welcome
Ages Welcome
Between 18 Years and 70 Years
Full Criteria Checklist
Who Can Join
Diagnosis of HeFH or premature CAD
Who Cannot Join
Homozygous familial hypercholesterolemia
Active or history of chronic liver disease
Current treatment with PCSK9 inhibitor or prior treatment within specified timeframe
Clinically significant or abnormal laboratory values as defined by the protocol
What is this Study Measuring?
Primary Outcome Measures
Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) — Time Frame: up to Day 365
View Secondary Outcome Measures (5)
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
Percent and absolute change from baseline in plasma PCSK9 concentration — Time Frame: up to Day 365
Percent and absolute change from baseline in LDL-C — Time Frame: up to Day 365
Where to Participate
This trial is recruiting at 27 study facilities worldwide. Click below to see if there is a clinic or hospital in your area:
Show All 27 Facilities & Contact Sites
Clinical Study Center
Dothan, Alabama, United States
Status: Open
Clinical Study Center
Pomona, California, United States
Status: Open
Clinical Study Center
Boca Raton, Florida, United States
Status: Open
Clinical Study Center
Jacksonville, Florida, United States
Status: Open
Clinical Study Center
Winter Park, Florida, United States
Status: Open
Clinical Study Center
Indianapolis, Indiana, United States
Status: Open
Clinical Study Center
High Point, North Carolina, United States
Status: Open
Clinical Study Center
Philadelphia, Pennsylvania, United States
Status: Open
Clinical Study Center
DeSoto, Texas, United States
Status: Open
Clinical Study Center
Renton, Washington, United States
Status: Open
Clinical Study Center
Adelaide, Australia
Status: Open
Clinical Study Center
Melbourne, Australia
Status: Open
Clinical Study Center
Sydney, Australia
Status: Open
Clinical Study Center
Chicoutimi, Canada
Status: Open
Clinical Study Center
Hamilton, Canada
Status: Open
Clinical Study Center
Montreal, Canada
Status: Open
Clinical Study Center
Québec, Canada
Status: Open
Clinical Study Center
Toronto, Canada
Status: Open
Clinical Study Center
Vancouver, Canada
Status: Open
Clinical Study Center
Rehovot, Israel
Status: Open
Clinical Study Center
Christchurch, New Zealand
Status: Open
Clinical Study Center
Birmingham, United Kingdom
Status: Open
Clinical Study Center
Cambridge, United Kingdom
Status: Open
Clinical Study Center
Edinburgh, United Kingdom
Status: Open
Clinical Study Center
London, United Kingdom
Status: Open
Clinical Study Center
Manchester, United Kingdom
Status: Open
Clinical Study Center
Nottingham, United Kingdom
Status: Open
Trial Design & Scientific Setup
Allocation
non_randomized
Intervention Model
sequential
Primary Purpose
treatment
Masking
none
Sponsoring Teams & Contacts
Lead Sponsoring Institution
Verve Therapeutics, Inc.
Primary Research Contact
Trial questions or participation questions: 1-877-CTLILLY (1-877-285-4559) or
Want to apply or inquire about joining this study? Fill out this simple questionnaire, and our assistant will instantly generate a professional email draft you can copy and send to the central contact coordinator listed above!
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Subject: Inquiry about clinical trial enrollment: NCT06164730 - A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease
Dear Study Coordinator / Research Team,
I hope this message finds you well.
My name is [Your Name], and I am writing to express my strong interest in participating in the clinical trial titled "A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease" (NCT ID: NCT06164730).
I am [Age] years old and have been diagnosed with Heterozygous Familial Hypercholesterolemia. I have reviewed the eligibility criteria for this study on Med Code Info and believe I may be a suitable candidate.
Could you please let me know if this study is actively enrolling patients at any site near my location, and what the next steps are to undergo screening?
Thank you for your time and research efforts.
Warm regards,
[Your Name]
Scientific Literature & 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 ↗
View Official Medical/Scientific Title
Open-label, Phase 1b, Single Ascending Dose Study to Evaluate the Safety of VERVE-102 Administered to Patients With Heterozygous Familial Hypercholesterolemia or Premature Coronary Artery Disease Who Require Additional Lowering of Low-density Lipoprotein Cholesterol
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A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease — NCT06164730 | Med-Code