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Recruiting NCT06164730

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

Phase I Interventional Heterozygous Familial Hypercholesterolemia Premature Coronary Heart Disease

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: VERVE-102.
Who it may be relevant to
Registry conditions: Heterozygous Familial Hypercholesterolemia, Premature Coronary Heart Disease. Basic parameters: 18 years — 70 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Australia, Canada, Israel, New Zealand +1
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official 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

Overview

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.

Interventions

  • Drug VERVE-102
    Intravenous (IV) infusion

Primary outcome measures

  • Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) [Time frame: up to Day 365]
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]

Eligibility criteria

Inclusion criteria

  • Diagnosis of HeFH or premature CAD

Exclusion criteria

  • 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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 10 centers
  • Clinical Study Center — Dothan
  • Clinical Study Center — Pomona
  • Clinical Study Center — Boca Raton
  • Clinical Study Center — Jacksonville
  • Clinical Study Center — Winter Park
  • Clinical Study Center — Indianapolis
  • Clinical Study Center — High Point
  • Clinical Study Center — Philadelphia
  • … and 2 more centers
Canada · 6 centers
  • Clinical Study Center — Chicoutimi
  • Clinical Study Center — Hamilton
  • Clinical Study Center — Montreal
  • Clinical Study Center — Québec
  • Clinical Study Center — Toronto
  • Clinical Study Center — Vancouver
United Kingdom · 6 centers
  • Clinical Study Center — Birmingham
  • Clinical Study Center — Cambridge
  • Clinical Study Center — Edinburgh
  • Clinical Study Center — London
  • Clinical Study Center — Manchester
  • Clinical Study Center — Nottingham
Australia · 3 centers
  • Clinical Study Center — Adelaide
  • Clinical Study Center — Melbourne
  • Clinical Study Center — Sydney
Israel · 1 center
  • Clinical Study Center — Rehovot
New Zealand · 1 center
  • Clinical Study Center — Christchurch

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 Hypercholestero PMID 42187087

Identifiers

NCT: NCT06164730 · VT-10201

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗