Clinical trial NCT06164730: A Study of VERVE-102 in Patients with Familial Hypercholesterolemia or Premature Coronary Artery Disease
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Heterozygous Familial Hypercholesterolemia
Premature Coronary Heart Disease
Keywords: VERVE-102
Familial Hypercholesterolemia
Coronary Artery Disease
Dose Escalation
Gene Editing
Base Editing
Lead sponsor: Verve Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2023-12-11
Enrollment: 36
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Intervention model description: Single ascending dose escalation/adaptive design.
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: Inclusion Criteria:

* Diagnosis of HeFH or premature CAD
* Females of non-childbearing potential or males

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

Primary outcomes:
measure: Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
timeFrame: up to Day 365
Secondary outcomes:
measure: Evaluation of maximum observed concentration (Cmax)
timeFrame: up to Day 365
measure: Evaluation of time to maximum observed concentration (tmax)
timeFrame: up to Day 365
measure: Evaluation of terminal elimination half-life (t1/2)
timeFrame: up to Day 365
measure: Percent and absolute change from baseline in plasma PCSK9 concentration
timeFrame: up to Day 365
measure: Percent and absolute change from baseline in LDL-C
timeFrame: up to Day 365
Other outcomes:
Locations:
city: Adelaide
country: Australia
facility: Clinical Study Center
city: Melbourne
country: Australia
facility: Clinical Study Center
city: Chicoutimi
country: Canada
facility: Clinical Study Center
city: Montreal
country: Canada
facility: Clinical Study Center
city: Toronto
country: Canada
facility: Clinical Study Center
city: Christchurch
country: New Zealand
facility: Clinical Study Center
city: London
country: United Kingdom
facility: Clinical Study Center
city: Manchester
country: United Kingdom
facility: Clinical Study Center
Study officials:
References: