A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-70
SponsorVerve Therapeutics, Inc.
About this 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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Diagnosis of HeFH or premature CAD
Disqualifiers
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
Trial design
Design model
Sequential
Treatments tested in this trial
VERVE-102
DrugIntravenous (IV) infusion
Treatment groups
85 Participants
are divided into 10 treatment groups10
Treatment groupsSee each treatment group below.
Group A: Cohort 1: Single Ascending Dose EscalationExperimental treatment 1 intervention
Group B: Cohort 2: Single Ascending Dose EscalationExperimental treatment 1 intervention
Group C: Cohort 3: Single Ascending Dose EscalationExperimental treatment 1 intervention
Group D: Cohort 4: Single Ascending Dose EscalationExperimental treatment 1 intervention
Group E: Cohort 5: Single Ascending Dose EscalationExperimental treatment 1 intervention
Group F: Cohort 6: Single Ascending Dose EscalationExperimental treatment 1 intervention
Group G: Cohort 7: Single Ascending Dose EscalationExperimental treatment 1 intervention
Group H: Cohort 8: Single Ascending Dose EscalationExperimental treatment 1 intervention
Group I: Cohort 9: Single Fixed DoseExperimental treatment 1 intervention
Group J: Cohort 10: Single Fixed DoseExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Time frame
up to Day 365Secondary outcomes
1
Evaluation of maximum observed concentration (Cmax)
Time frame
up to Day 3652
Evaluation of time to maximum observed concentration (tmax)
Time frame
up to Day 3653
Evaluation of terminal elimination half-life (t1/2)
Time frame
up to Day 3654
Percent and absolute change from baseline in plasma PCSK9 concentration
Time frame
up to Day 365Sponsors and contacts
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Source ClinicalTrials.gov