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VT-1001 is an open-label, phase 1b, single-ascending dose study that will evaluate the safety of VERVE-101 administered to patients with heterozygous familial hypercholesterolemia (HeFH), atherosclerotic cardiovascular disease (ASCVD), and uncontrolled hypercholesterolemia. VERVE-101 uses base-editing technology designed to disrupt the expression of the PCSK9 gene in the liver and lower circulating PCSK9 and LDL-C in patients with established ASCVD due to HeFH. This study is designed to determine the safety and pharmacodynamic profile of VERVE-101 in this patient population.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Part A: Single Ascending Dose Escalation/Adaptive Design | Experimental | Participants will receive a single dose of VERVE-101 in multiple dose-escalation cohorts. |
|
| Part B: Single Dose Expansion | Experimental | Participants will receive a single dose of VERVE-101 selected based on the doses studied in Part A. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VERVE-101 | Drug | Intravenous (IV) infusion. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events of special interest (AESIs). | up to Day 365 |
| Measure | Description | Time Frame |
|---|---|---|
| Evaluation of maximum observed concentration (Cmax) | up to Day 365 | |
| Evaluation of time to maximum observed concentration (tmax) | up to Day 365 | |
| Evaluation of terminal elimination half-life (t1/2) |
| Measure | Description | Time Frame |
|---|---|---|
| Percent and absolute change from baseline in plasma PCSK9 concentration. | up to Day 365 | |
| Percent and absolute change from baseline in LDL-C. | up to Day 365 |
Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Study Center | Auckland | New Zealand | ||||
| Clinical Study Center |
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Single ascending dose escalation/adaptive design followed by single dose expansion.
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| up to Day 365 |
| Christchurch |
| New Zealand |
| Clinical Study Center | London | United Kingdom |
| ID | Term |
|---|---|
| D050197 | Atherosclerosis |
| D006937 | Hypercholesterolemia |
| D006938 | Hyperlipoproteinemia Type II |
| D002318 | Cardiovascular Diseases |
| ID | Term |
|---|---|
| D001161 | Arteriosclerosis |
| D001157 | Arterial Occlusive Diseases |
| D014652 | Vascular Diseases |
| D006949 | Hyperlipidemias |
| D050171 | Dyslipidemias |
| D052439 | Lipid Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D008052 | Lipid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D006951 | Hyperlipoproteinemias |
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