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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1: Single Ascending Dose Escalation | Experimental | Participants will receive a single dose of VERVE-102. |
|
| Cohort 2: Single Ascending Dose Escalation | Experimental | Participants will receive a single dose of VERVE-102. |
|
| Cohort 3: Single Ascending Dose Escalation | Experimental | Participants will receive a single dose of VERVE-102. |
|
| Cohort 4: Single Ascending Dose Escalation | Experimental | Participants will receive a single dose of VERVE-102. |
|
| Cohort 5: Single Ascending Dose Escalation | Experimental | Participants will receive a single dose of VERVE-102. |
|
| Cohort 6: Single Ascending Dose Escalation | Experimental |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VERVE-102 | Drug | Intravenous (IV) infusion |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) | 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) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Clinical Development | Contact | 781-970-6833 | verve102clinicaltrials@lists.lilly.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Study Center | Recruiting | Dothan | Alabama | 36305 | United States | |
| Clinical Study Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 42187087 | Derived | 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. |
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Single ascending dose escalation/adaptive design.
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Participants will receive a single dose of VERVE-102.
|
| Cohort 7: Single Ascending Dose Escalation | Experimental | Participants will receive a single dose of VERVE-102. |
|
| Cohort 8: Single Ascending Dose Escalation | Experimental | Participants will receive a single dose of VERVE-102. |
|
| Cohort 9: Single Fixed Dose | Experimental | Participants will receive a single dose of VERVE-102. |
|
| Cohort 10: Single Fixed Dose | Experimental | Participants will receive a single dose of VERVE-102. |
|
| up to Day 365 |
| 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 |
| Recruiting |
| Pomona |
| California |
| 91768 |
| United States |
| Clinical Study Center | Recruiting | Boca Raton | Florida | 33434 | United States |
| Clinical Study Center | Recruiting | Jacksonville | Florida | 32216 | United States |
| Clinical Study Center | Recruiting | Winter Park | Florida | 32789 | United States |
| Clinical Study Center | Recruiting | High Point | North Carolina | 27260 | United States |
| Clinical Study Center | Recruiting | DeSoto | Texas | 75115 | United States |
| Clinical Study Center | Recruiting | Renton | Washington | 98057 | United States |
| Clinical Study Center | Recruiting | Adelaide | Australia |
| Clinical Study Center | Recruiting | Melbourne | Australia |
| Clinical Study Center | Recruiting | Sydney | Australia |
| Clinical Study Center | Recruiting | Chicoutimi | Canada |
| Clinical Study Center | Recruiting | Hamilton | Canada |
| Clinical Study Center | Recruiting | Montreal | Canada |
| Clinical Study Center | Recruiting | Québec | Canada |
| Clinical Study Center | Recruiting | Toronto | Canada |
| Clinical Study Center | Recruiting | Vancouver | Canada |
| Clinical Study Center | Recruiting | Rehovot | Israel |
| Clinical Study Center | Recruiting | Christchurch | New Zealand |
| Clinical Study Center | Recruiting | Birmingham | United Kingdom |
| Clinical Study Center | Recruiting | Edinburgh | United Kingdom |
| Clinical Study Center | Recruiting | London | United Kingdom |
| Clinical Study Center | Recruiting | Manchester | United Kingdom |
| Clinical Study Center | Recruiting | Nottingham | United Kingdom |
| ID | Term |
|---|---|
| D006938 | Hyperlipoproteinemia Type II |
| D003324 | Coronary Artery Disease |
| ID | Term |
|---|---|
| D008052 | Lipid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D006951 | Hyperlipoproteinemias |
| D006949 | Hyperlipidemias |
| D050171 | Dyslipidemias |
| D052439 | Lipid Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D003327 | Coronary Disease |
| D017202 | Myocardial Ischemia |
| D006331 | Heart Diseases |
| D002318 | Cardiovascular Diseases |
| D001161 | Arteriosclerosis |
| D001157 | Arterial Occlusive Diseases |
| D014652 | Vascular Diseases |
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