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A Study of VERVE-101 in Patients With Familial Hypercholesterolemia and Cardiovascular Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05398029
Recruitment Status : Active, not recruiting
First Posted : May 31, 2022
Last Update Posted : April 8, 2024
Sponsor:
Information provided by (Responsible Party):
Verve Therapeutics, Inc.

Study Description
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Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Heterozygous Familial Hypercholesterolemia Atherosclerotic Cardiovascular Disease Hypercholesterolemia Drug: VERVE-101 Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 44 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Intervention Model Description: Single ascending dose escalation/adaptive design followed by single dose expansion.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open-label, Phase 1b, Single-ascending Dose and Optional re Dosing Study to Evaluate the Safety of VERVE-101 Administered to Patients With Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease, and Uncontrolled Hypercholesterolemia
Actual Study Start Date : July 5, 2022
Estimated Primary Completion Date : December 2024
Estimated Study Completion Date : December 2024

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Part A: Single Ascending Dose Escalation/Adaptive Design
Participants will receive a single dose of VERVE-101 in multiple dose-escalation cohorts.
 Drug: VERVE-101
Intravenous (IV) infusion.
Experimental: Part B: Single Dose Expansion
Participants will receive a single dose of VERVE-101 selected based on the doses studied in Part A.
 Drug: VERVE-101
Intravenous (IV) infusion.


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events of special interest (AESIs). [ Time Frame: up to Day 365 ]

Secondary Outcome Measures :
  1. Evaluation of maximum observed concentration (Cmax) [ Time Frame: up to Day 365 ]
  2. Evaluation of time to maximum observed concentration (tmax) [ Time Frame: up to Day 365 ]
  3. Evaluation of terminal elimination half-life (t1/2) [ Time Frame: up to Day 365 ]

Other Outcome Measures:
  1. Percent and absolute change from baseline in plasma PCSK9 concentration. [ Time Frame: up to Day 365 ]
  2. Percent and absolute change from baseline in LDL-C. [ Time Frame: up to Day 365 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male and/or female participants 18 up to 75 years at time of signing of informed consent
  • Female participants not of child-bearing potential
  • Diagnosis of HeFH
  • Established ASCVD

Exclusion Criteria:

  • Active or history of chronic liver disease
  • Current treatment with PCSK9 monoclonal antibody therapy
  • Current or past treatment with inclisiran
  • Clinically significant or abnormal laboratory values as defined by the protocol
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05398029


Locations
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New Zealand
Clinical Study Center
Auckland, New Zealand
Clinical Study Center
Christchurch, New Zealand
United Kingdom
Clinical Study Center
London, United Kingdom
Sponsors and Collaborators
Verve Therapeutics, Inc.
More Information
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Responsible Party: Verve Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT05398029    
Other Study ID Numbers: VT-1001
First Posted: May 31, 2022    Key Record Dates
Last Update Posted: April 8, 2024
Last Verified: April 2024

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Keywords provided by Verve Therapeutics, Inc.:
VERVE-101
Familial Hypercholesterolemia
Cardiovascular Disease
 Dose Escalation
Gene Editing
Base Editing
Additional relevant MeSH terms:
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Cardiovascular Diseases
Atherosclerosis
Hyperlipoproteinemia Type II
Hypercholesterolemia
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
 Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias
Arteriosclerosis
Arterial Occlusive Diseases
Vascular Diseases