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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05398029
Registration number
NCT05398029
Ethics application status
Date submitted
19/05/2022
Date registered
31/05/2022
Date last updated
8/04/2024
Titles & IDs
Public title
A Study of VERVE-101 in Patients With Familial Hypercholesterolemia and Cardiovascular Disease
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Scientific 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
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Secondary ID [1]
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VT-1001
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Heterozygous Familial Hypercholesterolemia
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Atherosclerotic Cardiovascular Disease
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Hypercholesterolemia
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Condition category
Condition code
Cardiovascular
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Diseases of the vasculature and circulation including the lymphatic system
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Metabolic and Endocrine
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Other metabolic disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - VERVE-101
Experimental: Part A: Single Ascending Dose Escalation/Adaptive Design - Participants will receive a single dose of VERVE-101 in multiple dose-escalation cohorts.
Experimental: Part B: Single Dose Expansion - Participants will receive a single dose of VERVE-101 selected based on the doses studied in Part A.
Treatment: Drugs: VERVE-101
Intravenous (IV) infusion.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events of special interest (AESIs).
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Assessment method [1]
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Timepoint [1]
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up to Day 365
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Secondary outcome [1]
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Evaluation of maximum observed concentration (Cmax)
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Assessment method [1]
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Timepoint [1]
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up to Day 365
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Secondary outcome [2]
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Evaluation of time to maximum observed concentration (tmax)
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Assessment method [2]
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Timepoint [2]
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up to Day 365
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Secondary outcome [3]
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Evaluation of terminal elimination half-life (t1/2)
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Assessment method [3]
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Timepoint [3]
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up to Day 365
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Eligibility
Key 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
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Minimum age
18
Years
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Maximum age
75
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Other
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Other design features
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Phase
Phase 1
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
5/07/2022
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/12/2024
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Actual
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Sample size
Target
44
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
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Recruitment outside Australia
Country [1]
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New Zealand
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State/province [1]
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Auckland
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Country [2]
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New Zealand
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State/province [2]
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Christchurch
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Country [3]
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United Kingdom
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State/province [3]
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Verve Therapeutics, Inc.
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05398029
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05398029
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