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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
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
Secondary ID [1] 0 0
VT-1001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Heterozygous Familial Hypercholesterolemia 0 0
Atherosclerotic Cardiovascular Disease 0 0
Hypercholesterolemia 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events of special interest (AESIs).
Timepoint [1] 0 0
up to Day 365
Secondary outcome [1] 0 0
Evaluation of maximum observed concentration (Cmax)
Timepoint [1] 0 0
up to Day 365
Secondary outcome [2] 0 0
Evaluation of time to maximum observed concentration (tmax)
Timepoint [2] 0 0
up to Day 365
Secondary outcome [3] 0 0
Evaluation of terminal elimination half-life (t1/2)
Timepoint [3] 0 0
up to Day 365

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
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
5/07/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2024
Actual
Sample size
Target
44
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
New Zealand
State/province [1] 0 0
Auckland
Country [2] 0 0
New Zealand
State/province [2] 0 0
Christchurch
Country [3] 0 0
United Kingdom
State/province [3] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Verve Therapeutics, Inc.
Address
Country

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05398029
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries