A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease

Study at a glance

From the registry record, last updated by the sponsor on 2 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title

Open-label, Phase 1b, Single Ascending Dose Study to Evaluate the Safety of VERVE-102 Administered to Patients With Heterozygous Familial Hypercholesterolemia or Premature Coronary Artery Disease Who Require Additional Lowering of Low-density Lipoprotein Cholesterol

ClinicalTrials.gov ID

NCT06164730

Acronym / sponsor ID

VT-10201

Conditions

Heterozygous Familial Hypercholesterolemia, Premature Coronary Heart Disease

Keywords

VERVE-102, Familial Hypercholesterolemia, Coronary Artery Disease, Dose Escalation, Gene Editing, Base Editing, heart-2

Phase

Phase 1

Study type

Interventional

Recruitment status

Recruiting

Enrolment

85 participants (estimated)

Lead sponsor

Verve Therapeutics, Inc.Industry (pharma, biotech, devices)

Study start

30 Apr 2024 (actual)

Primary completion

Oct 2026 (estimated)

Study completion

Oct 2027 (estimated)

First posted

11 Dec 2023 (actual)

Results

Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose

Treatment

Allocation

Non-randomised

Intervention model

Sequential

Masking

None (open label)

Number of arms

10

Model description

Single ascending dose escalation/adaptive design.

Who may qualify

Ages

18 to 70 years

Sex

All

Healthy volunteers

Not accepted

Age groups

Adult, Older adult

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Primary outcome

1. Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)

   Time frame: up to Day 365

Results and safety

This study is still under way. Results are not available in the source record yet.

Research links

• Official record on ClinicalTrials.govLast updated by the sponsor 2 Sep 2026.
• Derived from this study: PubMed 42187087Vafai 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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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.

27 sites in 6 countries: see every site and its status.