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NCT05398029 (VERVE-101 in heterozygous familial hypercholesterolemia): BVCT ex-ante prediction vs. reported outcome

On 31 Oct 2023, BioinvestGPT's BVCT predicted technical failure and commercial failure for this trial. The 12 Nov 2023 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically maybe significant and commercially insufficient.

Clinical Trial

NCTID
NCT05398029
Acronym
Heart-1
Drug Name
VERVE-101
Drug MoA
in vivo CRISPR base-editing targeting PCSK9
Drug Modality
gene therapy
Drug Class
First-In-Class
Therapeutic Area
Nutritional and Metabolic Diseases
Indication
heterozygous familial hypercholesterolemia (HeFH)
Human Patients
13
Sponsor
Verve Therapeutics
Phase
Phase 1

Prediction of , index 1306

Prospective Prediction
Prediction Index
1306
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient additive clinical benefit)
Technical Prediction
FAILURE (statistically insignificant additive clinical benefit)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
12 days in advance
Readout Data Interpretation
VERVE101 achieved 47% PCSK9 protein reduction, 55% LDL-C reduction and grade 3 TEAEs, both of which are non-superior to the SoC evolocumab that achieved 91% PCSK9 protein reduction, 76% LDL-C reduction without grade 3 TEAEs (see add'l link below), further development discontinued on 22 May 2024
Press Release
Additional Readout Data
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically maybe significant and commercially insufficient
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

ClinicalTrials.gov record, retrieved 2026-09-24. Third-party data.

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
Phase
Phase 1
Status
Completed
Enrollment
13
Lead sponsor
Verve Therapeutics, Inc.
Conditions
Heterozygous Familial Hypercholesterolemia; Atherosclerotic Cardiovascular Disease; Hypercholesterolemia
Interventions
DRUG: VERVE-101
Design
Non randomized · Sequential · None
Primary purpose
Treatment
Start
2022-07-05
Primary completion
2025-02-14
Completion
2025-02-14
Primary outcomes
  • Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events of special interest (AESIs). (up to Day 365)

NCT05398029 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

How to read these records: evidence · methodology · all trials