Table of Contents
- Overview of the Vericiguat trial program
- Heart failure studies in adults
- Studies in children with heart failure
- Studies in other conditions
- Trial design and main endpoints
- Who the trials are for
Overview of the Vericiguat trial program
The trial data show that Vericiguat is being tested in more than one disease area, with the main focus on heart failure.[1][2] The studies include adults and children, and they use different trial phases, mainly Phase 2 and Phase 3, with one Phase 4 study listed.[1][2]
Several studies are completed, while others are authorised, which means they are planned or allowed to start.[1][2] The trials are designed to look at both benefit and safety, depending on the condition being studied.[1][2]
Heart failure studies in adults
Most of the studies in this set focus on heart failure with reduced ejection fraction (HFrEF) or related forms of heart failure.[1][3] In one large Phase 3 trial, researchers compared Vericiguat with placebo in 6,836 participants with chronic HFrEF and measured the time to the first event of cardiovascular death or heart failure hospitalization.[3]
Another completed Phase 3 study in 17 participants with HFrEF and a CardioMEMS HF System looked at the lowering of diastolic pulmonary artery pressure (dPAP) compared with placebo.[1] This is a pressure measure in the blood vessels that carry blood from the heart to the lungs.[1]
A Phase 2 study in 138 participants with chronic HFrEF examined whether starting Vericiguat at 5 mg was tolerated over two weeks.[4] The main safety questions were whether participants could finish the 5 mg dose and whether they had moderate to severe symptomatic hypotension, which means low blood pressure causing symptoms such as dizziness or fainting.[4]
Studies in children with heart failure
Two trials focus on pediatric participants with heart failure due to left ventricular systolic dysfunction.[2][5] One is a Phase 4 randomized, placebo-controlled, double-blind study with 320 participants, and it looks at change in NT-proBNP from baseline to Week 16 in the base period, plus safety in the extension period.[2]
The open-label extension study included 254 participants and was designed to monitor safety and tolerability of Vericiguat.[5] In an open-label study, everyone knows what treatment is being given.[5]
Studies in other conditions
Not all of the studies are in heart failure. One Phase 2 trial in 21 participants with treatment-resistant hypertension is testing Vericiguat as part of a combination approach and measuring change in systolic blood pressure by 24-hour ambulatory blood pressure monitoring.[6]
Another Phase 2 study in 55 participants with unequivocal epicardial and/or microvascular vasospastic angina is examining microvascular function and daily angina episodes.[7] This study uses placebo and compares 10-week treatment periods to see whether symptoms and blood vessel function change.[7]
A completed Phase 2 study also looked at Vericiguat in participants with post-COVID-19 syndrome, with or without chronic fatigue syndrome criteria, and measured physical function using the SF-36 physical function score over 10 weeks.[8]
Trial design and main endpoints
Several studies use a randomized, double-blind, and placebo-controlled design, which helps make the comparison fair.[1][2][3] In these designs, participants are assigned by chance, neither the participants nor the study team know who gets the active treatment, and placebo is used as the comparison treatment.[1][2]
The main endpoints vary by trial. They include cardiovascular death, heart failure hospitalization, NT-proBNP change, dPAP lowering, treatment tolerability, safety events, blood pressure change, angina frequency, microvascular conductance, and physical function scores.[1][2][3][4][6][7][8]
Some studies also track adverse events, which are unwanted medical problems that happen during a trial, and discontinuation due to adverse events.[2][5] This helps researchers understand not only whether the treatment may help, but also how well it is tolerated in the study group.[2][5]
Who the trials are for
The target groups in these trials include adults with chronic HFrEF, children with heart failure due to left ventricular systolic dysfunction, people with treatment-resistant hypertension, people with documented vasospastic angina, and people with post-COVID-19 syndrome.[1][2][3][6][7][8]
The studies are not all the same size: some are small, such as the 17-participant and 21-participant studies, while others are much larger, such as the 6,836-participant heart failure trial.[1][3][6] This mix of sizes suggests that researchers are testing different questions, from early signals to larger outcome studies.[1][3][6]
