Efficacy, Safety, and Pharmacokinetics of Vericiguat in Pediatric Participants With Heart Failure Due to Left Ventricular Systolic Dysfunction (MK-1242-036)

Merck Sharp & Dohme (MSD)

Status and phase: Enrolling; Phase 3; Phase 2

Conditions: Left Ventricular Systolic Dysfunction; Heart Failure

Treatments: Drug: Vericiguat tablet; Drug: Placebo tablet; Drug: Vericiguat suspension; Drug: Placebo suspension

Study type: Interventional

Funder types: Industry

Identifiers

NCT05714085

MK-1242-036 (Other Identifier)

1242-036

2021-004399-33 (EudraCT Number)

Details and patient eligibility

About

This study aims to compare the efficacy of vericiguat versus placebo on change in n-terminal pro-brain natriuretic peptide (NTproBNP) from baseline to Week 16. The primary hypothesis is Vericiguat is superior to placebo in reducing NT-proBNP at Week 16.

Enrollment: 342 estimated patients

Sex: All

Ages: 29 days to 17 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Has a history of symptomatic chronic heart failure (HF) resulting from systemic left ventricular (LV) systolic dysfunction
• Has biventricular physiology with a morphologic systemic left ventricle
• Is currently receiving stable medical therapy for HF
• Has left ventricular ejection fraction (LVEF) <45% assessed within 3 months before randomization
• Female is eligible to participate if not pregnant or breastfeeding, and at least one of the following: is not a participant of childbearing potential (POCBP); or is a POCBP who uses a highly effective contraceptive method; has a negative highly sensitive pregnancy test; abstains from breastfeeding for at least 30 days after study intervention; and their medical history; their menstrual history, and recent sexual activity has been reviewed

Exclusion criteria

• Is clinically unstable-with at least one of the following: hypotensive for age, recent use of intravenous (IV) inotrope and/or IV vasodilator, or recent IV diuretic or oral diuretic dose increase
• Has a known allergy or sensitivity to vericiguat, any of its constituents, or any other soluble guanylate cyclase (sGC) stimulator
• Has a history of single ventricle heart disease or has a morphologic systemic right ventricle
• Has undergone heart transplantation, is awaiting heart transplantation United Network for Organ Sharing (UNOS) Class 1A or equivalent, is receiving continuous IV infusion of an inotrope, or has an implanted ventricular assist device
• Has sustained or symptomatic dysrhythmia uncontrolled with drug or device therapy
• Has had recent cardiovascular (CV) surgical procedure or percutaneous intervention to palliate or correct congenital CV malformations
• Has unoperated or residual hemodynamically significant congenital cardiac malformations
• Has hypertrophic or restrictive cardiomyopathy
• Has active myocarditis or has been recently diagnosed with presumed or definitive myocarditis
• Has severe pulmonary hypertension
• Requires continuous home oxygen for significant pulmonary disease and/or has known interstitial lung disease
• Has severe chronic kidney disease
• Has hepatic disorder such as hepatic encephalopathy, hepatic laboratory abnormalities or Child Pugh Class C
• Has a gastrointestinal or biliary disorder that could impair absorption, metabolism, or excretion of medications
• Has concurrent or anticipated concomitant use of phosphodiesterase type 5 inhibitors or an sGC stimulator

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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342 participants in 2 patient groups, including a placebo group

Vericiguat

Experimental group

Description:

2.5 mg or 5 mg or 10 mg vericiguat administered orally once daily in tablet form for 52 weeks; or 0.2 mg/mL or 1 mg/mL vericiguat administered orally once daily in suspension form for 52 weeks

Treatment:

Drug: Vericiguat suspension

Drug: Vericiguat tablet

Placebo

Placebo Comparator group

Description:

Placebo for vericiguat administered orally once daily in tablet form for 52 weeks, or administered orally once daily in suspension form for 52 weeks

Treatment:

Drug: Placebo suspension

Drug: Placebo tablet