Study

Overview

Official Title: A Phase 2/3 Randomized, Placebo-Controlled, Double-blind, Clinical Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Vericiguat in Pediatric Participants with Heart Failure due to Systemic Left Ventricular Systolic Dysfunction (VALOR)

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 of the Base Period. The primary hypothesis is that vericiguat is superior to placebo in reducing NT-proBNP at Week 16 of the Base Period.

Study Type:

Interventional

Study Phase:

Phase 2/Phase 3

Estimated Enrollment:

342

Study Start Date:

May 2023

Estimated Study Completion Date:

April 2032

Estimated Primary Completion Date:

April 2032

Eligibility

Ages Eligible for Study:

29 days to 17 years

Genders Eligible for Study:

All

Accepts Healthy Volunteers:

No

CRITERIA

Inclusion Criteria:

Has 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.
Is of any sex/gender, from >28 days to <18 years of age inclusive. Must weigh ≥3 kg to participate.
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 during the study intervention period and for at least 30 days after study intervention; and their medical history; their menstrual history, and recent sexual activity has been reviewed.
Extension Period: Was randomized, received at least 1 dose of study intervention (vericiguat or placebo), did not permanently discontinue study intervention, and completed the Week 52 visit and safety follow-up period of the Base Period

Exclusion Criteria:

Is clinically unstable-with at least one of the following: has symptomatic hypotension or is hypotensive for age, recent use of intravenous (IV) inotrope and/or IV vasodilator, or recent IV diuretic.
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 acute coronary syndrome, undergone recent coronary intervention, or indication for coronary revascularization.
Has symptomatic carotid stenosis or other symptomatic cerebrovascular disease
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 significant bone disease (other than osteopenia) that in the assessment of the investigator can alter bone formation
Has concurrent or anticipated concomitant use of phosphodiesterase type 5 inhibitors or an sGC stimulator.
Has received a COVID-19 vaccination within 1 week before randomization.

Locations

United States Toll Free Number 1-800-770-4674

Loma Linda, California, 92354
Los Angeles, California, 90095
Aurora, Colorado, 80045
Washington, District of Columbia, 20010
Saint Petersburg, Florida, 33701
Atlanta, Georgia, 30329
Ann Arbor, Michigan, 48109
St. Louis, Missouri, 63110
Bronx, New York, 10467
New York, New York, 10032
Cincinnati, Ohio, 45245
Cleveland, Ohio, 44195
Philadelphia, Pennsylvania, 19104
Pittsburgh, Pennsylvania, 15224
Memphis, Tennessee, 38103
Dallas, Texas, 75235
Seattle, Washington, 98105

Canada Study Coordinator 1-888-577-8839

Results

No Study Results Posted

Study Detail

Efficacy, safety, and pharmacokinetics of vericiguat in pediatric participants with heart failure due to left ventricular systolic dysfunction (MK-1242-036)

Overview

Eligibility

CRITERIA

Locations

Results