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

NCT05714085 | Recruiting Phase 2 DMC FDA Drug

• 6 other identifiers: 1242-036MK-1242-0362022-501238-52-00U1111-1275-1768MK-1242-0432021-004399-33
• Study Type: interventional
• Target: 342
• Locations: 28 countries
• Sites: 102

Brief Summary

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.

Conditions

Heart Failure; Left Ventricular Systolic Dysfunction

Outcome Measures

Primary Outcomes (3)

• Base Period: Change from baseline to Week 16 in N-terminal pro-brain natriuretic peptide (NT-proBNP)

  The change from baseline to Week 16 of the Base Period in log-transformed NT-proBNP will be reported.

  Baseline and Week 16 of Base Period

• Extension Period: Percentage of participants with one or more adverse events (AEs)

  An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. The percentage of participants with one or more AEs in the Extension Period will be reported.

  Includes data collected up to a maximum of approximately 8 years

• Extension Period: Percentage of participants who discontinued study drug due to an AE

  An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. The percentage of participants who discontinue study drug in the Extension Period due to an AE will be reported.

  Includes data collected up to a maximum of approximately 8 years

Secondary Outcomes (8)

• Base Period: Change from baseline to Week 52 in log-transformed NT-proBNP

  Baseline and Week 52 of Base Period

• Base Period: First event of cardiovascular (CV) death, heart failure hospitalization (HFH), or worsening of heart failure (HF) without hospitalization

  Up to Week 54 of Base Period

• Base Period: Percentage of participants with one or more adverse events (AEs)

  Up to Week 54 of Base Period

• Base Period: Percentage of participants who discontinued study drug due to an AE

  Up to Week 52 of Base Period

• Base Period: Area under the curve from time 0-24 hours post-dose (AUC0-24) of plasma vericiguat

  Pre-dose, 2, 6, 16, 32 and 52 weeks post-dose (Base Period)

Study Arms (3)

Base Period: Vericiguat

EXPERIMENTAL

Participants in the Base Period receive 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.

Drug: Vericiguat tablet; Drug: Vericiguat suspension

Base Period: Placebo

PLACEBO COMPARATOR

Participants in the Base Period receive placebo for vericiguat administered orally once daily in tablet form for 52 weeks, or administered orally once daily in suspension form for 52 weeks.

Drug: Placebo tablet; Drug: Placebo suspension

Extension Period: Vericiguat

EXPERIMENTAL

Participants in the Extension Period receive either 2.5 mg or 5 mg or 10 mg vericiguat administered orally once daily in tablet form; or 0.2 mg/mL or 1 mg/mL vericiguat administered orally once daily in suspension form; following completion of the Base Period.

Drug: Vericiguat tablet; Drug: Vericiguat suspension

Interventions

Vericiguat tablet DRUG

2.5 mg or 5 mg or 10 mg vericiguat administered orally once daily in tablet form

Also known as: MK-1242

Base Period: Vericiguat; Extension Period: Vericiguat

Vericiguat suspension DRUG

0.2 mg/mL or 1 mg/mL vericiguat administered orally once daily in suspension form

Also known as: MK-1242

Base Period: Vericiguat; Extension Period: Vericiguat

Placebo tablet DRUG

Placebo for vericiguat administered orally once daily in tablet form

Also known as: Placebo for MK-1242

Base Period: Placebo

Placebo suspension DRUG

Placebo for vericiguat administered orally once daily in suspension form

Also known as: Placebo for MK-1242

Base Period: Placebo

Eligibility Criteria

• Age: 29 Days - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Merck Sharp & Dohme LLC: lead

Study Sites (105)

The Regents of the University of California - Los Angeles (UCLA Pediatrics) ( Site 0002)

Los Angeles, California, 90095, United States

RECRUITING

Lucile Packard Children's Hospital ( Site 0040)

Palo Alto, California, 94304, United States

RECRUITING

Loma Linda University Health System ( Site 0008)

San Bernardino, California, 92408, United States

RECRUITING

Children's Hospital Colorado ( Site 0012)

Aurora, Colorado, 80045, United States

RECRUITING

Children's National Medical Center ( Site 0020)

Washington D.C., District of Columbia, 20010, United States

RECRUITING

Johns Hopkins All Children's Hospital ( Site 0029)

St. Petersburg, Florida, 33701, United States

RECRUITING

Children's Healthcare of Atlanta - Arthur M. Blank Hospital ( Site 0001)

Atlanta, Georgia, 30329, United States

RECRUITING

Boston Children's Hospital ( Site 0035)

Boston, Massachusetts, 02115, United States

RECRUITING

C.S. Mott Children's Hospital ( Site 0033)

Ann Arbor, Michigan, 48109, United States

RECRUITING

Washington University-Pediatric Cardiology/ St. Louis Children's Hospital ( Site 0006)

St Louis, Missouri, 63110, United States

RECRUITING

Columbia University Medical Center-Pediatric Cardiology ( Site 0016)

New York, New York, 10032, United States

RECRUITING

The Children's Hospital at Montefiore ( Site 0030)

The Bronx, New York, 10467, United States

RECRUITING

Cincinnati Children's Hospital Medical Center ( Site 0034)

Cincinnati, Ohio, 45229, United States

RECRUITING

Cleveland Clinic-Cleveland Clinic Chidren's ( Site 0022)

Cleveland, Ohio, 44195, United States

RECRUITING

Children's Hospital of Philadelphia (CHOP) ( Site 0004)

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Children's Hospital of Pittsburgh ( Site 0010)

Pittsburgh, Pennsylvania, 15224, United States

RECRUITING

Le Bonheur Children's Hospital ( Site 0007)

Memphis, Tennessee, 38103, United States

RECRUITING

Children's Health-The Heart Center ( Site 0015)

Dallas, Texas, 75235, United States

RECRUITING

Texas Children's Hospital ( Site 0039)

Houston, Texas, 77030, United States

RECRUITING

Seattle Children's Hospital-Cardiology/Fetal Therapy ( Site 0019)

Seattle, Washington, 98105, United States

RECRUITING

Centre Hospitalier Régional de la Citadelle ( Site 0302)

Liège, Liege, 4000, Belgium

RECRUITING

UZ Gent ( Site 0301)

Ghent, Oost-Vlaanderen, 9000, Belgium

RECRUITING

UZ Leuven ( Site 0300)

Leuven, Vlaams-Brabant, 3000, Belgium

RECRUITING

Instituto Dante Pazzanese de Cardiology ( Site 0402)

São Paulo, São Paulo, 04012-909, Brazil

RECRUITING

Incor - Instituto do Coracao ( Site 0400)

São Paulo, 05403-000, Brazil

RECRUITING

Stollery Children's Hospital ( Site 0501)

Edmonton, Alberta, T6G 2B7, Canada

RECRUITING

Centre intégré universitaire de santé et de services sociaux-Centre de recherche du CHUS ( Site 0502)

Sherbrooke, Quebec, J1H 5H4, Canada

RECRUITING

Clinica Somer ( Site 0607)

Rionegro, Antioquia, 054040, Colombia

RECRUITING

Ciensalud Ips S A S ( Site 0608)

Barranquilla, Atlántico, 08001, Colombia

RECRUITING

Fundación Cardioinfantil Instituto de Cardiología ( Site 0603)

Bogotá, Bogota D.C., 110131, Colombia

RECRUITING

Fundación Valle del Lili ( Site 0604)

Cali, Valle del Cauca Department, 760032, Colombia

RECRUITING

Clínica Imbanaco S.A.S ( Site 0602)

Cali, Valle del Cauca Department, 760042, Colombia

RECRUITING

Klinički bolnički centar Zagreb ( Site 3700)

Zagreb, City of Zagreb, 10000, Croatia

RECRUITING

Rigshospitalet-BørneUngeAfdelingen ( Site 0800)

Copenhagen, Capital Region, DK-2100, Denmark

RECRUITING

Tampereen yliopistollinen sairaala-Pediatric Early Phase Trials Unit ( Site 0900)

Tampere, Pirkanmaa, 33520, Finland

RECRUITING

CHU Bordeaux Haut-Leveque ( Site 1000)

Pessac, Aquitaine, 33600, France

COMPLETED

Centre Hospitalier Universitaire de Nantes - Hôpital Femme-Enfant-Adolescent Chu De Nantes ( Site 1002)

Nantes, Loire-Atlantique, 44093, France

COMPLETED

CHU Lille - Institut Coeur Poumon ( Site 1005)

Lille, Nord, 59037, France

COMPLETED

Assistance Publique Hôpitaux de Marseille - Hôpital de la Timone ( Site 1003)

Marseille, Provence-Alpes-Côte d'Azur Region, 13005, France

COMPLETED

Hôpital Universitaire Necker Enfants Malades ( Site 1001)

Paris, 75015, France

COMPLETED

Assistance Publique - Hopitaux de Paris (AP-HP) - Hopital Robert Debre - Centre Hospitalo Universita ( Site 1004)

Paris, 75019, France

COMPLETED

Universitaetsklinikum Freiburg ( Site 1102)

Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

RECRUITING

Universitaetsklinikum Heidelberg ( Site 1100)

Heidelberg, Baden-Wurttemberg, 69120, Germany

COMPLETED

Kinderklinik des Uni-Klinikums Erlangen ( Site 1104)

Erlangen, Bavaria, 91054, Germany

RECRUITING

Medizinische Hochschule Hannover ( Site 1108)

Hanover, Lower Saxony, 30625, Germany

RECRUITING

Deutsches Herzzentrum Berlin ( Site 1101)

Berlin, 13353, Germany

RECRUITING

Gottsegen György Országos Kardiovaszkuláris Intézet-Gyermeksziv Kozpont ( Site 1300)

Budapest, 1096, Hungary

RECRUITING

Children's Health Ireland (CHI) at Crumlin ( Site 1400)

Dublin, D12 N512, Ireland

RECRUITING

IRCCS Istituto Giannina Gaslini ( Site 1603)

Genoa, Liguria, 16147, Italy

RECRUITING

Ospedale dei Bambini "Vittore Buzzi" ( Site 1606)

Milan, Lombardy, 20154, Italy

RECRUITING

Ospedale Pediatrico Bambino Gesù IRCCS ( Site 1602)

Rome, Roma, 00165, Italy

RECRUITING

A.O.Universitaria Meyer ( Site 1600)

Florence, Tuscany, 50139, Italy

COMPLETED

Azienda Ospedale - Università Padova ( Site 1601)

Padova, Veneto, 35128, Italy

RECRUITING

Hospital Universiti Sains Malaysia ( Site 1703)

Kota Bharu, Kelantan, 16150, Malaysia

RECRUITING

University Malaya Medical Centre ( Site 1701)

Lembah Pantai, Kuala Lumpur, 59100, Malaysia

RECRUITING

Hospital Queen Elizabeth II ( Site 1706)

Kota Kinabalu, Sabah, Kota Kinab, Malaysia

RECRUITING

Hospital Tunku Azizah-Paediatric ( Site 1700)

Kuala Lumpur, 50300, Malaysia

RECRUITING

Institut Jantung Negara ( Site 1705)

Kuala Lumpur, 50400, Malaysia

RECRUITING

Morales Vargas Centro de Investigacion ( Site 1810)

León, Guanajuato, 37000, Mexico

RECRUITING

CINVEC Medica ( Site 1814)

Guadalajara, Jalisco, 44690, Mexico

RECRUITING

Instituto Nacional de Pediatria ( Site 1803)

Mexico City, Mexico City, 04530, Mexico

RECRUITING

Hospital Universitario "Dr. Jose Eleuterio Gonzalez"-Pediatria ( Site 1816)

Monterrey, Nuevo León, 66460, Mexico

RECRUITING

INVECORDIS S.C. ( Site 1808)

Hacienda de Las Palmas, State of Mexico, 52763, Mexico

RECRUITING

Centro de Estudios de Investigacion Metabolicos y Cardiovasculares ( Site 1800)

Ciudad Madero, Tamaulipas, 89440, Mexico

RECRUITING

Centro de Atención e Investigación Clínica ( Site 1813)

Aguascalientes, 20129, Mexico

RECRUITING

UROLAP ( Site 1812)

Colima, 28000, Mexico

RECRUITING

Instituto Nacional de Cardiologia Ignacio Chavez ( Site 1804)

México, 14080, Mexico

RECRUITING

Erasmus Medisch Centrum ( Site 1900)

Rotterdam, South Holland, 3015 CN, Netherlands

RECRUITING

University Medical Center Groningen ( Site 1901)

Groningen, 9713 GZ, Netherlands

RECRUITING

Universitair Medisch Centrum Utrecht ( Site 1902)

Utrecht, 3584 CX, Netherlands

RECRUITING

Auckland City Hospital ( Site 2000)

Auckland, 1023, New Zealand

RECRUITING

Instituto Nacional Cardiovascular INCOR Carlos Peschiera Carrillo - EsSalud ( Site 2100)

Jesús María, Lima, 15072, Peru

RECRUITING

Uniwersyteckie Centrum Kliniczne-Klinika Kardiologii Dziecięcej i Wad Wrodzonych Serca ( Site 2302)

Gdansk, Pomeranian Voivodeship, 80-952, Poland

COMPLETED

Unidade Local de Saude Lisboa Ocidental - Hospital de Santa Cruz ( Site 2401)

Lisbon, Lisbon District, 2790-134, Portugal

RECRUITING

Unidade Local de Saude de Santa Maria - Hospital de Santa Maria ( Site 2402)

Lisbon, 1649-035, Portugal

RECRUITING

Unidade Local de Saúde de São João ( Site 2403)

Porto, 4200-319, Portugal

RECRUITING

Childrens University Hospital ( Site 4200)

Belgrade, Beograd, 11000, Serbia

RECRUITING

Institut za zdravstvenu zastitu majke i deteta Srbije ( Site 4220)

Belgrade, Beograd, 11000, Serbia

RECRUITING

Institute for Child and Youth Helth Care of Vojvodina ( Site 4210)

Novi Sad, Vojvodina, 21000, Serbia

RECRUITING

National University Hospital-Paediatrics ( Site 2600)

Singapore, South West, 119074, Singapore

RECRUITING

KK Women's and Children's Hospital ( Site 2601)

Singapore, South West, 229899, Singapore

RECRUITING

TREAD Research ( Site 2700)

Cape Town, Western Cape, 7500, South Africa

RECRUITING

Children's Heart Disease Research Unit ( Site 2704)

Cape Town, Western Cape, 7700, South Africa

RECRUITING

Pusan National University Yangsan Hospital ( Site 2802)

Pusan, Kyongsangnam-do, 50612, South Korea

RECRUITING

Seoul National University Hospital ( Site 2803)

Seoul, 03080, South Korea

RECRUITING

Severance Hospital, Yonsei University Health System ( Site 2800)

Seoul, 03722, South Korea

RECRUITING

Samsung Medical Center ( Site 2801)

Seoul, 06351, South Korea

RECRUITING

Hospital Sant Joan de Déu-Pediatric cardiology ( Site 2902)

Esplugues de Llobregat, Barcelona, 08950, Spain

RECRUITING

Hospital Universitari Vall d'Hebron ( Site 2903)

Barcelona, Catalonia, 08035, Spain

RECRUITING

Hospital Materno-Infantil Teresa Herrera ( Site 2905)

A Coruña, La Coruna, 15009, Spain

RECRUITING

HOSPITAL GENERAL UNIVERSITARIO GREGORIO MARAÑON ( Site 2904)

Madrid, Madrid, Comunidad de, 28007, Spain

RECRUITING

Hospital Universitario La Paz ( Site 2912)

Madrid, 28046, Spain

RECRUITING

HOSPITAL UNIVERSITARIO VIRGEN DEL ROCIO ( Site 2907)

Seville, 41013, Spain

RECRUITING

Skånes Universitetssjukhus Lund ( Site 3000)

Lund, Skåne County, 22185, Sweden

RECRUITING

Astrid Lindgrens Barnsjukhus ( Site 3001)

Solna, Stockholm County, 171 64, Sweden

RECRUITING

Faculty of Medicine Siriraj Hospital ( Site 3200)

Bangkoknoi, Bangkok, 10700, Thailand

RECRUITING

Faculty of Medicine - Khon Kaen University ( Site 3202)

Muang, Changwat Khon Kaen, 40002, Thailand

COMPLETED

Maharaj Nakorn Chiang Mai Hospital-Department of Pediatrics ( Site 3201)

Chiang Mai, 50200, Thailand

RECRUITING

Hacettepe Universite Hastaneleri ( Site 3304)

Ankara, 06230, Turkey (Türkiye)

RECRUITING

Baskent Universitesi Ankara Hastanesi ( Site 3301)

Ankara, 06490, Turkey (Türkiye)

RECRUITING

Ankara Bilkent Şehir Hastanesi. ( Site 3300)

Ankara, 06800, Turkey (Türkiye)

RECRUITING

Dr. Siyami Ersek Göğüs Kalp Ve Damar Cerrahisi Eğitim Ve Araştırma Hastanesi ( Site 3302)

Istanbul, 34668, Turkey (Türkiye)

RECRUITING

S.B.Ü. DR. BEHÇET UZ ÇOCUK HASTALIKLARI VE CERRAHİSİ EĞİTİM VE ARAŞTIRMA HASTANESİ ( Site 3303)

Izmir, 35210, Turkey (Türkiye)

RECRUITING

Great Ormond Street Hospital For Children NHS Foundation Trust ( Site 3401)

London, London, City of, WC1N 3JH, United Kingdom

RECRUITING

Freeman Hospital ( Site 3400)

Newcastle upon Tyne, NE7 7DN, United Kingdom

RECRUITING

Related Publications (1)

• Fritsch A, Meyer M, Blaustein RO, Trujillo ME, Kauh E, Roessig L, Boettcher M, Becker C. Clinical Pharmacokinetic and Pharmacodynamic Profile of Vericiguat. Clin Pharmacokinet. 2024 Jun;63(6):751-771. doi: 10.1007/s40262-024-01384-1. Epub 2024 Jun 25.

  PMID: 38916717 DERIVED

Related Links

• Plain Language Summary
• Merck Clinical Trials Information

MeSH Terms

Conditions

Heart Failure; Ventricular Dysfunction, Left

Interventions

vericiguat

Condition Hierarchy (Ancestors)

Heart Diseases; Cardiovascular Diseases; Ventricular Dysfunction

Study Officials

• Medical Director

  Merck Sharp & Dohme LLC

  STUDY DIRECTOR

Central Study Contacts

Toll Free Number

CONTACT

1-888-577-8839; Trialsites@msd.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 26, 2023
• First Posted: February 6, 2023
• Study Start: May 31, 2023
• Primary Completion (Estimated): April 15, 2032
• Study Completion (Estimated): April 15, 2032
• Last Updated: April 20, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

ME (9); CA (2); ZA (2); TH (3); CO (5); FI (1); NL (3); FR (6); IT (5); NZ (1); ES (6); BE (3); US (20); DE (5); PL (1); MY (5); KR (4); HU (1); SE (2); BR (2); SG (2); PE (1); TU (5); PT (3); GB (2); DK (1); IE (1); CR (1)