Open-label Extension of Vericiguat in Pediatric Participants with Heart Failure due to Left Ventricular Systolic Dysfunction

2023-506210-40-00 Protocol MK-1242-043 Therapeutic confirmatory (Phase III) Ended

Start 28 Jun 2024 · End 15 Aug 2025 · Status Ended · 13 EU/EEA countries · 29 sites · Protocol MK-1242-043

Overview

Sponsor-declared trial summary

Phase Therapeutic confirmatory (Phase III)
Status Ended
Participants planned 254
Countries 13
Sites 29

Heart Failure due to Systemic Left Ventricular Systolic Dysfunction

To monitor safety and tolerability of vericiguat (MK-1242).

Key facts

Sponsor
Merck Sharp & Dohme LLC
Participant type
Pediatric, Patients
Age range
0-17 years, 18-64 years
Gender
Male and Female
Therapeutic area
Diseases [C] - Cardiovascular Diseases [C14]
Trial duration
28 Jun 2024 → 15 Aug 2025
Decision date (initial)
2024-03-18
Transition trial
No
Low-intervention
No
Rare-disease indication
No
Vulnerable population
Yes
Funding sources
Merck Sharp & Dohme LLC · Bayer AG

External identifiers

EU CT number
2023-506210-40-00
WHO UTN
U1111-1293-4744

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Therapy, Safety

To monitor safety and tolerability of vericiguat (MK-1242).

Secondary objectives 1

  1. To evaluate change in NT-proBNP from baseline to Week 16.

Conditions and MedDRA coding

Heart Failure due to Systemic Left Ventricular Systolic Dysfunction

VersionLevelCodeTermSystem organ class
20.0 LLT 10069501 Left ventricular systolic dysfunction 10007541
20.0 LLT 10019279 Heart failure 10007541

Regulatory references

Plan to share IPD
Yes
EU CT numberTitleSponsor
2022-501238-52-00 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) Merck Sharp & Dohme LLC

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

  1. Has received at least 1 dose of study intervention (vericiguat or placebo) and completed the Week 52 visit and safety follow-up period for the VALOR base study. This includes participants who prematurely discontinued study intervention unless premature discontinuation of study intervention was due to a drug related adverse event (AE) or to noncompliance with study intervention
  2. Is able to receive medication via the oral or gastric route

Exclusion criteria 6

  1. Is hypotensive for age at Visit 1
  2. Has undergone heart transplantation or has an implanted ventricular assist device
  3. Has severe chronic kidney disease or requires chronic dialysis
  4. Has hepatic disorder or Child Pugh Class C
  5. Has concurrent or anticipated concomitant use of phosphodiesterase type 5 inhibitors during the study
  6. Has concurrent or anticipated use of an soluble guanylate cyclase (sGC) stimulator

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

  1. Number of Participants Who Experience an Adverse Event (AE)
  2. Number of Participants Who Discontinue Study Treatment Due to an AE

Secondary endpoints 1

  1. Change from baseline to Week 16 in N-terminal pro-brain natriuretic peptide (NT-proBNP)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 4

Vericiguat

PRD9354183 · Product

Active substance
Vericiguat
Substance synonyms
BAY 1021189, MK-1242
Pharmaceutical form
TABLET
Route of administration
ORAL USE
Max daily dose
10 mg milligram(s)
Max total dose
33 g gram(s)
Max treatment duration
108 Month(s)
Authorisation status
Not Authorised
MA holder
MERCK & CO. INC.
Paediatric formulation
No
Orphan designation
No

Vericiguat

PRD9744926 · Product

Active substance
Vericiguat
Substance synonyms
BAY 1021189, MK-1242
Pharmaceutical form
ORAL SUSPENSION
Route of administration
ORAL USE
Max daily dose
10 mg milligram(s)
Max total dose
33 g gram(s)
Max treatment duration
108 Month(s)
Authorisation status
Not Authorised
MA holder
MERCK & CO. INC.
Paediatric formulation
Yes
Orphan designation
No

Vericiguat

PRD9354181 · Product

Active substance
Vericiguat
Substance synonyms
BAY 1021189, MK-1242
Pharmaceutical form
TABLET
Route of administration
ORAL USE
Max daily dose
10 mg milligram(s)
Max total dose
33 g gram(s)
Max treatment duration
108 Month(s)
Authorisation status
Not Authorised
MA holder
MERCK & CO. INC.
Paediatric formulation
No
Orphan designation
No

Vericiguat

PRD9354182 · Product

Active substance
Vericiguat
Substance synonyms
BAY 1021189, MK-1242
Pharmaceutical form
TABLET
Route of administration
ORAL USE
Max daily dose
10 mg milligram(s)
Max total dose
33 g gram(s)
Max treatment duration
108 Month(s)
Authorisation status
Not Authorised
MA holder
MERCK & CO. INC.
Paediatric formulation
No
Orphan designation
No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Merck Sharp & Dohme LLC

290 Total trials 92 Recruiting 184 Ended
Commercial
Sponsor organisation
Merck Sharp & Dohme LLC
Address
126 East Lincoln Avenue
City
Rahway
Postcode
07065-4607
Country
United States

Scientific contact point

Organisation
Merck Sharp & Dohme LLC
Contact name
Mackenzie Ford

Public contact point

Organisation
Merck Sharp & Dohme LLC
Contact name
Mackenzie Ford

Third parties 5

OrganisationCity, countryDuties
Bioclinica Inc.
ORG-100033079
 Princeton, United States Other
Parexel International Corp.
ORG-100007310
 Auburndale, United States Other
Perceptive Eclinical Limited
ORG-100041144
 Nottingham, United Kingdom Other
Almac Clinical Technologies LLC
ORG-100043036
 Souderton, United States Interactive response technologies (IRT)
PPD Global Central Labs
ORG-100046496
 Zaventem, Belgium Laboratory analysis

Locations

13 EU/EEA countries · 29 investigational sites

By country

CountryMS statusPlanned subjectsSites
Belgium Ended 8 3
Denmark Ended 3 1
Finland Ended 3 1
France Ended 21 6
Germany Ended 18 5
Hungary Ended 6 1
Ireland Ended 4 1
Italy Ended 7 2
Netherlands Ended 7 2
Poland Ended 3 1
Portugal Ended 10 3
Spain Ended 6 2
Sweden Ended 3 1
Rest of world
Korea, Republic of, Peru, New Zealand, Turkey, Colombia, United Kingdom, Singapore, Thailand, Canada, Brazil, South Africa, Malaysia, United States, Mexico
 — 155 —

Investigational sites

Belgium

3 sites · Ended
UZ Leuven
Kindercardiologie, Herestraat 49, 3000, Leuven
Centre Hospitalier Regional De La Citadelle
Cardiologie pédiatrique, Bld Du Douzieme-De-Ligne 1, 4000, Liege
Universitair Ziekenhuis Gent
Kindercardiologie, Corneel Heymanslaan 10, 9000, Gent

Denmark

1 site · Ended
Rigshospitalet
BørneUngeKlinikken Børnekardiologisk afsnit 5004, Blegdamsvej 9, 2100, Copenhagen Oe

Finland

1 site · Ended
Tampere University Hospital
Pediatric Early Phase Trials Unit, Elamanaukio 2, 33520, Tampere

France

6 sites · Ended
Hopital Necker Enfants Malades
Service de Cardiologie Pédiatrique et Congénitale, 149 Rue De Sevres, 75015, Paris
Centre Hospitalier Universitaire De Bordeaux
Unité Médico-chirurgicale des pathologies cardiaques congénitales du fœtus, de l'enfant, de l'adulte, Avenue De Magellan, 33600, Pessac
Assistance Publique Hopitaux De Marseille
Cardiologie pédiatrique et congénitale, 264 Rue Saint Pierre, 13005, Marseille
Robert Debre University Hospital
Cardiologie pédiatrique, 48 Boulevard Serurier, 75019, Paris
Centre Hospitalier Universitaire De Nantes
Département Médico-chirurgical de cardiologie pédiatrique et congénitale, 7 Quai Moncousu, 44000, Nantes
Centre Hospitalier Universitaire De Lille
Cardiologie infantile et congénitale, Avenue Eugene Avinee, 59037, Lille Cedex

Germany

5 sites · Ended
Medical Center - University Of Freiburg
Klinik für Angeborene Herzfehler und Pädiatrische Kardiologie, Hugstetter Strasse 55, Stuehlinger, Freiburg Im Breisgau
Medizinische Hochschule Hannover
Klinik für Pädiatrische Kardiologie und Pädiatrische Intensivmedizin, Carl-Neuberg-Strasse 1, Gross Buchholz, Hanover
Charite Universitaetsmedizin Berlin KöR
Deutsches Herzzentrum Berlin Klinik für angeborene Herzfehler, Augustenburger Platz 1, Wedding, Berlin
Universitaetsklinikum Erlangen AöR
Kinderkardiologie, Loschgestrasse 15, Innenstadt, Erlangen
Universitaetsklinikum Heidelberg AöR
Klinik für Kinderkardiologie und Angeborene Herzfehler, Im Neuenheimer Feld 430, Neuenheim, Heidelberg

Hungary

1 site · Ended
Gottsegen National Cardiovascular Center
Gyermekszív Központ, Kerulet, Haller Utca 29/IX., Budapest

Ireland

1 site · Ended
Children's Health Ireland
Department of Paediatric Cardiology, Cooley Road, Crumlin, Dublin

Italy

2 sites · Ended
Azienda Ospedaliera Universitaria Meyer IRCCS
Cardiologia Pediatrica, Viale Gaetano Pieraccini 24, 50139, Florence
Azienda Ospedale-Universita Padova
Cardiologia Pediatrica, Via Nicolo' Giustiniani 2, 35128, Padova

Netherlands

2 sites · Ended
Universitair Medisch Centrum Groningen
Kindercardiologie, Hanzeplein 1, 9713 GZ, Groningen
Universitair Medisch Centrum Utrecht
Kindercardiologie, Heidelberglaan 100, 3584 CX, Utrecht

Poland

1 site · Ended
Uniwersyteckie Centrum Kliniczne
Klinika Kardiologii Dziecięcej i Wad Wrodzonych Serca, Ul. Debinki 7, 80-952, Gdansk

Portugal

3 sites · Ended
Centro Hospitalar Universitario De Lisboa Norte E.P.E.
Serviço de Cardiologia Pediátrica, Avenida Professor Egas Moniz, 1649-035, Lisbon
Sao Joao University Hospital Center
Serviço de Cardiologia Pediátrica, Alameda Professor Hernani Monteiro, 4200-319, Porto
Centro Hospitalar De Lisboa Ocidental E.P.E.
Serviço de Cardiologia Pediátrica, Av Prof Dr Reinaldo Dos Santos, 2790-134, Carnaxide

Spain

2 sites · Ended
Hospital General Universitario Gregorio Maranon
Cardiología, Calle Del Doctor Esquerdo 46, 28009, Madrid
Sant Joan De Deu Barcelona Hospital
Cardiología, Passeig De Sant Joan De Deu 2, 08950, Esplugues De Llobregat

Sweden

1 site · Ended
Region Skane Skanes Universitetssjukhus
Barnhjärtcentrum, Entregatan 7, 222 42, Lund

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Country Trial startTrial end Recruitment startRecruitment end Early termination
Hungary 2024-06-28 2024-07-03
Ireland 2024-11-25 2025-02-24 2025-02-24

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Summary of results Art. 37(4) CTR

TitleSubmission dateStatusType
Summary of results_2023-506210-40_for pub
SUM-119575
 2026-02-16T16:50:16 Submitted Summary of Results

Layperson summary Annex V

TitleSubmission dateStatusType
RPLS_2023-506210-40_for pub 2026-02-16T16:55:22 Submitted Laypersons Summary of Results

Documents 43 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

TypeTitleVersion
Laypersons summary of results (for publication) D1_RPLS_2023-506210-40_ESP_ES_for pub 09JAN2026
Laypersons summary of results (for publication) D1_RPLS_2023-506210-40_FIN_FI_for pub 09JAN2026
Laypersons summary of results (for publication) RPLS_2023-506210-40_BEL_DE_for pub 09JAN2026
Laypersons summary of results (for publication) RPLS_2023-506210-40_BEL_FR_for pub 09JAN2026
Laypersons summary of results (for publication) RPLS_2023-506210-40_BEL_NL_for pub 09JAN2026
Laypersons summary of results (for publication) RPLS_2023-506210-40_DEU_DE_for pub 09JAN2026
Laypersons summary of results (for publication) RPLS_2023-506210-40_DNK_DA_for pub 09JAN2026
Laypersons summary of results (for publication) RPLS_2023-506210-40_for pub 09JAN2026
Laypersons summary of results (for publication) RPLS_2023-506210-40_FRA_FR_for pub 09JAN2026
Laypersons summary of results (for publication) RPLS_2023-506210-40_HUN_HU_for pub 09JAN2026
Laypersons summary of results (for publication) RPLS_2023-506210-40_ITA_IT_for pub 09JAN2026
Laypersons summary of results (for publication) RPLS_2023-506210-40_NLD_NL_for pub 09JAN2026
Laypersons summary of results (for publication) RPLS_2023-506210-40_POL_PL_for pub 09JAN2026
Laypersons summary of results (for publication) RPLS_2023-506210-40_PRT_PT_for pub 09JAN2026
Laypersons summary of results (for publication) RPLS_2023-506210-40_SWE_SV_for pub 09JAN2026
Protocol (for publication) D1_Protocol_2023-506210-40_for pub 00
Protocol (for publication) D4_Study intervention log_16W_EN_for pub 1.0 00 1.0
Protocol (for publication) D4_Study intervention log_24W_EN_for pub 1.0 00 1.0
Protocol (for publication) D4_Study intervention log_6W_EN_for pub 1.0 00 1.0
Recruitment arrangements (for publication) K1_Recruitment Arrangements and IC Procedure_IRL_EN_for pub 1.0
Subject information and informed consent form (for publication) L1_ICF_Main assent 02-05 yr_IRL_EN_for pub 00
Subject information and informed consent form (for publication) L1_ICF_Main assent 06-09 yr_IRL_EN_for pub 00
Subject information and informed consent form (for publication) L1_ICF_Main assent 10-15 yr_IRL_EN_for pub 00
Subject information and informed consent form (for publication) L1_ICF_Main consent 16-17 yr_IRL_EN_for pub 0.00a
Subject information and informed consent form (for publication) L1_ICF_Main data protection 18 yr_IRL_EN_for pub 0.00a
Subject information and informed consent form (for publication) L1_ICF_Main data protection parent guardian 16-17 yr_IRL_EN_for pub 0.00a
Subject information and informed consent form (for publication) L1_ICF_Main parent guardian_IRL_EN_Access_SM01_not pub 01
Subject information and informed consent form (for publication) L1_ICF_Main parent guardian_IRL_EN_SM01_for pub 01
Subject information and informed consent form (for publication) L1_ICF_Optional_Greenphire guardian_IRL_EN_Access_SM01_not pub 00
Subject information and informed consent form (for publication) L1_ICF_Optional_Greenphire guardian_IRL_EN_SM01_for pub 00
Summary of results (for publication) Summary of results_2023-506210-40_for pub 09JAN2026
Synopsis of the protocol (for publication) D1_PPLS_BEL_DE_2023-506210-40_for pub 1.0
Synopsis of the protocol (for publication) D1_PPLS_BEL_FR_2023-506210-40_for pub 1.0
Synopsis of the protocol (for publication) D1_PPLS_BEL_NL_2023-506210-40_for pub 1.0
Synopsis of the protocol (for publication) D1_PPLS_DEU_DE_2023-506210-40_for pub 1.0
Synopsis of the protocol (for publication) D1_PPLS_EN_2023-506210-40_for pub 1.0
Synopsis of the protocol (for publication) D1_PPLS_ESP_ES_2023-506210-40 _for pub 1.0
Synopsis of the protocol (for publication) D1_PPLS_FRA_FR_2023-506210-40_for pub 1.0
Synopsis of the protocol (for publication) D1_PPLS_HUN_HU_2023-506210-40_for pub 1.0
Synopsis of the protocol (for publication) D1_PPLS_ITA_IT_for pub 1
Synopsis of the protocol (for publication) D1_PPLS_NLD_NL_2023-506210-40_for pub 1.0
Synopsis of the protocol (for publication) D1_PPLS_POL_PL_2023-506210-40_for pub 1.0
Synopsis of the protocol (for publication) D1_PPLS_SWE_SV_2023-506210-40_for pub 1.0

Application history

6 submissions — initial application plus substantial / non-substantial modifications

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2023-11-16 Sweden Acceptable
2024-03-11
 2024-03-12
2 NON SUBSTANTIAL MODIFICATION NSM-1 2024-03-26 Acceptable
2024-03-11
 2024-03-26
3 NON SUBSTANTIAL MODIFICATION NSM-3 2024-04-11 Sweden Acceptable
2024-03-11
 2024-04-11
4 SUBSTANTIAL MODIFICATION SM-1 2024-10-30 Acceptable 2024-12-04
5 NON SUBSTANTIAL MODIFICATION NSM-4 2025-03-12 Sweden Acceptable 2025-03-12
6 NON SUBSTANTIAL MODIFICATION NSM-5 2025-04-23 Sweden Acceptable 2025-04-23

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