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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05714085




Registration number
NCT05714085
Ethics application status
Date submitted
26/01/2023
Date registered
6/02/2023

Titles & IDs
Public title
Efficacy, Safety, and Pharmacokinetics of Vericiguat in Pediatric Participants With Heart Failure Due to Left Ventricular Systolic Dysfunction (MK-1242-036)
Scientific 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)
Secondary ID [1] 0 0
MK-1242-036
Secondary ID [2] 0 0
1242-036
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Heart Failure 0 0
Left Ventricular Systolic Dysfunction 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Coronary heart disease
Cardiovascular 0 0 0 0
Other cardiovascular diseases
Cardiovascular 0 0 0 0
Other cardiovascular diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Vericiguat tablet
Treatment: Drugs - Vericiguat suspension
Treatment: Drugs - Placebo tablet
Treatment: Drugs - Placebo suspension

Experimental: Base Period: Vericiguat - 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.

Placebo comparator: Base Period: Placebo - 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.

Experimental: Extension Period: Vericiguat - 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.


Treatment: Drugs: Vericiguat tablet
2.5 mg or 5 mg or 10 mg vericiguat administered orally once daily in tablet form

Treatment: Drugs: Vericiguat suspension
0.2 mg/mL or 1 mg/mL vericiguat administered orally once daily in suspension form

Treatment: Drugs: Placebo tablet
Placebo for vericiguat administered orally once daily in tablet form

Treatment: Drugs: Placebo suspension
Placebo for vericiguat administered orally once daily in suspension form
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Base Period: Change from baseline to Week 16 in N-terminal pro-brain natriuretic peptide (NT-proBNP)
Timepoint [1] 0 0
Baseline and Week 16 of Base Period
Primary outcome [2] 0 0
Extension Period: Percentage of participants with one or more adverse events (AEs)
Timepoint [2] 0 0
Includes data collected up to a maximum of approximately 8 years
Primary outcome [3] 0 0
Extension Period: Percentage of participants who discontinued study drug due to an AE
Timepoint [3] 0 0
Includes data collected up to a maximum of approximately 8 years
Secondary outcome [1] 0 0
Base Period: Change from baseline to Week 52 in log-transformed NT-proBNP
Timepoint [1] 0 0
Baseline and Week 52 of Base Period
Secondary outcome [2] 0 0
Base Period: First event of cardiovascular (CV) death, heart failure hospitalization (HFH), or worsening of heart failure (HF) without hospitalization
Timepoint [2] 0 0
Up to Week 54 of Base Period
Secondary outcome [3] 0 0
Base Period: Percentage of participants with one or more adverse events (AEs)
Timepoint [3] 0 0
Up to Week 54 of Base Period
Secondary outcome [4] 0 0
Base Period: Percentage of participants who discontinued study drug due to an AE
Timepoint [4] 0 0
Up to Week 52 of Base Period
Secondary outcome [5] 0 0
Base Period: Area under the curve from time 0-24 hours post-dose (AUC0-24) of plasma vericiguat
Timepoint [5] 0 0
Pre-dose, 2, 6, 16, 32 and 52 weeks post-dose (Base Period)
Secondary outcome [6] 0 0
Base Period: Half-life (t1/2) of vericiguat in plasma
Timepoint [6] 0 0
Pre-dose, 2, 6, 16, 32 and 52 weeks post-dose (Base Period)
Secondary outcome [7] 0 0
Base Period: Oral clearance (CL/F) of plasma vericiguat
Timepoint [7] 0 0
Pre-dose, 2, 6, 16, 32 and 52 weeks post-dose (Base Period)
Secondary outcome [8] 0 0
Extension Period: Change from extension period baseline to extension period Week 16 in NT-proBNP
Timepoint [8] 0 0
Extension Period Baseline (Study Week 54) and Extension Period Week 16 (Study Week 70)

Eligibility
Key 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
Minimum age
29 Days
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
31/05/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
15/04/2032
Actual
Sample size
Target
342
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
District of Columbia
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Georgia
Country [6] 0 0
United States of America
State/province [6] 0 0
Michigan
Country [7] 0 0
United States of America
State/province [7] 0 0
Missouri
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
Ohio
Country [10] 0 0
United States of America
State/province [10] 0 0
Pennsylvania
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United States of America
State/province [11] 0 0
Tennessee
Country [12] 0 0
United States of America
State/province [12] 0 0
Texas
Country [13] 0 0
United States of America
State/province [13] 0 0
Washington
Country [14] 0 0
Belgium
State/province [14] 0 0
Liege
Country [15] 0 0
Belgium
State/province [15] 0 0
Oost-Vlaanderen
Country [16] 0 0
Belgium
State/province [16] 0 0
Vlaams-Brabant
Country [17] 0 0
Brazil
State/province [17] 0 0
Sao Paulo
Country [18] 0 0
Canada
State/province [18] 0 0
Alberta
Country [19] 0 0
Canada
State/province [19] 0 0
Quebec
Country [20] 0 0
Colombia
State/province [20] 0 0
Antioquia
Country [21] 0 0
Colombia
State/province [21] 0 0
Atlantico
Country [22] 0 0
Colombia
State/province [22] 0 0
Distrito Capital De Bogota
Country [23] 0 0
Colombia
State/province [23] 0 0
Valle Del Cauca
Country [24] 0 0
Denmark
State/province [24] 0 0
Hovedstaden
Country [25] 0 0
Finland
State/province [25] 0 0
Pirkanmaa
Country [26] 0 0
France
State/province [26] 0 0
Aquitaine
Country [27] 0 0
France
State/province [27] 0 0
Nord
Country [28] 0 0
France
State/province [28] 0 0
Pays-de-la-Loire
Country [29] 0 0
France
State/province [29] 0 0
Provence-Alpes-Cote-d Azur
Country [30] 0 0
France
State/province [30] 0 0
Paris
Country [31] 0 0
Germany
State/province [31] 0 0
Baden-Wurttemberg
Country [32] 0 0
Germany
State/province [32] 0 0
Bayern
Country [33] 0 0
Germany
State/province [33] 0 0
Niedersachsen
Country [34] 0 0
Germany
State/province [34] 0 0
Berlin
Country [35] 0 0
Hungary
State/province [35] 0 0
Budapest
Country [36] 0 0
Ireland
State/province [36] 0 0
Dublin
Country [37] 0 0
Italy
State/province [37] 0 0
Liguria
Country [38] 0 0
Italy
State/province [38] 0 0
Toscana
Country [39] 0 0
Korea, Republic of
State/province [39] 0 0
Kyongsangnam-do
Country [40] 0 0
Korea, Republic of
State/province [40] 0 0
Seoul
Country [41] 0 0
Malaysia
State/province [41] 0 0
Kuala Lumpur
Country [42] 0 0
Mexico
State/province [42] 0 0
Distrito Federal
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Mexico
State/province [43] 0 0
Guanajuato
Country [44] 0 0
Mexico
State/province [44] 0 0
Aguascalientes
Country [45] 0 0
Mexico
State/province [45] 0 0
Hacienda De Las Palmas
Country [46] 0 0
Netherlands
State/province [46] 0 0
Zuid-Holland
Country [47] 0 0
Netherlands
State/province [47] 0 0
Groningen
Country [48] 0 0
Netherlands
State/province [48] 0 0
Utrecht
Country [49] 0 0
New Zealand
State/province [49] 0 0
Auckland
Country [50] 0 0
Peru
State/province [50] 0 0
Lima
Country [51] 0 0
Portugal
State/province [51] 0 0
Lisboa
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Portugal
State/province [52] 0 0
Porto
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Singapore
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South West
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South Africa
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Western Cape
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Spain
State/province [55] 0 0
Barcelona
Country [56] 0 0
Spain
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La Coruna
Country [57] 0 0
Spain
State/province [57] 0 0
Madrid, Comunidad De
Country [58] 0 0
Spain
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Sevilla
Country [59] 0 0
Sweden
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Skane Lan
Country [60] 0 0
Thailand
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Khon Kaen
Country [61] 0 0
Thailand
State/province [61] 0 0
Krung Thep Maha Nakhon
Country [62] 0 0
Thailand
State/province [62] 0 0
Chiang Mai
Country [63] 0 0
Turkey
State/province [63] 0 0
Ankara
Country [64] 0 0
Turkey
State/province [64] 0 0
Istanbul
Country [65] 0 0
Turkey
State/province [65] 0 0
Izmir
Country [66] 0 0
United Kingdom
State/province [66] 0 0
London, City Of
Country [67] 0 0
United Kingdom
State/province [67] 0 0
Newcastle upon Tyne

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Merck Sharp & Dohme LLC
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Merck Sharp & Dohme LLC
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Toll Free Number
Address 0 0
Country 0 0
Phone 0 0
1-888-577-8839
Fax 0 0
Email 0 0
Trialsites@msd.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
http://engagezone.msd.com/doc/ProcedureAccessClinicalTrialData.pdf
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: http://engagezone.msd.com/ds_documentation.php


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05714085