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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
Date last updated
18/11/2024

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: Vericiguat - 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: Placebo - 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: 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
Change from baseline to Week 16 in N-terminal pro-brain natriuretic peptide (NT-proBNP)
Timepoint [1] 0 0
Baseline and Week 16
Secondary outcome [1] 0 0
Change from baseline to Week 52 in log-transformed NT-proBNP
Timepoint [1] 0 0
1Baseline and Week 52
Secondary outcome [2] 0 0
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
Secondary outcome [3] 0 0
Participants with one or more adverse events (AE)
Timepoint [3] 0 0
Up to Week 54
Secondary outcome [4] 0 0
Participants who discontinued study drug due to an AE
Timepoint [4] 0 0
Up to Week 52
Secondary outcome [5] 0 0
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
Secondary outcome [6] 0 0
Half-life (t1/2) of vericiguat in plasma
Timepoint [6] 0 0
Pre-dose, 2, 6, 16, 32 and 52 weeks post-dose
Secondary outcome [7] 0 0
Oral clearance (CL/F) of plasma vericiguat
Timepoint [7] 0 0
Pre-dose, 2, 6, 16, 32 and 52 weeks post-dose

Eligibility
Key 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
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: 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

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
30/04/2030
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
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United States of America
State/province [2] 0 0
Colorado
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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
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United States of America
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New York
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United States of America
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Ohio
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United States of America
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Pennsylvania
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United States of America
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Tennessee
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United States of America
State/province [12] 0 0
Texas
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United States of America
State/province [13] 0 0
Washington
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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
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Colombia
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Atlantico
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Colombia
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Distrito Capital De Bogota
Country [23] 0 0
Colombia
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Valle Del Cauca
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Denmark
State/province [24] 0 0
Hovedstaden
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Finland
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Pirkanmaa
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France
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Aquitaine
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France
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Nord
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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
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Germany
State/province [34] 0 0
Berlin
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Hungary
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Budapest
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Ireland
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Dublin
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Italy
State/province [37] 0 0
Liguria
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Italy
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Toscana
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Korea, Republic of
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Kyongsangnam-do
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Korea, Republic of
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Seoul
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Malaysia
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Kuala Lumpur
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Mexico
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Distrito Federal
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Mexico
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Guanajuato
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Mexico
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Aguascalientes
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Mexico
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Hacienda De Las Palmas
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Netherlands
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Zuid-Holland
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Netherlands
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Groningen
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Netherlands
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Utrecht
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New Zealand
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Auckland
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Portugal
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Lisboa
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Portugal
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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
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Barcelona
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Spain
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Madrid, Comunidad De
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Spain
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Sevilla
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Sweden
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Skane Lan
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Thailand
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Khon Kaen
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Thailand
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Krung Thep Maha Nakhon
Country [60] 0 0
Thailand
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Chiang Mai
Country [61] 0 0
Turkey
State/province [61] 0 0
Ankara
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Turkey
State/province [62] 0 0
Istanbul
Country [63] 0 0
Turkey
State/province [63] 0 0
Izmir
Country [64] 0 0
United Kingdom
State/province [64] 0 0
London, City Of
Country [65] 0 0
United Kingdom
State/province [65] 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
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.
Trial website
https://clinicaltrials.gov/study/NCT05714085
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
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05714085