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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05587712




Registration number
NCT05587712
Ethics application status
Date submitted
17/10/2022
Date registered
20/10/2022

Titles & IDs
Public title
Study to Evaluate Sotatercept (MK-7962) in Children With Pulmonary Arterial Hypertension (PAH) (MK-7962-008)
Scientific title
A Phase 2 Open-label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sotatercept (MK-7962) in Children From 1 to Less Than 18 Years of Age With PAH on Standard of Care
Secondary ID [1] 0 0
MK-7962-008
Secondary ID [2] 0 0
7962-008
Universal Trial Number (UTN)
Trial acronym
MOONBEAM
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pulmonary Arterial Hypertension 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Cardiovascular 0 0 0 0
Hypertension

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Sotatercept

Experimental: Children =1 to <18 years old - Participants will receive a subcutaneous (SC) injection every 3 weeks (Q3W) of 0.3 mg/kg. Dosage may be adjusted based on protocol-specific guidelines.


Treatment: Drugs: Sotatercept
SC injection every 3 weeks (Q3W) of 0.3 mg/kg. Dosage may be adjusted based on protocol-specific guidelines.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Serum Trough Concentration (Ctrough) of Sotatercept
Timepoint [1] 0 0
Predose Day 1, Day 21, Day 42, Day 63, Day 84, Day105, Day 126, Day 147, Day 168, Day 189. Postdose Day 7, Day 14, Day 64, Day 69 and Day 76
Primary outcome [2] 0 0
Area Under the Curve at Steady State (AUCss) of Sotatercept
Timepoint [2] 0 0
Predose Day 1, Day 21, Day 42, Day 63, Day 84, Day105, Day 126, Day 147, Day 168, Day 189. Postdose Day 7, Day 14, Day 64, Day 69 and Day 76
Primary outcome [3] 0 0
Area Under the Curve from 0 to 3 weeks (AUC0-3 weeks) of Sotatercept
Timepoint [3] 0 0
Predose Day 1, Day 7, Day 14, and Predose Day 21
Primary outcome [4] 0 0
Percentage of Participants Who Experience at Least 1 Adverse Event (AE)
Timepoint [4] 0 0
Up to 24 weeks
Primary outcome [5] 0 0
Percentage of Participants Who Discontinue Study Drug Due to an AE
Timepoint [5] 0 0
Up to 24 weeks
Primary outcome [6] 0 0
Laboratory Parameter (Hematology): Concentration of Hemoglobin
Timepoint [6] 0 0
Up to 24 weeks
Primary outcome [7] 0 0
Laboratory Parameter (Hematology): Hematocrit
Timepoint [7] 0 0
Up to 24 weeks
Primary outcome [8] 0 0
Laboratory Parameter (Hematology): Red Blood Cell (RBC) Count
Timepoint [8] 0 0
Up to 24 weeks
Primary outcome [9] 0 0
Laboratory Parameter (Hematology): Reticulocyte Count
Timepoint [9] 0 0
Up to 24 weeks
Primary outcome [10] 0 0
Laboratory Parameter (Hematology): Platelet Count
Timepoint [10] 0 0
Up to 24 weeks
Primary outcome [11] 0 0
Blood Pressure (BP)
Timepoint [11] 0 0
Up to 24 weeks
Primary outcome [12] 0 0
Titer of Anti-drug Antibody (ADA) to Sotatercept
Timepoint [12] 0 0
Up to 24 weeks
Secondary outcome [1] 0 0
Mean Change from Baseline in 6-Minute Walk Distance (6MWD) (Cohorts 1 and 2)
Timepoint [1] 0 0
Baseline and Week 24
Secondary outcome [2] 0 0
Mean Change from Baseline in Tricuspid Annular Plane Systolic Excursion (TAPSE)
Timepoint [2] 0 0
Baseline and Week 24
Secondary outcome [3] 0 0
Mean Change from Baseline in Pulmonary Artery Systolic Pressure (PASP)
Timepoint [3] 0 0
Baseline and Week 24
Secondary outcome [4] 0 0
Mean Change from Baseline in Right Ventricular Fractional Area Change (RVFAC)
Timepoint [4] 0 0
Baseline and Week 24
Secondary outcome [5] 0 0
Mean Change from Baseline in Eccentricity Index
Timepoint [5] 0 0
Baseline and Week 24
Secondary outcome [6] 0 0
Mean Change from Baseline in Right Ventricular (RV) Function (Cohorts 1 and 2)
Timepoint [6] 0 0
Baseline and Week 24
Secondary outcome [7] 0 0
Mean Change from Baseline on Cardiac Output (Cohorts 1 and 2)
Timepoint [7] 0 0
Baseline and Week 24
Secondary outcome [8] 0 0
Mean Change from Baseline in Pulmonary Arterial Pressure (PAP) (Cohorts 1 and 2)
Timepoint [8] 0 0
Baseline and Week 24
Secondary outcome [9] 0 0
Mean Change from Baseline in Pediatric Quality of Life (PedsQL) Generic Score
Timepoint [9] 0 0
Baseline and Week 24
Secondary outcome [10] 0 0
Mean Change from Baseline in N-terminal Prohormone B-type Natriuretic Peptide (NT-proBNP)
Timepoint [10] 0 0
Baseline and Week 24
Secondary outcome [11] 0 0
Percentage of Participants Who Either Improved or Maintained Their World Health Organization Functional Class (WHO FC)
Timepoint [11] 0 0
Baseline and Week 24

Eligibility
Key inclusion criteria
Inclusion Criteria

* Documented, historic diagnostic right heart catheterization (RHC) any time before Screening confirming the diagnosis of PAH WHO Group 1 in any of the following subtypes:
* Idiopathic pulmonary arterial hypertension (IPAH)
* Heritable PAH
* Drug/toxin-induced PAH
* PAH associated with connective tissue disease
* PAH-congenital heart disease (CHD) with shunt closure >6 months before Screening and subsequently confirmed by RHC before Screening
* PAH with coincidental shunt.
* Must be on a stable dose(s) of background PAH therapy (phosphdiesterase-5 (PDE5) inhibitors, endothelin receptor antagonists (ERAs), soluble guanylate cyclase stimulators (sGCS), or prostanoids [including subcutaneous and intravenous])
* If male, agree to the following during the intervention period and for at least 16 weeks (112 days) after the last dose of study intervention:
* Abstains from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long-term and persistent basis) and agrees to remain abstinent or
* Uses contraception unless confirmed to be azoospermic (vasectomized or secondary to medical cause, documented from the site personnel's review of the participant's medical records, medical examination, or medical history interview) as detailed below:
* Uses a male condom plus partner use of an additional contraceptive method when having penile-vaginal intercourse with a woman of childbearing potential (WOCBP) who is not currently pregnant Note: Men with a pregnant or breastfeeding partner must agree to remain abstinent from penile-vaginal intercourse or use a male condom during each episode of penile-vaginal penetration.
* If female, must be either not a WOCBP or use a contraceptive method that is highly effective or be abstinent from heterosexual intercourse during the intervention period and for at least 16 weeks (112 days) after the last dose of study intervention
* If male, agrees to refrain from donating blood or sperm for the duration of the study and for 16 weeks (112 days) after the last dose of study intervention
* If female, agrees to refrain from donating blood, eggs, or ovum for the duration of the study and for at least 16 weeks (112 days) after the last dose of study intervention
Minimum age
1 Year
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion Criteria

* History of left-sided heart disease, including valvular disease (eg, moderate or greater mitral or aortic regurgitation or stenosis), left ventricular outflow tract obstruction, and/or left heart failure (eg, restrictive or dilated cardiomyopathy)
* Severe (as based on the opinion of the investigator) congenital or developmental abnormalities of the lung, thorax, and/or diaphragm
* History of Eisenmenger syndrome, Potts shunt, or atrial septostomy
* Unrepaired or residual cardiac shunt
* Diagnosis of pulmonary veno-occlusive diseases, pulmonary capillary hemangiomatosis, or overt signs of capillary and/or venous involvement
* PAH associated with portal hypertension
* Known visceral (lung, liver, or brain) arteriovenous malformation(s)
* History of full or partial pneumonectomy
* Untreated more than mild obstructive sleep apnea
* History of known pericardial constriction
* Family history of sudden cardiac death or long QT syndrome
* Any current or prior history of symptomatic coronary disease (myocardial infarction, percutaneous coronary intervention, coronary artery bypass graft surgery, or cardiac anginal chest pain) within 6 months before Screening
* Cerebrovascular accident within 3 months before Screening
* Prior exposure to sotatercept or luspatercept or has had an allergic reaction to any of their excipients

Study design
Purpose of the study
Treatment
Allocation to intervention
NA
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
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
19/01/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
21/09/2028
Actual
Sample size
Target
42
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
The Children's Hospital at Westmead ( Site 0001) - Westmead
Recruitment postcode(s) [1] 0 0
2145 - Westmead
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
Ohio
Country [5] 0 0
United States of America
State/province [5] 0 0
Pennsylvania
Country [6] 0 0
United States of America
State/province [6] 0 0
Tennessee
Country [7] 0 0
United States of America
State/province [7] 0 0
Washington
Country [8] 0 0
United States of America
State/province [8] 0 0
Wisconsin
Country [9] 0 0
Colombia
State/province [9] 0 0
Antioquia
Country [10] 0 0
Colombia
State/province [10] 0 0
Valle Del Cauca
Country [11] 0 0
France
State/province [11] 0 0
Haute-Garonne
Country [12] 0 0
France
State/province [12] 0 0
Provence-Alpes-Cote-d Azur
Country [13] 0 0
France
State/province [13] 0 0
Paris
Country [14] 0 0
Germany
State/province [14] 0 0
Baden-Wurttemberg
Country [15] 0 0
Germany
State/province [15] 0 0
Bayern
Country [16] 0 0
Germany
State/province [16] 0 0
Niedersachsen
Country [17] 0 0
Israel
State/province [17] 0 0
Petah-Tikva
Country [18] 0 0
Israel
State/province [18] 0 0
Ramat Gan
Country [19] 0 0
Netherlands
State/province [19] 0 0
Groningen
Country [20] 0 0
Poland
State/province [20] 0 0
Mazowieckie
Country [21] 0 0
Poland
State/province [21] 0 0
Pomorskie
Country [22] 0 0
South Africa
State/province [22] 0 0
Gauteng
Country [23] 0 0
Spain
State/province [23] 0 0
Madrid, Comunidad De
Country [24] 0 0
Spain
State/province [24] 0 0
Valencia
Country [25] 0 0
Spain
State/province [25] 0 0
Barcelona
Country [26] 0 0
Spain
State/province [26] 0 0
Madrid
Country [27] 0 0
Turkey
State/province [27] 0 0
Ankara
Country [28] 0 0
Turkey
State/province [28] 0 0
Istanbul
Country [29] 0 0
United Kingdom
State/province [29] 0 0
London, City Of

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
[email protected]
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/NCT05587712