Study to Evaluate Sotatercept (MK-7962) in Children With Pulmonary Arterial Hypertension (PAH) (MK-7962-008)

• Register: ClinicalTrials.gov
• Last refreshed on: 31 March 2025
• Main ID: NCT05587712
• Date of registration: 17/10/2022
• Prospective Registration: Yes
• Primary sponsor: Merck Sharp & Dohme LLC
• Public title: Study to Evaluate Sotatercept (MK-7962) in Children With Pulmonary Arterial Hypertension (PAH) (MK-7962-008) MOONBEAM
• 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
• Date of first enrolment: January 19, 2023
• Target sample size: 42
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT05587712
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Australia, Colombia, France, Germany, Israel, Netherlands, Poland, South Africa, Spain, Turkey, United Kingdom, United States

Contacts

• Name: Medical Director
• Affiliation: Merck Sharp & Dohme LLC

• Name: Toll Free Number

Key inclusion & exclusion 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

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


Age minimum: 1 Year
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Pulmonary Arterial Hypertension

Intervention(s)

Drug: Sotatercept

Primary Outcome(s)

Laboratory Parameter (Hematology): Red Blood Cell (RBC) Count [Time Frame: Up to 24 weeks]

Blood Pressure (BP) [Time Frame: Up to 24 weeks]

Laboratory Parameter (Hematology): Concentration of Hemoglobin [Time Frame: Up to 24 weeks]

Laboratory Parameter (Hematology): Reticulocyte Count [Time Frame: Up to 24 weeks]

Area Under the Curve at Steady State (AUCss) of Sotatercept [Time Frame: 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]

Area Under the Curve from 0 to 3 weeks (AUC0-3 weeks) of Sotatercept [Time Frame: Predose Day 1, Day 7, Day 14, and Predose Day 21]

Titer of Anti-drug Antibody (ADA) to Sotatercept [Time Frame: Up to 24 weeks]

Percentage of Participants Who Discontinue Study Drug Due to an AE [Time Frame: Up to 24 weeks]

Laboratory Parameter (Hematology): Platelet Count [Time Frame: Up to 24 weeks]

Percentage of Participants Who Experience at Least 1 Adverse Event (AE) [Time Frame: Up to 24 weeks]

Laboratory Parameter (Hematology): Hematocrit [Time Frame: Up to 24 weeks]

Serum Trough Concentration (Ctrough) of Sotatercept [Time Frame: 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]

Secondary Outcome(s)

Percentage of Participants Who Either Improved or Maintained Their World Health Organization Functional Class (WHO FC) [Time Frame: Baseline and Week 24]

Mean Change from Baseline in 6-Minute Walk Distance (6MWD) (Cohorts 1 and 2) [Time Frame: Baseline and Week 24]

Mean Change from Baseline in N-terminal Prohormone B-type Natriuretic Peptide (NT-proBNP) [Time Frame: Baseline and Week 24]

Mean Change from Baseline in Right Ventricular Fractional Area Change (RVFAC) [Time Frame: Baseline and Week 24]

Mean Change from Baseline on Cardiac Output (Cohorts 1 and 2) [Time Frame: Baseline and Week 24]

Mean Change from Baseline in Tricuspid Annular Plane Systolic Excursion (TAPSE) [Time Frame: Baseline and Week 24]

Mean Change from Baseline in Eccentricity Index [Time Frame: Baseline and Week 24]

Mean Change from Baseline in Pulmonary Arterial Pressure (PAP) (Cohorts 1 and 2) [Time Frame: Baseline and Week 24]

Mean Change from Baseline in Pediatric Quality of Life (PedsQL) Generic Score [Time Frame: Baseline and Week 24]

Mean Change from Baseline in Pulmonary Artery Systolic Pressure (PASP) [Time Frame: Baseline and Week 24]

Mean Change from Baseline in Right Ventricular (RV) Function (Cohorts 1 and 2) [Time Frame: Baseline and Week 24]

Secondary ID(s)

7962-008

MK-7962-008

U1111-1290-2858

2022-000478-25

2023-504861-22

Source(s) of Monetary Support

Please refer to primary and secondary sponsors