The primary objectives of the study are to evaluate the safety and tolerability, and pharmacokinetics (PK) of sotatercept over 24 weeks of treatment in children ≥1 to \<18 years of age with PAH World Health Organization (WHO) Group 1 on standard of care (SoC). There is no formal hypothesis.
Idiopathic pulmonary arterial hypertension (IPAH)
Heritable PAH
Drug/toxin-induced PAH
PAH associated with connective tissue disease
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
Single group
Sotatercept
DrugSC injection every 3 weeks (Q3W) of 0.3 mg/kg. Dosage may be adjusted based on protocol-specific guidelines.
Serum Trough Concentration (Ctrough) of Sotatercept
Ctrough was the lowest concentration of Sotatercept in serum just before the next dose. Blood samples will be collected at multiple time points to estimate the Ctrough of Sotatercept.
Area Under the Curve at Steady State (AUCss) of Sotatercept
Blood samples will be collected at multiple time points to estimate the AUCss of Sotatercept.
Area Under the Curve from 0 to 3 weeks (AUC0-3 weeks) of Sotatercept
Blood samples will be collected at Predose Day 1, Day 7, Day 14, and Predose Day 21 to estimate the AUC0-3 weeks of Sotatercept.
Percentage of Participants Who Experience at Least 1 Adverse Event (AE)
An AE is any unfavourable and unintended sign, symptom, or disease temporally associated with the use of a medicinal product or protocol-specified procedure, whether or not considered related to the medicinal product or protocol-specified procedure. Any worsening of a preexisting condition considered related to the medicinal product or protocol-specified procedure. Any worsening of a preexisting condition that is temporally associated with the use of the Sponsor's product, is also an AE. The percentage of participants with 1 or more AEs will be assessed.
Mean Change from Baseline in 6-Minute Walk Distance (6MWD) (Cohorts 1 and 2)
6MWD will be assessed using the 6-minute walk test (6MWT).
Mean Change from Baseline in Tricuspid Annular Plane Systolic Excursion (TAPSE)
A two-dimensional echocardiogram (ECHO) will be performed with the results interpreted by a blinded independent central review (BICR) at baseline and after 24 weeks of treatment. The change from baseline in TAPSE will be reported.
Mean Change from Baseline in Pulmonary Artery Systolic Pressure (PASP)
A two-dimensional ECHO will be performed with the results interpreted by a BICR at baseline and after 24 weeks of treatment. The change from baseline in PASP will be reported.
Mean Change from Baseline in Right Ventricular Fractional Area Change (RVFAC)
A two-dimensional ECHO will be performed with the results interpreted by a BICR at baseline and after 24 weeks of treatment. The change from baseline in RVFAC will be reported.
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