The purpose of this study is to learn what happens to macitentan and its active metabolite (aprocitentan) in the body of children aged between 1 month and 2 years.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
BASIC_SCIENCE
Masking
NONE
Macitentan will be administered orally.
Universitatsklinikum Freiburg
Freiburg im Breisgau, Germany
Universitatsmedizin Gottingen
Göttingen, Germany
Instytut Pomnik Centrum Zdrowia Dziecka
Warsaw, Poland
Wojewodzki Szpital Specjalistyczny We Wroclawiu
Wroclaw, Poland
Trough Concentration of Macitentan and its Active Metabolite Aprocitentan at Week 4 in Steady-State
Trough concentration of macitentan and its active metabolite aprocitentan at week 4 in steady-state will be reported.
Time frame: Predose (at Week 4)
Number of Participants with Adverse Events (AEs)
An AE is any untoward medical occurrence in a clinical study participant administered a pharmaceutical (investigational or non-investigational) product. An AE does not necessarily have a causal relationship with the intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal finding), symptom, or disease temporally associated with the use of a medicinal (investigational or non-investigational) product, whether or not related to that medicinal (investigational or non-investigational) product.
Time frame: Up to 1.5 years
Number of Participants with Serious Adverse Events (SAEs)
A SAE is any AE that results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect and is a suspected transmission of any infectious agent via a medicinal product, is medically important.
Time frame: Up to 1.5 years
Number of Participants with AEs Leading to Premature Discontinuation of Macitentan
Number of participants with AEs Leading to premature discontinuation of macitentan will be reported.
Time frame: Up to 1.5 years
Number of Participants with Adverse Event of Special Interests (AESIs)
Number of participants with AESI will be reported. AEs considered to be of special interest are as hypotension, edema/fluid retention, hemoglobin decrease/anemia, liver events.
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Time frame: Up to 1.5 years
Number of Participants with Clinical Laboratory Abnormalities
Number of participants with clinical laboratory abnormalities (serum, chemistry and hematology) will be reported.
Time frame: Up to 1.5 years
Number of Participants with Change from Baseline in Clinical laboratory Values.
Number of participants with change from baseline in clinical laboratory values will be reported.
Time frame: Up to 1.5 years
Change from Baseline in Blood Pressure
Change from baseline in blood pressure will be reported.
Time frame: Up to 1.5 years
Change From Baseline in Heart Rate
Change from baseline in heart rate will be reported.
Time frame: Up to 1.5 years
Change From Baseline in Body Weight
Change from baseline in body weight will be reported.
Time frame: Up to 1.5 years
Change From Baseline in Length
Change from baseline in length will be reported.
Time frame: Up to 1.5 years
Change from Baseline in Height
Change from baseline in height will be reported.
Time frame: Up to 1.5 years
Plasma Concentration of Macitentan and its Active Metabolite (Aprocitentan) for Macitentan Naive Participants
Plasma concentrations of macitentan and its active metabolite (aprocitentan) after the first dose of macitentan for macitentan naive participants will be reported.
Time frame: At 2, 5, and 24 hours after the first dose of macitentan on Day 1
Trough Concentrations of Macitentan and its Active Metabolite Aprocitentan at Week 8 in Steady-State Conditions
Trough concentrations of macitentan and its active metabolite aprocitentan at week 8 in steady-state conditions will be reported.
Time frame: Predose (at Week 8)