Overview
A Study of Macitentan in Children Below 2 Years of Age
Status:
Recruiting
Recruiting
Trial end date:
2024-09-30
2024-09-30
Target enrollment:
0
0
Participant gender:
All
All
Summary
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.Phase:
Phase 1Accepts Healthy Volunteers?
NoDetails
Lead Sponsor:
ActelionTreatments:
Macitentan
Criteria
Inclusion Criteria:- Pulmonary arterial hypertension (PAH): 1) including participants with Down syndrome.
Diagnosis must have been confirmed by (historical, any time before screening) right
heart catheterization mean pulmonary arterial pressure (mPAP) greater than or equal to
(>=) 25 millimeter of mercury (mmHg), pulmonary arterial wedge pressure (PAWP) less
than or equal to (=<)15 mmHg, pulmonary vascular resistance index greater than (>) 3
Wood units * meter square (m^2) where in the absence of pulmonary vein obstruction
and/or significant lung disease PAWP can be replaced left atrium pressure or left
ventricular end diastolic pressure (in the absence of mitral stenosis) assessed by
heart catheterization. a) Idiopathic PAH, or b) Heritable PAH, or c) PAH associated
with congenital heart disease: i) Eisenmenger syndrome (Qp/Qs less than (<) 1.5 and
saturation of peripheral oxygen ≤ 90 percent (%) measured by pulse oximetry at room
air), or ii) Inoperable open left-to-right shunts (with a Pulmonary vascular
resistance [PVR] > 8 WU and Qp/Qs <2), or iii) Co-incidental shunt (that is, not
explaining hemodynamically the presence of PAH), or iv) Post-operative PAH
(persisting/recurring/developing ≥ 6 months after repair of shunt), or d) Drug or
toxin induced PAH, or e) PAH associated with Human immunodeficiency viruses (HIV)
- World Health Organization Functional Class (WHO FC) I, II, or III
- PAH-specific treatment-naive participants or participants on PAH specific monotherapy
or combination of 2 therapies. Use of macitentan before or during screening is allowed
- Body weight of greater than or equal to (>=) 3.5 kilogram (kg)
- Parent(s) (preferably both if available or as per local requirements) or participant's
legally designated representative must sign an informed consent form (ICF) indicating
that they understand the purpose of, and procedures required for, the study and is/are
willing to allow the child to participate in the study
Exclusion Criteria:
- PAH due to portal hypertension, schistosomiasis, pulmonary veno-occlusive disease
and/or pulmonary capillary hemangiomatosis
- Persistent pulmonary hypertension of the newborn
- The following congenital cardiac abnormalities: a) Cyanotic congenital cardiac lesions
such as transposition of the great arteries, truncus arteriosus, pulmonary atresia
with ventricular septal defect, unless operatively repaired and with no residual
shunt. b) Univentricular heart and/or participants with Fontan-palliation
- Pulmonary hypertension due to lung disease
- Known diagnosis of bronchopulmonary dysplasia