EUCTR2022-002754-74-DE
Active, not recruiting
Phase 1
A multicenter, open-label, single-arm study to assess the pharmacokinetics and safety of macitentan in children aged 1 month to <2 years with pulmonary arterial hypertension
ACTELION Pharmacteuticals Ltd.0 sites10 target enrollmentNovember 10, 2022
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Not specified
- Sponsor
- ACTELION Pharmacteuticals Ltd.
- Enrollment
- 10
- Status
- Active, not recruiting
- Last Updated
- 2 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •1\. Pediatric participants aged 1 month to \<2 years (at Screening).
- •Type of Participant and Disease Characteristic
- •2\. Pulmonary arterial hypertension including participants with Down syndrome. Diagnosis must have been confirmed by (historical, any time before screening) right heart catheterization mPAP \=25 mmHg, pulmonary arterial wedge pressure (PAWP) \=15 mmHg, pulmonary vascular resistance index \>3Wood units × m2\) 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 \<1\.5 and saturation of peripheral oxygen \=90% measured by pulse oximetry at room air), or
- •ii. Inoperable open left\-to\-right shunts (with a PVR \>8 WU and Qp/Qs \<2\), or
- •iii. Co\-incidental shunt (ie, not explaining hemodynamically the presence of PAH), or
- •iv. Post\-operative PAH (persisting/recurring/developing \=6 months after repair of shunt), or
Exclusion Criteria
- •Medical Conditions
- •1\. PAH due to portal hypertension, schistosomiasis, pulmonary veno\-occlusive disease and/or pulmonary capillary hemangiomatosis.
- •2\. Persistent pulmonary hypertension of the newborn.
- •3\. 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.
- •4\. Pulmonary hypertension due to lung disease.
- •5\. Known diagnosis of bronchopulmonary dysplasia.
- •6\. Pulmonary vein stenosis.
- •7\. Known concomitant life\-threatening disease with life expectancy \<12 months.
Outcomes
Primary Outcomes
Not specified
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