Pulmonary Arterial Hypertension MedDRA version: 21.1 Level: PT Classification code 10064911 Term: Pulmonary arterial hypertension System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders
Conditions
Interventions
Product Name: Macitentan
Product Code: JNJ-67896062/ACT-064922
Pharmaceutical Form: Dispersible tablet
INN or Proposed INN: Macitentan
CAS Number: 441798-33-0
Current Sponsor code: JNJ-67896062
Other
Sponsors
ACTELION Pharmacteuticals Ltd.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Age 1. Pediatric participants aged 1 month to 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 8 WU and Qp/Qs =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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 3 ×ULN. 9. Severe hepatic impairment, eg, Child-Pugh Class C. 10. Hemoglobin or hematocrit 221 µmol/L). 13. Known allergies, hypersensitivity, or intolerance to ERAs or macitentan or its excipients. 14. Known hereditary fructose intolerance. Prior/Concomitant Therapy 15. Triple combination therapy with PAH-specific treatments. 16. Treatment with intravenous or subcutaneous prostanoids within 4 weeks before Visit 2 (Day 1) unless given for vasoreactive testing. 17. Any PAH-related surgical intervention planned, or participants listed for organ transplantation related to PAH. 18. Treatment with strong inducers of CYP3A4 such as rifabutin, rifampicin, rifapentin, carbamazepine, phenobarbital, phenytoin, St. John’s wort (hypericum perforatum), within 4 weeks prior to Visit 2 (Day 1). 19. Systemic treatment with strong inhibitors of CYP3A4 such as boceprevir, clarithromycin, conivaptan, indinavir, itraconazole, ketoconazole, nefazodone, nelfinavir, posaconazole, ritonavir, saquinavir, telaprevir, telithromycin, and voriconazole within 4 weeks prior to Visit 2 (Day 1). 20. Systemic treatment with moderate dual CYP3A4/CYP2C9 inhibitor (eg, fluconazole and amiodarone), or administration of a combination of a moderate CYP3A4 (eg, ciprofloxacin, cyclosporine, diltiazem, erythromycin, verapamil) together with a moderate CYP2C9 inhibitor (eg, miconazole, piperine) within 4 weeks prior to Visit 2 (Day 1). 21. Switching PAH treatment from an ERA (eg, bosentan, ambrisentan) to macitentan when a participant’s clinical condition is unstable or undergoing continuous deterioration. Prior/Concurrent Clinical Study Experience 22. Received an investigational intervention (including investigational vaccines) or used an invasive investigational medical device within 4 weeks prior to Visit 2 (Day 1) or is currently enrolled in an investigational study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The Primary Objective is to evaluate the pharmacokinetics of macitentan and its active metabolite (aprocitentan) in children aged 1 month to <2 years.;Secondary Objective: The Secondary Objectives are: - To evaluate the safety and tolerability of macitentan in children aged 1 month to <2 years. Overall safety will be assessed. - To evaluate the PK of macitentan and its active metabolite (aprocitentan) in children aged 1 month to <2 years.;Primary end point(s): Primary endpoint is trough concentrations of macitentan and its active metabolite (aprocitentan) at Week 4 (steady-state).;Timepoint(s) of evaluation of this end point: Week 4 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary end points are: 1. Adverse events and serious adverse events. 2. Adverse events leading to premature discontinuation of macitentan. 3. Adverse events of special interest. 4. Marked laboratory abnormalities. 5. Change from baseline in selected laboratory parameters to all scheduled assessment timepoints. 6. Change from baseline in vital signs (blood pressure, heart rate) to all scheduled assessment timepoints. 7. Growth (body weight and length/height) from baseline to all scheduled assessment timepoints. 8. Concentrations of macitentan and its active metabolite (aprocitentan) at 2, 5, and 24 hours after the first dose of macitentan for macitentan naive participants. 9. Trough concentrations of macitentan and its active metabolite (aprocitentan) at Week 8 (steady-state conditions).;Timepoint(s) of evaluation of this end point: 1-7. Throughout the study. 8. 2, 5, and 24 hours after the first dose of macitentan 9. Week 8 | — |
Countries
Germany, Poland
Contacts
Public ContactClinical Registry Group
Janssen-Cilag International NV
Outcome results
None listed