Pulmonary arterial hypertension (PAH) in children MedDRA version: 14.1 Level: LLT Classification code 10064908 Term: Associated with (APAH) System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders MedDRA version: 14.1 Level: LLT Classification code 10064909 Term: Idiopathic (IPAH) System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders MedDRA version: 14.1 Level: LLT Classification code 10064910 Term: Familial (FPAH) System Organ Class: 10038738 -
Conditions
Sponsors
Actelion pharmaceuticals Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Signed informed consent by the parents or legal representatives prior to any study mandated procedure. 2. Male or female = 1 year and 3 Wood units for idiopathic, heritable PAH or PAH persisting/recurrent after complete repaired of congenital heart defect. or PVR > 8 Wood units for PAH associated with systemic-to-pulmonary open shunts with a ratio Qp/Qs =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Non-stable disease status, e.g., history of recurrent (near-) syncope or signs and symptoms of non-compensated right heart failure. 2. Patients with bronchopulmonary dysplasia, congenital diaphragmatic hernia or other chronic lung diseases. 3. Need or plan to wean patient from any PAH-specific therapies during the study. 4. Systolic blood pressure 1.5 times the upper limit of normal range. 6. Moderate or severe hepatic impairment (i.e., Child-Pugh Class B or C). 7. Hemoglobin and/or hematocrit levels < 75% of the lower limit of normal range. 8. Known intolerance or hypersensitivity to bosentan or any of the excipients of the bosentan dispersible tablet. 9. Treatment with bosentan or any other endothelin receptor antagonists within 4 months prior to randomization (including any ERA investigational drug). 10. Treatment with another investigational drug within 1 month prior to randomization or planned treatment. 11. Pregnancy or breastfeeding. 12. Any condition that prevents compliance with the protocol or adherence to therapy. 13. Administration of prohibited medication within 2 weeks or 5 times the half-life, prior to randomization, whichever is the longest.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective is to demonstrate that 12 weeks of treatment with either dose (0.5 mg/kg b.i.d or 2 mg/kg b.i.d) of the bosentan dispersible tablet formulation improves PVRi in pediatric patients with PAH, aged from = 1 year to < 17 years.;Secondary Objective: The other objectives are to evaluate the following in this patient population: - Other hemodynamic variables, WHO functional class, global clinical status, exercise capacity, time to clinical worsening. - Tolerability and safety variables. - Echocardiographic variables (imaging and Doppler evaluation). - The steady-state PK of the bosentan dispersible tablet formulation at doses of 0.5 mg/kg b.i.d. and 2 mg/kg b.i.d. - The PK-efficacy relationship on PVRi and other selected efficacy endpoints.;Primary end point(s): Primary efficacy endpoint = PVRi at Week 12 expressed as percent change from the baseline value. ‘baseline’ is defined as the last assessment performed prior to first study drug intake.;Timepoint(s) of evaluation of this end point: See above | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary efficacy endpoints = Change from baseline to Week 12 in RHC variables.;Timepoint(s) of evaluation of this end point: See above | — |
Countries
Argentina, Bulgaria, Chile, China, Colombia, Croatia, France, Guatemala, India, Mexico, Netherlands, Peru, Philippines, Portugal, Russian Federation, Serbia, South Africa, Ukraine, United States, Vietnam
Contacts
Public ContactGLOBAL MEDICAL INFORMATION
Actelion Pharmaceuticals Ltd.
Outcome results
None listed