Idiopathic or familial pulmonary arterial hypertension
Conditions
Interventions
Trade Name: Tracleer
Product Name: bosentan
Product Code: Ro 47-0203
Pharmaceutical Form: Tablet
INN or Proposed INN: bosentan monohydra
Sponsors
Actelion Pharmaceuticals Ltd.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: · Signed informed consent by the parents or the legal representatives. · Patients who completed the FUTURE 1 study. · Patients who tolerated bosentan pediatric formulation and for whom bosentan is considered beneficial at the end of FUTURE 1. · Male or female >= 2 and =65 years) F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: · Intolerance to bosentan despite dose reductions. · Any clinically significant laboratory abnormality that precludes continuation of bosentan therapy. · Pregnancy or breast-feeding. · Known hypersensitivity to bosentan or any of the excipients. · Premature and permanent study drug discontinuation during FUTURE 1.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the long-term safety and tolerability of the pediatric formulation of bosentan in children with IPAH or familial PAH. The primary endpoint analysis will be the analysis of tolerability and safety endpoints: · Treatment-emergent adverse events up to 24 hours after permanent discontinuation of study drug. · Adverse events leading to premature discontinuation of study drug. · Serious adverse events up to 28 days after permanent discontinuation of study drug. · Changes from baseline to Study End in vital signs, body weight, and height. · Treatment-emergent marked laboratory abnormalities. ; Secondary Objective: Exploratory efficacy endpoints: · Change from Baseline in FUTURE 1 to Study End or Premature study drug discontinuation (FUTURE 1 or 2) in: - WHO functional class - Quality of life questionnaire (SF-10 for childrenTM) - Global Clinical Impression scale according to the parents/legal representatives - Global Clinical Impression scale according to the physician · From Baseline in FUTURE 1, time to worsening of PAH, defined as death or transplantation or hospitalization for PAH worsening. · From Baseline in FUTURE 1, time to worsening of PAH or initiation of new therapy for PAH or new right heart failure or worsening of right heart failure. ;Primary end point(s): In addition to long-term tolerability and safety information, the FUTURE 2 study will provide an opportunity to assess quality of life and functional status of the paediatric population using the new formulation. | — |
Countries
Germany, Italy, United Kingdom
Outcome results
None listed