Pulmonary arterial hypertension in children
Conditions
Interventions
Bosentan dispersible tablet (32 mg) in the dosage of 2 mg/kg b.i.d. or 2 mg/kg t.i.d.
Sponsors
Actelion Pharmaceuticals
Gewerbestrasse 16
CH-4123 Allschwil
Switzerland
Eligibility
Inclusion criteria
Inclusion criteria: 1. Patients who completed the FUTURE 3 core study (AC-052-373) or prematurely discontinued due to PAH progression, if bosentan was not permanently discontinued; 2. Patients who tolerated bosentan pediatric formulation and for whom bosentan is considered beneficial by the investigator at the end of FUTURE 3 core study (AC-052-373); 3. Signed informed consent by the parents or the legal representatives prior to any study-mandated procedure.
Exclusion criteria
Exclusion criteria: 1. Known intolerance or hypersensitivity to bosentan or any of the excipients of the dispersible bosentan tablet; 2. Any clinically significant laboratory abnormality that precludes continuation of bosentan therapy; 3. Pregnancy; 4. AST and/or ALT values > 3 times the upper limit of normal range (ULN); 5. Moderate to severe hepatic impairment, i.e., Child-Pugh Class B or C; 6. Premature and permanent study drug discontinuation during the FUTURE 3 core study (AC-052-373); 7. Any major violation of the FUTURE 3 core study (AC 052 373) protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| No primary endpoint was defined in this study. | — |
Secondary
| Measure | Time frame |
|---|---|
| Exploratory efficacy endpoints: 1. Change from baseline to end of study in WHO functional class; 2. Time to death, lung transplantation or hospitalization for PAH-Progression; 3. Time to death, lung transplantation, hospitalization for PAH-progression or initiation of new therapy for PAH or new/worsening right heart failure; 4. Changes from baseline to 3, 6, 9, 12, 15 and 18 months of treatment over AC 052 373 and AC-052-374 in Global Clinical Impression scale assessed by the physician and parents. Safety and tolerability endpoints: 1. Treatment-emergent AEs and serious adverse events (SAEs) up to 7 days after permanent discontinuation of study drug; 2. Adverse events leading to premature discontinuation of study drug; 3. Serious adverse events from 7 up to 60 days after permanent discontinuation of study drug; 4. Changes from baseline to end of study in vital signs, body weight, and height/length; 5. Treatment-emergent marked laboratory abnormalities up to end of study. | — |
Contacts
Public ContactPeter Westerveld
Beneluxbaan 2B
Outcome results
None listed