Clinical trial NCT00319020: Bosentan in Children With Pulmonary Arterial Hypertension Extension Study
Official title: An Open Label, Long-term, Safety, and Tolerability Extension Study Using the Pediatric Formulation of Bosentan in the Treatment of Children With Idiopathic or Familial Pulmonary Arterial Hypertension Who Completed FUTURE 1
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Pulmonary Arterial Hypertension
Keywords: bosentan
pulmonary arterial hypertension
children
FUTURE 1
Lead sponsor: Actelion
Sponsor class: INDUSTRY
First posted: 2006-04-27
Enrollment: 33
Sex: ALL
Minimum age (years): 2
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The main objective of the FUTURE 2 study was to assess the long-term safety and tolerability of the pediatric formulation of bosentan in children with idiopathic pulmonary arterial hypertension or familial pulmonary arterial hypertension who completed FUTURE 1 study.


Eligibility 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.
* Males or females \>= 2 and \< 12 years of age at enrollment in FUTURE 2 (this study). Females who are menstruating must have a negative pregnancy test. A reliable method of contraception must be considered, if appropriate.

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.

Primary outcomes:
measure: Change From Baseline to End of Study (EOS) in Height for Age.
timeFrame: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average
measure: Change From Baseline to End of Study (EOS) in Body Weight
timeFrame: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average
measure: Change From Baseline to End of Study (EOS) in Systolic Blood Pressure (SBP)
timeFrame: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average
measure: Change From Baseline to End of Study (EOS) in Diastolic Blood Pressure (DBP)
timeFrame: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average
measure: Change From Baseline to End of Study (EOS) in Pulse Rate
timeFrame: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average
measure: Proportion of Patients With Treatment-emergent Liver Function Abnormalities
timeFrame: After baseline, up to 1 calendar day after study treatment discontinuation in FUTURE 1 or FUTURE 2, i.e. 31 months in average
measure: Proportion of Patients With Treatment-emergent Hemoglobin Abnormalities
timeFrame: After baseline, up to 1 calendar day after study treatment discontinuation in FUTURE 1 or FUTURE 2, i.e. 31 months in average
measure: Number of Subjects With Adverse Events Leading to Premature Discontinuation of Study Treatment
timeFrame: From the first study drug administration in FUTURE 1, for an average of 31 months
Secondary outcomes:
Other outcomes:
Locations:
Study officials:
affiliation: Actelion
name: Andjela Kusic-Pajic, MD
role: STUDY_DIRECTOR
References:
citation: Berger RM, Haworth SG, Bonnet D, Dulac Y, Fraisse A, Galie N, Ivy DD, Jais X, Miera O, Rosenzweig EB, Efficace M, Kusic-Pajic A, Beghetti M. FUTURE-2: Results from an open-label, long-term safety and tolerability extension study using the pediatric FormUlation of bosenTan in pUlmonary arterial hypeRtEnsion. Int J Cardiol. 2016 Jan 1;202:52-8. doi: 10.1016/j.ijcard.2015.08.080. Epub 2015 Aug 9.