FUTURE 3 Study Extension

Study Overview
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Brief Summary
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The objectives of the FUTURE 3 Study Extension are to evaluate the long-term safety, tolerability and efficacy of the pediatric formulation of bosentan two versus three times a day in children with Pulmonary Arterial Hypertension (PAH).

Official Title
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A Prospective, Multicenter, Open-label Extension of FUTURE 3 to Assess the Safety, Tolerability and Efficacy of the Pediatric Formulation of Bosentan Two Versus Three Times a Day in Children With Pulmonary Arterial Hypertension

Conditions
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Pulmonary Arterial Hypertension

Intervention / Treatment
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* Drug: Bosentan


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients who completed the FUTURE 3 core study (AC-052-373) or prematurely discontinued due to PAH-progression, if bosentan was not permanently discontinued Patients who tolerated bosentan pediatric formulation and for whom bosentan is considered beneficial at the end of the FUTURE 3 core study (AC-052-373) Signed informed consent by the parents or the legal representatives prior to any study-mandated procedure. Exclusion Criteria: Known intolerance or hypersensitivity to bosentan or any of the excipients of the dispersible bosentan tablet Any clinically significant laboratory abnormality that precludes continuation of bosentan therapy Pregnancy AST and/or ALT values > 3 times the upper limit of normal range (ULN) Moderate to severe hepatic impairment, i.e., Child-Pugh Class B or C Premature and permanent study drug discontinuation during the FUTURE 3 core study (AC-052-373) Any major violation of the FUTURE 3 core study (AC-052-373) protocol.

Ages Eligible for Study
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Minimum Age: 3 Months
Maximum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: bosentan 2mg/kg b.i.d.<br>Patients who received 2 mg/kg bosentan twcie daily (b.i.d.) during the FUTURE 3 core study and continued with the same dose regimen during the extension study | Drug: Bosentan<br>* Oral dispersible tablet administered as 2mg/kg two (b.i.d.) or three (t.i.d.) times per day<br>* Other names: ACT-050088;|
| Experimental: bosentan 2mg/kg t.i.d.<br>Patients who received 2 mg/kg bosentan 3 times a day (t.i.d.) during the FUTURE 3 core study and continued with the same dose regimen during the extension study | Drug: Bosentan<br>* Oral dispersible tablet administered as 2mg/kg two (b.i.d.) or three (t.i.d.) times per day<br>* Other names: ACT-050088;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Treatment Emergent Adverse Events (AEs) up to 7 Days After Permanent Study Drug Discontinuation | This is the total number of subjects with at least one adverse event (serious or not serious) whether or not causally related to the study drug and presented cumulatively in the FUTURE 3 and FUTURE 3 Extension study. NOTE: FUTURE 3 extension study was exploratory and no primary efficacy and safety endpoints were defined in the protocol. So, this safety outcome measure was selected and reported as primary endpoint here. | Up to 62 weeks in average | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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pediatric, pulmonary arterial hypertension, bosentan, Tracleer