Bosentan in Children With Pulmonary Arterial Hypertension Extension Study - Clinical Trial for Pulmonary Arterial Hypertension | NCT00319020

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Primary Purpose

Status

Completed

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

Bosentan

About this trial

This is an interventional treatment trial for Pulmonary Arterial Hypertension focused on measuring bosentan, pulmonary arterial hypertension, children, FUTURE 1

Eligibility Criteria

2 Years - 11 Years (Child)All SexesDoes not accept healthy volunteers

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.

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Bosentan

Arm Description

Bosentan was administered at 4 mg/kg twice daily (b.i.d.) until the end of the study. It could be down-titrated to 2 mg/kg b.i.d. if not well tolerated.

Outcomes

Primary Outcome Measures

Change From Baseline to End of Study (EOS) in Height for Age.

In order to compare the growth data with those of healthy children, growth curves are calculated from height data collected throughout the follow-up period. For each patient, height measured at each study visit was converted to a z-score and expressed in standard deviations (SD) from WHO growth standards. The Z-score was calculated according to the following formula: Z-score = (observed value of the study participant - median value of the reference population) / SD value of the reference population

Change From Baseline to End of Study (EOS) in Body Weight

The main study objective was to assess the long-term safety and tolerability of bosentan in children with PAH, including growth as measured by changes from baseline in body weight and height.

Change From Baseline to End of Study (EOS) in Systolic Blood Pressure (SBP)

The main study objective was to assess the long-term safety and tolerability of bosentan in children with PAH, including changes from baseline in blood pressure.

Change From Baseline to End of Study (EOS) in Diastolic Blood Pressure (DBP)

The main study objective was to assess the long-term safety and tolerability of bosentan in children with PAH, including changes from baseline in blood pressure.

Change From Baseline to End of Study (EOS) in Pulse Rate

The main study objective was to assess the long-term safety and tolerability of bosentan in children with PAH, including changes from baseline in pulse rate.

Proportion of Patients With Treatment-emergent Liver Function Abnormalities

The main study objective was to assess the long-term safety and tolerability of bosentan in children with PAH, including laboratory abnormalities related to liver enzymes. Proportion of patients with increase in alanine aminotransferase (ALT) or aspartate aminotransferase (AST) above 3 times upper limit of normal (ULN) is reported here.

Proportion of Patients With Treatment-emergent Hemoglobin Abnormalities

The main study objective was to assess the long-term safety and tolerability of bosentan in children with PAH, including hemoglobin abnormalities. Proportion of patients with marked hemoglobin decreases (i.e., decrease of or above 15% of the lower normal limit (LL)) is reported here.

Number of Subjects With Adverse Events Leading to Premature Discontinuation of Study Treatment

Secondary Outcome Measures

Full Information

NCT ID

NCT00319020

First Posted

April 26, 2006

Last Updated

May 16, 2017

Sponsor

Actelion

1. Study Identification

Unique Protocol Identification Number

NCT00319020

Brief Title

Bosentan in Children With Pulmonary Arterial Hypertension Extension Study

Acronym

FUTURE 2

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

2. Study Status

Record Verification Date

May 2017

Overall Recruitment Status

Completed

Study Start Date

August 23, 2005 (Actual)

Primary Completion Date

October 28, 2011 (Actual)

Study Completion Date

October 28, 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Actelion

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Pulmonary Arterial Hypertension

Keywords

bosentan, pulmonary arterial hypertension, children, FUTURE 1

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

33 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Bosentan

Arm Type

Experimental

Arm Description

Bosentan was administered at 4 mg/kg twice daily (b.i.d.) until the end of the study. It could be down-titrated to 2 mg/kg b.i.d. if not well tolerated.

Intervention Type

Drug

Intervention Name(s)

Bosentan

Other Intervention Name(s)

Ro 47-0203, Tracleer

Intervention Description

32-mg dispersible and breakable tablet. The body weight-adjusted dose of the dispersible tablet was dispersed in a teaspoon of water (not mixed with food) before being administered orally

Primary Outcome Measure Information:

Title

Change From Baseline to End of Study (EOS) in Height for Age.

Description

In order to compare the growth data with those of healthy children, growth curves are calculated from height data collected throughout the follow-up period. For each patient, height measured at each study visit was converted to a z-score and expressed in standard deviations (SD) from WHO growth standards. The Z-score was calculated according to the following formula: Z-score = (observed value of the study participant - median value of the reference population) / SD value of the reference population

Time Frame