Bosentan in Children With Pulmonary Arterial Hypertension Extension Study (FUTURE 2)

May 16, 2017 updated by: Actelion

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

33

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Clamart, France, 92140
        • Hôpital Antoine Béclère
      • Paris, France, 75743
        • Hôpital Necker
      • Toulouse, France
        • CHE de Toulouse Hopital d'Enfants
  • Germany
      • Berlin, Germany
        • Deutsches Herzzentrum
      • Giessen, Germany
        • Universitats Kinderklinik
  • Italy
      • Bologna, Italy, 40138
        • Policlinico S. Orsola-Malpighi
  • Netherlands
      • Groningen, Netherlands
        • Beatrix Children's Hospital
  • Switzerland
      • Geneva, Switzerland
        • Hôpital des Enfants
  • United Kingdom
      • London, United Kingdom
        • The Institute of Child Health
  • United States
    • Colorado
      • Denver, Colorado, United States, 80218
        • The Children's Hospital Cardiac Care Center
    • New York
      • New York, New York, United States, 10032
        • Columbia University Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 11 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Bosentan
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.
Drug: Bosentan
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
Other Names:
  • Tracleer
  • Ro 47-0203

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline to End of Study (EOS) in Height for Age.
Time Frame: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average

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
From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average
Change From Baseline to End of Study (EOS) in Body Weight
Time Frame: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average
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.
From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average
Change From Baseline to End of Study (EOS) in Systolic Blood Pressure (SBP)
Time Frame: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average
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.
From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average
Change From Baseline to End of Study (EOS) in Diastolic Blood Pressure (DBP)
Time Frame: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average
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.
From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average
Change From Baseline to End of Study (EOS) in Pulse Rate
Time Frame: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average
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.
From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation), i.e. 32 months in average
Proportion of Patients With Treatment-emergent Liver Function Abnormalities
Time Frame: After baseline, up to 1 calendar day after study treatment discontinuation in FUTURE 1 or FUTURE 2, i.e. 31 months in average

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.
After baseline, up to 1 calendar day after study treatment discontinuation in FUTURE 1 or FUTURE 2, i.e. 31 months in average
Proportion of Patients With Treatment-emergent Hemoglobin Abnormalities
Time Frame: After baseline, up to 1 calendar day after study treatment discontinuation in FUTURE 1 or FUTURE 2, i.e. 31 months in average

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.
After baseline, up to 1 calendar day after study treatment discontinuation in FUTURE 1 or FUTURE 2, i.e. 31 months in average
Number of Subjects With Adverse Events Leading to Premature Discontinuation of Study Treatment
Time Frame: From the first study drug administration in FUTURE 1, for an average of 31 months
From the first study drug administration in FUTURE 1, for an average of 31 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Actelion

Investigators

  • Study Director: Andjela Kusic-Pajic, MD, Actelion

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 23, 2005

Primary Completion (Actual)

October 28, 2011

Study Completion (Actual)

October 28, 2011

Study Registration Dates

First Submitted

April 26, 2006

First Submitted That Met QC Criteria

April 26, 2006

First Posted (Estimate)

April 27, 2006

Study Record Updates

Last Update Posted (Actual)

June 14, 2017

Last Update Submitted That Met QC Criteria

May 16, 2017

Last Verified

May 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AC-052-367
  • 2005-001967-70 (EudraCT Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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