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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Actelion
ClinicalTrials.gov Identifier:
NCT00319020
First received: April 26, 2006
Last updated: June 2, 2016
Last verified: June 2016
  Purpose
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.

Condition Intervention Phase
Pulmonary Arterial Hypertension
Drug: Bosentan
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Actelion:

Primary Outcome Measures:
  • 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) ] [ Designated as safety issue: Yes ]
    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) [ Time Frame: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation) ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation) ] [ Designated as safety issue: Yes ]
    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.

  • 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) ] [ Designated as safety issue: Yes ]
    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 height for age. [ Time Frame: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation) ] [ Designated as safety issue: Yes ]

    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.

    For each patient, height was put in the perspective of the height of healthy children of the same age according to the WHO growth standards.


  • 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 ] [ Designated as safety issue: Yes ]

    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 x ULN (upper limit of normal) is reported here.


  • 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 ] [ Designated as safety issue: Yes ]

    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 [ Time Frame: From the first study drug administration in FUTURE 1 ] [ Designated as safety issue: Yes ]

Enrollment: 33
Study Start Date: August 2005
Study Completion Date: October 2011
Primary Completion Date: October 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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:
  • Ro 47-0203
  • Tracleer

  Eligibility

Ages Eligible for Study:   2 Years to 11 Years   (Child)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00319020

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

Publications:
Responsible Party: Actelion
ClinicalTrials.gov Identifier: NCT00319020     History of Changes
Other Study ID Numbers: AC-052-367  2005-001967-70 
Study First Received: April 26, 2006
Last Updated: June 2, 2016
Health Authority: Switzerland: Swissmedic
United States: Food and Drug Administration
Netherlands: Medicines Evaluation Board
Germany: Federal Institute for Drugs and Medical Devices
Italy: The Italian Medicines Agency
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Actelion:
bosentan
pulmonary arterial hypertension
children
FUTURE 1

Additional relevant MeSH terms:
Hypertension
Familial Primary Pulmonary Hypertension
Vascular Diseases
Cardiovascular Diseases
Hypertension, Pulmonary
Lung Diseases
Respiratory Tract Diseases
Bosentan
Antihypertensive Agents
Endothelin Receptor Antagonists
Molecular Mechanisms of Pharmacological Action

ClinicalTrials.gov processed this record on July 27, 2016