Bosentan in Children With Pulmonary Arterial Hypertension

This study has been completed.
Information provided by:
Actelion Identifier:
First received: April 26, 2006
Last updated: February 11, 2010
Last verified: February 2010
The aim of the study is to demonstrate that the exposure to bosentan in children with idiopathic pulmonary arterial hypertension (PAH) or familial pulmonary arterial hypertension, using a pediatric formulation, is similar to that in adults with PAH and to evaluate the tolerability and safety of a pediatric formulation of bosentan in this patient population.

Condition Intervention Phase
Pulmonary Hypertension
Drug: bosentan
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Pharmacokinetics Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open Label, Multicenter Study to Assess the Pharmacokinetics, Tolerability, and Safety of a Pediatric Formulation of Bosentan in Children With Idiopathic or Familial Pulmonary Arterial Hypertension

Resource links provided by NLM:

Further study details as provided by Actelion:

Primary Outcome Measures:
  • Pharmacokinetic (PK) assessments of bosentan - area under the curve to last time point of bosentan at PK visits

Secondary Outcome Measures:
  • Adverse events up to 24 hours after permanent discontinuation of drug

Estimated Enrollment: 33
Study Start Date: May 2005
Study Completion Date: February 2007
Primary Completion Date: December 2006 (Final data collection date for primary outcome measure)

Ages Eligible for Study:   2 Years to 12 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Signed informed consent by the parents or the legal representatives.
  • Males or females >= 2 and < 12 years of age. Females who are menstruating must have a negative serum pregnancy test. A reliable method of contraception must be considered, if appropriate.
  • Idiopathic PAH or familial PAH diagnosed by right heart catheterization. (Clinical classification of pulmonary hypertension - Venice 2003.)
  • World Health Organization (WHO) functional class II or III.
  • Oxygen saturation (SpO2) >= 88% (at rest, on room air).
  • PAH treatment-naïve patients or patients already treated with either:

    • Bosentan monotherapy
    • Intravenous epoprostenol monotherapy
    • Intravenous or inhaled iloprost monotherapy
    • Combination of bosentan and intravenous epoprostenol
    • Combination of bosentan and intravenous or inhaled iloprost.
  • All patients should start the study drug (bosentan pediatric formulation) at 2 mg/kg twice daily (b.i.d.), whether or not they were previously treated with bosentan.
  • PAH therapy must have been stable for at least 3 months prior to Screening.
  • Stable treatment with calcium channel blockers, if any, for at least 3 months prior to Screening.
  • Patient's PAH condition must have been stable for at least 3 months prior to Screening.

Exclusion Criteria:

  • PAH associated with conditions other than idiopathic or familial PAH.
  • Non-stable patients, e.g., history (in the last 3 months prior to Screening) of recurrent syncope, or signs and symptoms of non-compensated right heart failure.
  • Need or plan to wean patients from intravenous epoprostenol, or intravenous, or inhaled iloprost.
  • Body weight < 4 kg.
  • Systolic blood pressure < 80%, the lower limit of normal range, according to age and gender.
  • AST and/or ALT values > 3 times the upper limit of normal ranges.
  • Moderate to severe hepatic impairment, i.e., Child-Pugh Class B or C.
  • Hemoglobin and/or hematocrit levels < 75% of the lower limit of normal ranges.
  • Pregnancy.
  • Known intolerance or hypersensitivity to bosentan or any of the excipients.
  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 identifier: NCT00319267

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
Hopital Antoine Beclere
Clamart, France, 92140
Hopital Necker
Paris, France, 75743
CHE de Toulouse Hopital d'Enfants
Toulouse, France
Deutsches Herzzentrum
Augustenburger, Germany
Universitats Kinderklinik
Giessen, Germany
Policlinico S. Orsola-Malpighi
Bologna, Italy, 40138
Beatrix Children's Hospital
Groningen, Netherlands
Hopital des Enfants
Geneva, Switzerland
United Kingdom
The Institute of Child Health
London, United Kingdom
Sponsors and Collaborators
  More Information

No publications provided Identifier: NCT00319267     History of Changes
Other Study ID Numbers: AC-052-365  FUTURE 1 
Study First Received: April 26, 2006
Last Updated: February 11, 2010
Health Authority: United States: Food and Drug Administration

Keywords provided by Actelion:
pulmonary arterial hypertension

Additional relevant MeSH terms:
Hypertension, Pulmonary
Cardiovascular Diseases
Lung Diseases
Respiratory Tract Diseases
Vascular Diseases
Antihypertensive Agents
Cardiovascular Agents
Pharmacologic Actions
Therapeutic Uses processed this record on February 04, 2016