Bosentan in Children With Pulmonary Arterial Hypertension

NCT ID: NCT00319267

Last Updated: 2025-02-03

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 36 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2005-05-31
• Study Completion Date: 2007-02-28

Brief Summary

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.

Conditions

Pulmonary Arterial Hypertension

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Bosentan

The initial dose of bosentan was 2 mg/kg b.i.d. for 4 weeks. After 4 weeks, the initial dose was up-titrated to the maintenance dose of 4 mg/kg b.i.d. up to the end of the study treatment at Week 12. If the maintenance dose was not well tolerated, the dose could be down-titrated to the initial dose.

Group Type: EXPERIMENTAL

Bosentan

Intervention Type: DRUG

Pediatric oral formulation of bosentan, i.e., 32 mg dispersible and breakable tablets

Interventions

Bosentan

Pediatric oral formulation of bosentan, i.e., 32 mg dispersible and breakable tablets

Intervention Type: DRUG

Other Intervention Names

ACT-050088; Ro 47-0203

Eligibility Criteria

Inclusion Criteria

• Signed informed consent by the parents or the legal representatives.
• Males or females >= 2 and < 12 years of age.
• 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 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 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.

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Actelion

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

References

Beghetti M, Haworth SG, Bonnet D, Barst RJ, Acar P, Fraisse A, Ivy DD, Jais X, Schulze-Neick I, Galie N, Morganti A, Dingemanse J, Kusic-Pajic A, Berger RM. Pharmacokinetic and clinical profile of a novel formulation of bosentan in children with pulmonary arterial hypertension: the FUTURE-1 study. Br J Clin Pharmacol. 2009 Dec;68(6):948-55. doi: 10.1111/j.1365-2125.2009.03532.x.

Reference Type: RESULT

PMID: 20002090 (View on PubMed)

Other Identifiers

2004-005157-63

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

AC-052-365

Identifier Type: -

Identifier Source: org_study_id