A 5-month research project for children aged from 1 year to 17 years who have high blood pressure in the lung, to see how well bosentan decreases this high blood pressure and improves children's physical capabilities compared to an inactive compound

Phase 1

• Conditions: Pulmonary arterial hypertension (PAH) in children; MedDRA version: 14.1Level: LLTClassification code 10064908Term: Associated with (APAH)System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders; MedDRA version: 14.1Level: LLTClassification code 10064909Term: Idiopathic (IPAH)System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders; MedDRA version: 14.1Level: LLTClassification code 10064910Term: Familial (FPAH)System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders; Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]

• Registration Number: EUCTR2011-004106-16-BG
• Lead Sponsor: Actelion pharmaceuticals Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Completion: 2012-06-01
• Study Start: 2012-06-11
• Last Update Posted: 8 August 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 105

Inclusion Criteria

1. Signed informed consent by the parents or legal representatives prior to any study mandated procedure.

2. Male or female = 1 year and < 17 years of age (maximum age
at randomization is 16 years and 9 months).

3. WHO functional class I–IV.

4. PAH diagnosed with Right Heart Catheterization (RHC).

5. PAH etiologies:
Idiopathic or heritable PAH,
or
Associated PAH persisting/recurrent for at least 6 months
after complete repair of a congenital heart defect,
or
CHD (PAH associated with systemic-to-pulmonary shunts
including Eisenmenger syndrome).

6. PVR > 3 Wood units for idiopathic, heritable PAH or PAH
persisting/recurrent after complete repaired of congenital
heart defect.
or
PVR > 8 Wood units for PAH associated with systemic-to-pulmonary open shunts with a ratio Qp/Qs < 2.

7. Body weight = 8 kg and = 95 kg.

8. Patients naïve to PAH-specific treatment
or
Patients already treated with PAH-specific medication at a stable dose since at least 3 months:
Prostanoids, PDE-5 inhibitors
No combination of PAH-specific therapy is allowed.

9. Patients naïve to PAH related, non-specific medication
or
Patients already treated with PAH related, non-specific medication, such as
Calcium channel blockers (stable dose since at least 3 months)
Anticoagulants
Diuretics
Digoxin

10. Females of childbearing potential must have a negative
pre-treatment pregnancy test and must use reliable methods of
contraception during the whole treatment period and for 1 month after study drug discontinuation.
Are the trial subjects under 18? yes
Number of subjects for this age range: 105
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Non-stable disease status, e.g., history of recurrent (near-)
syncope or signs and symptoms of non-compensated right heart failure.

2. Patients with bronchopulmonary dysplasia, congenital
diaphragmatic hernia or other chronic lung diseases.

3. Need or plan to wean patient from any PAH-specific therapies
during the study.

4. Systolic blood pressure < 80% of the lower limit of normal
range.

5. Aspartate aminotransferase (AST) and/or alanine
aminotransferase (ALT) values > 1.5 times the upper limit of
normal range.

6. Moderate or severe hepatic impairment (i.e., Child-Pugh Class
B or C).

7. Hemoglobin and/or hematocrit levels < 75% of the lower limit
of normal range.

8. Known intolerance or hypersensitivity to bosentan or any of
the excipients of the bosentan dispersible tablet.

9. Treatment with bosentan or any other endothelin receptor
antagonists within 4 months prior to randomization (including any ERA investigational drug).

10. Treatment with another investigational drug within 1 month
prior to randomization or planned treatment.

11. Pregnancy or breastfeeding.

12. Any condition that prevents compliance with the protocol or
adherence to therapy.

13. Administration of prohibited medication within 2 weeks or
5 times the half-life, prior to randomization, whichever is the
longest.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified