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. - FUTURE 1
- Conditions
- Idiopathic or familial PAH
- Registration Number
- EUCTR2004-005157-63-GB
- Lead Sponsor
- Actelion Pharmaceuticals Ltd.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 33
All patients should start the study drug (bosentan pediatric formulation) at the 2mg/kg b.i.d whether or not they were previously treated with bosentan.
Eligible patients must meet all of the following inclusion criteria during the Screening visit:
· Signed informed consent by the parents or the legal representatives.
· Male or female =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. If using hormonal contraception, an additional alternative reliable method of contraception must be used.
- Reliable method of contraception are:
Barrier type devices (e.g., female condom, diaphragm, contraceptive sponge) only in combination with a spermicide.
Intra-uterine devices.
Oral, injectable or implantable contraceptives only in combination with a barrier method.
- Hormone-based contraceptives alone, regardless of the route of administration, are not considered as reliable methods of contraception.
- Abstention, rhythm method, and contraception by the partner alone are not acceptable methods of contraception.
. Idiopathic PAH or familial PAH diagnosed by right heart catheterization
(Clinical classification of pulmonary hypertension - Venice 2003).
· WHO functional class II or III. (see Appendix 3 of the protocol).
· 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.
· 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.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years)
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years)
F.1.3.1 Number of subjects for this age range
Patients will not be included in the study if any of the following exclusion criteria are present at the Screening visit:
· 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. (See Appendix 1 of the protocol).
· 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. (See Appendix 4 of the protocol).
· 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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The primary endpoint is to demonstrate that the exposure to bosentan in children with IPAH or familial PAH, using a pediatric formulation, is similar to that in adults with PAH.;Secondary Objective: The secondary objective of this study is to evaluate the tolerability and safety of a pediatric formulation of bosentan in this patient population.;Primary end point(s): The main analysis of the primary endpoint is performed on the PK-evaluable set for this equivalence study.
- Secondary Outcome Measures
Name Time Method