Pulmonary Arterial HypertensionPhase 3FUTURE-1NCT00319267
Bosentan in Children With Pulmonary Arterial Hypertension
A Phase 3 study testing Bosentan in people with Pulmonary Arterial Hypertension, run by Actelion.
Trial 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… Read more about this study
- Who is it for?
- Ages 2 to 12 years
- Conditions the study is for
- Pulmonary Arterial Hypertension
- What is being tested?
- Bosentan
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- No sites listed yet
- Study dates
- May 2005to Feb 2007
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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:
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- 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.
Who cannot join
- 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.
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- 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.
About this study
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
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
- Being tested
- Bosentan
- Keywords
- bosentan, children, pharmacokinetics, pulmonary arterial hypertension
- Medical subject headings
- Pulmonary Arterial Hypertension
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BosentanDrug
Also called ACT-050088, Ro 47-0203
Pediatric oral formulation of bosentan, i.e., 32 mg dispersible and breakable tablets
| Study group | Role | What they receive |
|---|---|---|
| Bosentan | Experimental | 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. Receives: Drug: Bosentan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Area under the plasma concentration-time curve during a dose interval (AUCt) for bosentan
AUCt was assessed at steady state (i.e., after at least 2 weeks of treatment with a same dose of the study drug) over 12 hours .
Measured: At pre-dose and 0.5h, 1h, 3h, 7.5h, and 12h post-dose
Secondary outcomes (3)
Maximum plasma concentration (Cmax) of bosentan and its metabolites
Maximum observed plasma concentration for bosentan and its metabolites was directly derived from their respective plasma concentration-time curves.
Measured: At pre-dose and 0.5h, 1h, 3h, 7.5h, and 12h post-dose
Time to reach the maximum plasma concentration (tmax) of bosentan and its metabolites
Measured: At pre-dose and 0.5h, 1h, 3h, 7.5h, and 12h post-dose
Area under the plasma concentration-time curve during a dose interval (AUCt) for the metabolites of bosentan
AUCt was assessed at steady state (i.e., after at least 2 weeks of treatment with a same dose of the study drug) over 12 hours.
Measured: At pre-dose and 0.5h, 1h, 3h, 7.5h, and 12h post-dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2006 (estimated)
- Study startMay 2005
- Primary completionDec 2006
- Study completionFeb 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Pulmonary Arterial HypertensionStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Actelion
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00319267
- Sponsor's protocol ID
- AC-052-365
- Other IDs
- 2004-005157-63, 2004
- Acronym
- FUTURE-1
- Status last verified
- Jan 2025
- First submitted
- 26 Apr 2006
- Last updated
- 3 Feb 2025
- Results posted
- No
Publications
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.
PubMed 20002090
Study at a glance
From the registry record, last updated by the sponsor on 3 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00319267
- Acronym / sponsor ID
- FUTURE-1 / AC-052-365
- Conditions
- Pulmonary Arterial Hypertension
- Keywords
- bosentan, children, pharmacokinetics, pulmonary arterial hypertension
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- ActelionIndustry (pharma, biotech, devices)
- Study start
- May 2005
- Primary completion
- Dec 2006 (actual)
- Study completion
- Feb 2007 (actual)
- First posted
- 27 Apr 2006 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Bosentan | Experimental | Drug: BosentanThe 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. |
Who may qualify
- Ages
- 2 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (15 listed)
- 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).
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BosentanAlso: ACT-050088, Ro 47-0203Pediatric oral formulation of bosentan, i.e., 32 mg dispersible and breakable tablets | Drug | Bosentan |
Primary outcome
Area under the plasma concentration-time curve during a dose interval (AUCt) for bosentan
Time frame: At pre-dose and 0.5h, 1h, 3h, 7.5h, and 12h post-dose
AUCt was assessed at steady state (i.e., after at least 2 weeks of treatment with a same dose of the study drug) over 12 hours .
3 secondary outcomes
Maximum plasma concentration (Cmax) of bosentan and its metabolites
Time frame: At pre-dose and 0.5h, 1h, 3h, 7.5h, and 12h post-dose
Time to reach the maximum plasma concentration (tmax) of bosentan and its metabolites
Time frame: At pre-dose and 0.5h, 1h, 3h, 7.5h, and 12h post-dose
Area under the plasma concentration-time curve during a dose interval (AUCt) for the metabolites of bosentan
Time frame: At pre-dose and 0.5h, 1h, 3h, 7.5h, and 12h post-dose
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Feb 2025.
- Results publication: PubMed 20002090Beghetti 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Bosentan in people with Pulmonary Arterial Hypertension, run by Actelion.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Area under the plasma concentration-time curve during a dose interval (AUCt) for bosentan (time frame: At pre-dose and 0.5h, 1h, 3h, 7.5h, and 12h post-dose). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00319267, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Feb 2025.