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Effects of Two Dosing Regimens of Bosentan in Children With Pulmonary Arterial Hypertension (FUTURE 3)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Actelion
ClinicalTrials.gov Identifier:
NCT01223352
First received: October 12, 2010
Last updated: May 29, 2017
Last verified: May 2017
Results First Received: March 2, 2017  
Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: No masking;   Primary Purpose: Treatment
Condition: Pulmonary Arterial Hypertension
Intervention: Drug: bosentan

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Children with pulmonary arterial hypertension (PAH) were recruited from 45 expert pediatric centers in 20 countries worldwide

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Bosentan 2 mg/kg t.i.d. 2 mg/kg bosentan (dispersible tablets) was administered orally three times a day (t.i.d.) for a planned duration of 24 weeks
Bosentan 2 mg/kg b.i.d. 2 mg/kg bosentan (dispersible tablets) was administered orally twice daily (b.i.d.) for a planned duration of 24 weeks

Participant Flow:   Overall Study
    Bosentan 2 mg/kg t.i.d.   Bosentan 2 mg/kg b.i.d.
STARTED   31   33 
COMPLETED   31   33 
NOT COMPLETED   0   0 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Bosentan 2 mg/kg t.i.d. 2 mg/kg bosentan (dispersible tablets) was administered orally three times a day (t.i.d.) for a planned duration of 24 weeks
Bosentan 2 mg/kg b.i.d. 2 mg/kg bosentan (dispersible tablets) was administered orally twice daily (b.i.d.) for a planned duration of 24 weeks
Total Total of all reporting groups

Baseline Measures
   Bosentan 2 mg/kg t.i.d.   Bosentan 2 mg/kg b.i.d.   Total 
Overall Participants Analyzed 
[Units: Participants]
 31   33   64 
Age 
[Units: Years]
Mean (Standard Deviation)
 5.2  (3.81)   4.5  (3.35)   4.8  (3.57) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female      10  32.3%      18  54.5%      28  43.8% 
Male      21  67.7%      15  45.5%      36  56.3% 
Region of Enrollment 
[Units: Participants]
     
Australia   2   0   2 
Belarus   1   2   3 
China   2   4   6 
Czech Republic   1   1   2 
France   1   4   5 
Germany   3   1   4 
Hungary   3   0   3 
India   1   2   3 
Israel   0   2   2 
Italy   1   0   1 
Mexico   1   0   1 
Poland   2   3   5 
Russia   4   6   10 
Serbia   2   3   5 
South Africa   4   2   6 
Spain   1   2   3 
USA   1   1   2 
Ukraine   1   0   1 
Etiology of pulmonary arterial hypertension (PAH) [1] 
[Units: Participans]
     
Idiopathic   15   14   29 
Heritable   0   2   2 
PAH-Congenital heart disease   2   6   8 
Associated PAH   13   11   24 
Missing data   1   0   1 
[1]

Children included in the study had a diagnosis of PAH belonging to one of the following categories:

  • Idiopathic PAH
  • Heritable PAH
  • Associated PAH persisting after complete repair of a congenital heart defect
  • PAH-congenital heart disease (PAH-CHD) associated with open shunts
World Health Organization functional class (WHO FC) [1] 
[Units: Participants]
     
FC I   10   9   19 
FC II   15   12   27 
FC III   6   12   18 
[1]

The World Health Organization (WHO) defines 4 classes to classify the functional status of patients with pulmonary hypertension:

Class I (FC I): No limitation of physical activity; Class II (FC II): Slight limitation of physical activity; Class IIII (FC III): Marked limitation of physical activity; Class IV (FC IV): Inability to carry out any physical activity without symptoms.

PAH-specific therapy at baseline 
[Units: Participants]
     
Bosentan (adult tablet formulation)   4   3   7 
Prostanoid   1   0   1 
Phosphodiesterase type-5 (PDE-5) inhibitor   13   10   23 
Bosentan / PDE-5 inhibitor combination   2   2   4 
Bosentan /PDE-5 inhibitor /Prostanoid combination   2   5   7 
None   9   13   22 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Dose-corrected Daily Exposure [AUC(0-24c)] to Bosentan   [ Time Frame: 0, 0.5, 1, 3, 5 (or 7.5), 8 (or 12 hours) post-dose at Week 4, after at least 2 weeks of stable study drug treatment ]

2.  Other Pre-specified:   Dose-corrected Maximum Plasma Concentration [Cmaxc] of Bosentan   [ Time Frame: 0, 0.5, 1, 3, 5 (or 7.5), 8 (or 12 hours) post-dose at Week 4, after at least 2 weeks of stable study drug treatment ]

3.  Other Pre-specified:   Time to Reach Cmax [Tmax] of Bosentan   [ Time Frame: 0, 0.5, 1, 3, 5 (or 7.5), 8 (or 12 hours) post-dose at Week 4, after at least 2 weeks of stable study drug treatment ]

4.  Other Pre-specified:   Dose-corrected Daily Exposure [AUC(0-24c)] to Bosentan Metabolites (Ro 478634, Ro 485033, Ro 641056)   [ Time Frame: 0, 0.5, 1, 3, 5 (or 7.5), 8 (or 12 hours) post-dose at Week 4, after at least 2 weeks of stable study drug treatment ]

5.  Other Pre-specified:   Change From Baseline in WHO Functional Class at End of Study   [ Time Frame: Baseline, up to Week 24 on average ]

6.  Other Pre-specified:   Change From Baseline in Global Clincial Impression Scale (GCIS) at End of Study   [ Time Frame: Baseline, up to Week 24 on average ]

7.  Other Pre-specified:   Number of Patients With Treatment-emergent Liver Function Abnormalities   [ Time Frame: Baseline, up to Week 24 on average ]

8.  Other Pre-specified:   Number of Patients With Treatment-emergent Hemoglobin Abnormalities   [ Time Frame: Baseline, up to Week 24 on average ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: clinical trial disclosure desk
Organization: Actelion Pharmaceuticals Ltd
e-mail: clinical-trials-disclosure@actelion.com



Responsible Party: Actelion
ClinicalTrials.gov Identifier: NCT01223352     History of Changes
Other Study ID Numbers: AC-052-373
Study First Received: October 12, 2010
Results First Received: March 2, 2017
Last Updated: May 29, 2017