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Bosentan in Children With Pulmonary Arterial Hypertension Extension Study (FUTURE 2)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Actelion
ClinicalTrials.gov Identifier:
NCT00319020
First received: April 26, 2006
Last updated: February 9, 2017
Last verified: February 2017
Results First Received: February 9, 2017  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Single Group 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
36 Children ( >= 2 years and < 12 years) with idiopathic or familial pulmonary arterial hypertension were recruited from 11 centers across Europe and USA and enrolled in the FUTURE 1 trial (baseline). Only patients who completed FUTURE 1 (n=34) could be enrolled in FUTURE 2. Enrollment in FUTURE 2 started August 23, 2005.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
The actual number of patients enrolled in FUTURE 2 (F-2) was 33 because 2 patients did not complete FUTURE 1 (F-1) and one patient completed F-1 but was not enrolled in F-2.

Reporting Groups
  Description
Patients With Previous Bosentan

This group included patients who already received bosentan (film-coated tablets) before enrollment in FUTURE 1, and then received the pediatric formulation of bosentan (dispersible tablets) during FUTURE 1 and FUTURE 2 (initiatied at 2 mg/kg b.i.d. for 4 weeks, then up-titrated to the maintenance dose of 4 mg/kg b.i.d. for the next 8 weeks of the FUTURE 1 trial (AC-052- 365) and to be continued in FUTURE 2. The dose could be down-titrated to 2 mg/kg b.i.d. if not tolerated).

Note: In this single arm trial, data are presented according to whether patients received bosentan or not before enrollment in FUTURE 1 but all the subjects received the study drug according to the same regimen.

Bosentan-naive Patients

This group included patients who were not treated with bosentan before enrollment in FUTURE 1, and received the pediatric formulation of bosentan (dispersible tablets) during FUTURE 1 and FUTURE 2 according to the same dosing regimen as described for "Patients with previous bosentan".

Note: In this single arm trial, data are presented according to whether patients received bosentan or not before enrollment in FUTURE 1 but all the subjects received the study drug according to the same regimen.


Participant Flow:   Overall Study
    Patients With Previous Bosentan   Bosentan-naive Patients
STARTED   15   21 
Enrollment in FUTURE 2 (F-2)   13   20 
COMPLETED   8   8 
NOT COMPLETED   7   13 
Death                2                2 
Withdrawal by Subject                4                1 
F-1 completed but not enrolled in F-2                1                0 
Administrative reason                0                5 
Disease progression                0                2 
Transplant                0                1 
Treatment failure                0                1 
Adverse Event                0                1 



  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
Patients With Previous Bosentan This group included patients who already received bosentan (film-coated tablets) before enrollment in FUTURE 1, and then received the pediatric formulation of bosentan (dispersible tablets) during FUTURE 1 and FUTURE 2 (initiation at 2 mg/kg b.i.d. for 4 weeks, then up-titrated to the maintenance dose of 4 mg/kg b.i.d. for the next 8 weeks of the FUTURE 1 trial (AC-052- 365) and to be continued in FUTURE 2. The dose could be down-titrated to 2 mg/kg b.i.d. if not tolerated).
Bosentan-naive Patients

This group included patients who were not treated with bosentan before enrollment in FUTURE 1, and received the pediatric formulation of bosentan (dispersible tablets) during FUTURE 1 and FUTURE 2 according to the same dosing regimen as described for "Patients with previous bosentan".

Note: In this single arm trial, data are presented according to whether patients received bosentan or not before enrollment in FUTURE 1 but all the subjects received the study drug according to the same regimen.

Total Total of all reporting groups

Baseline Measures
   Patients With Previous Bosentan   Bosentan-naive Patients   Total 
Overall Participants Analyzed 
[Units: Participants]
 15   21   36 
Age 
[Units: Years]
Median (Full Range)
 7 
 (3 to 10) 
 7 
 (2 to 11) 
 7 
 (2 to 11) 
Age, Customized 
[Units: Participants]
     
2-3 years old   1   3   4 
4-5 years old   3   6   9 
6-11 years old   11   12   23 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female      5  33.3%      10  47.6%      15  41.7% 
Male      10  66.7%      11  52.4%      21  58.3% 
Etiology of PAH 
[Units: Participants]
     
Idiopathic PAH   12   19   31 
Familial PAH   3   2   5 
Duration of PAH 
[Units: Months]
Median (Full Range)
 37.6 
 (1.2 to 82.6) 
 14 
 (0 to 133.5) 
 25.8 
 (0 to 133.5) 


  Outcome Measures
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1.  Primary:   Change From Baseline to End of Study (EOS) in Systolic Blood Pressure (SBP)   [ Time Frame: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation) ]

2.  Primary:   Change From Baseline to End of Study (EOS) in Diastolic Blood Pressure (DBP)   [ Time Frame: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation) ]

3.  Primary:   Change From Baseline to End of Study (EOS) in Pulse Rate   [ Time Frame: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation) ]

4.  Primary:   Change From Baseline to End of Study (EOS) in Body Weight   [ Time Frame: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation) ]

5.  Primary:   Change From Baseline to End of Study (EOS) in Height for Age.   [ Time Frame: From baseline (FUTURE 1) up to 28 days after study treatment discontinuation (EOS or premature study treatment discontinuation) ]

6.  Primary:   Proportion of Patients With Treatment-emergent Liver Function Abnormalities   [ Time Frame: After baseline, up to 1 calendar day after study treatment discontinuation in FUTURE 1 or FUTURE 2 ]

7.  Primary:   Proportion of Patients With Treatment-emergent Hemoglobin Abnormalities   [ Time Frame: After baseline, up to 1 calendar day after study treatment discontinuation in FUTURE 1 or FUTURE 2 ]

8.  Primary:   Number of Subjects With Adverse Events Leading to Premature Discontinuation of Study Treatment   [ Time Frame: From the first study drug administration in FUTURE 1 ]


  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
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  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


Publications of Results:

Responsible Party: Actelion
ClinicalTrials.gov Identifier: NCT00319020     History of Changes
Other Study ID Numbers: AC-052-367
2005-001967-70 ( EudraCT Number )
Study First Received: April 26, 2006
Results First Received: February 9, 2017
Last Updated: February 9, 2017