Clinical trial NCT01662037: Bosentan Therapy in Children With Functional Single Ventricle
Official title: Bosentan Therapy for High Risk Staged Fontan Procedure in Children With Functional Single Ventricle
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Congenital Heart Defects
Functional Single Ventricle
Keywords: Pulmonary vascular resistance
Heart defects, Congenital
Receptors, endothelin
Bosentan
Lead sponsor: Shanghai Jiao Tong University School of Medicine
Sponsor class: OTHER
First posted: 2012-08-10
Enrollment: 34
Sex: ALL
Minimum age (years): 4
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Bosentan is a kind of dual endothelin receptor antagonist.The purpose of this study is to investigate if Bosentan therapy can modify the outcome of children with functional single ventricle.

Detailed description: Increased pulmonary vascular resistance (PVR) is a serous issues in children with functional single ventricle during the staged operative period. The purpose of this study is to investigate if Bosentan therapy can improve the survival and life quality after staged Fontan procedure in the children with high risk of increased PVR.

Eligibility criteria: Inclusion Criteria:

* Signed informed consent obtained from patient's legally acceptable representative.
* Pediatric patients waiting for staged Fontan procedure with high risk of increased PVR after bidirectional cavopulmonary connection (BCPC)

  * Transpulmonary pressure gradiant (TPG) \> 10mmHg when the obstruction of anastomosis and lung problem were excluded.
  * With the diagnosis of high risk of increased PVR, such as associated with TAPVC, after pulmonary artery banding, after systemic to pulmonary shunt more than 6 months, and et al.
  * Diagnosed as increased PVR with catheterization.

Exclusion Criteria:

* PAH associated with conditions other than those mentioned above, e.g., iPAH, PAH secondary to portal hypertension, HIV patient with opportunistic infection
* Psychotic, addictive or other disorder limiting the ability to provide informed consent or to comply with study requirements
* AST and/or ALT \> 3 times the upper limit of normal ranges.
* Hemoglobin concentration \< 75% the lower limit of normal ranges
* Treatment or planned treatment with another investigational drug within 3 months of screening
* Treatment with calcineurin-inhibitors (e.g., cyclosporine A and tacrolimus), fluconazole, glibenclamide (glyburide) within 1 week of enrollment of this study
* Known hypersensitivity to bosentan or any of the excipients

Primary outcomes:
measure: Length of hospital stay and ICU stay
timeFrame: 12 months after Fontan operation
Secondary outcomes:
measure: Symptoms of increased PVR
timeFrame: 12 months after Fontan operation
measure: WHO functional class
timeFrame: 12 months after Fontan operation
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Cardiac intensive care unit, Department of cardiothoracic vascular surgery, Shanghai Children's Medical Center, Medical college of Shanghai Jiaotong University
state: Shanghai
Study officials:
affiliation: Cardiac intensive care unit, Department of Thoracic and cardiovascular Surgery, Shanghai children's Medical Center
name: Zhuoming Xu, MD PhD
role: STUDY_DIRECTOR
References: