Bosentan Therapy in Children With Functional Single Ventricle

Shanghai Jiao Tong University School of Medicine

Status and phase: Completed; Phase 2

Conditions: Congenital Heart Defects; Functional Single Ventricle

Treatments: Drug: Bosentan

Study type: Interventional

Funder types: Other

Identifiers

NCT01662037

SJTUMS-20120314

Details and patient eligibility

About

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.

Full 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.

Enrollment: 34 patients

Sex: All

Ages: 4 months to 18 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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34 participants in 2 patient groups

Bosentan group

Experimental group

Description:

Bosentan 2mg/kg/dose twice a day and routinely therapy in children with functional single ventricle during the period of staged Fontan procedure with high risk of increased PVR.

Treatment:

Drug: Bosentan

Routinely group

No Intervention group

Description:

Routinely therapy in children with functional single ventricle during the period of staged Fontan procedure with high risk of increased PVR.