A Phase 2 interventional study of Bosentan and Placebo in Hypoplastic Left Heart Syndrome, Tricuspid Atresia and Other Specified Congenital Anomalies of Heart, sponsored by Copenhagen University Hospital, Rigshospitalet. Completed at 4 sites in 2 countries. Open to participants aged 15 Years and older. Per ClinicalTrials.gov, last updated 2013-04-19.
Sponsored by Copenhagen University Hospital, Rigshospitalet · Phase 2, Interventional, and Treatment
The purpose of this study is to determine whether Bosentan is an effective and safe treatment to adolescent and adult (15 years and older) patients, born with one ventricle of the heart instead of two (single ventricle physiology) and who have undergone TCPC as a palliative surgical treatment. The aim of the TCPC operation is to use the one functioning ventricle to pump the blood flow to the body, while the blood to the lungs is received directly from the caval veins, and is thus a passive flow, without the aid of a ventricle to actively pump the blood through the pulmonary circulation. The resistance in the pulmonary circulation is therefore critical to these patients. These patients have markedly lower work capacity in bicycle test than the general public. Furthermore they have a high risk of developing complications e.g. loss of protein from the intestines.
Bosentan is a medication that lowers the resistance in the pulmonary circulation. It is routinely used for patients with pulmonary hypertension. Some studies have shown that drugs that lower the pulmonary resistance can increase exercise capacity significantly in patients with single ventricle physiology.
In this study 80 patients will receive either placebo or Bosentan for 14 weeks. Before and after the treatment, bicycle test along with blood samples, stool samples and quality of life interviews will be performed. Every four weeks during the study blood samples, physical exam and interviews will be performed to ensure the safety of the treatment.
The investigators expect to find a significant increase in work capacity after 14 weeks in the treatment group compared with the placebo group.
Moreover the investigators hope to find a decrease in intestinal protein loss and an improved quality of life.
In the statistical analysis the investigators wish to analyse interactions between the primary endpoint and predefined subgroups in order to distinguish responders from non-responders to the treatment. The predefined subgroups are:
NT-proBNP > 100 (yes/no) Ventricular anatomy (RV/LV) CT-proEndothelin-1. In the latter, the investigators do not wish to predefine a specific value, due to limited experience with this analysis. We wish to use the data from the study to find a cut-off value, that is able to predict positive response to treatment.
65 studies on the registry are indexed under Hypoplastic Left Heart Syndrome; 12 are open to participants now.
This study's enrollment of 75 is above the median of 29 across 39 interventional studies indexed under Hypoplastic Left Heart Syndrome.
Browse Hypoplastic Left Heart Syndrome studies →Copenhagen University Hospital, Rigshospitalet is the lead sponsor of 34 studies on the registry; 11 are open to participants now.
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Drug: Bosentan
Drug: Placebo
tablets Bosentan 62,5 mg x 2 daily for two weeks, then 125 mg x 2 daily for 12 weeks
Also known as: Bosentan: Tracleer
Placebo tablets 2 x daily for 2 weeks, then change to different placebo tablets to match Bosentan group, 2 x daily for 12 weeks
Change from baseline in VO2max at 14 weeks
Maximal O2 uptake in ml/min/kg in ergometer bicycle test
Time frame: Baseline and 14 weeks
Change from baseline in blood samples at 14 weeks
blood samples measured: plasma CT pro endothelin-1, IgG, IgA, NT pro BNP, albumin, free protein
Time frame: Baseline and 14 weeks
Change from baseline in SF36 questionnaire score at 14 weeks
SF36 quality of life interview
Time frame: Baseline and 14 weeks
Change from baseline in feces alfa 1 antitrypsin at 14 weeks
Fecal alfa 1 antitrypsin in mg/g
Time frame: Baseline and 14 weeks
Number of participants with adverse events
general interview on adverse effects, and questions with special focus on typical adverse effects in Bosentan
Time frame: 2, 6, 10 and 14 weeks after start of treatment
Change from baseline in vital signs
Systemic bloodpressure in mmHg, Pulse in min-1, Oxygen saturation in percent
Time frame: Baseline, 2, 6, 10 and 14 weeks
Change from baseline in control blood samples
Liver and renal biomarkers, Hb, PCV, trc and hCG for women
Time frame: Baseline, 2, 6, 10 and 14 weeks
Change from baseline in cardiac output/pulmonary blood flow
CO measured by Stringer Wassermann method during ergometer bicycle test
Time frame: Baseline and 14 weeks
This study is completed, as verified in Apr 2013. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Bosentan
Hypoplastic Left Heart Syndrome →
Copenhagen University Hospital, Rigshospitalet