Biomarkers in Pediatric Congenital Heart Disease and PAH
Congenital Heart DiseasePulmonary Arterial HypertensionNowadays, biomarkers are broadly used in clinical practice. Blood-derived biomarkers fulfil an important role in the field of cardiology. However, most biomarkers have been investigated for adult left ventricular disease. In congenital heart diseases (CHD) and pulmonary arterial hypertension (PAH), which involves children and mostly the right ventricle, less is known about the clinical and predictive value of blood-derived biomarkers. Since the group of survivors of CHD and PAH is growing because of the improved techniques nowadays, development of better tools to maintain the quality of life for the longer term in these patients is urgently needed. Blood-derived biomarkers are minimally invasive biomarkers, are quantitative and have shown to be able to reveal pathological processes in an early stage. Hence, blood-derived biomarkers may be a good addition to current diagnostic means in CHD and PAH. Objective: The primary objective of this study is to investigate cross-sectionally the association between various emerging blood-derived biomarkers and right ventricular (RV) function:defined as tricuspid annular plane systolic excursion (TAPSE) measured with echocardiography, in children with (a history of ) an abnormally loaded, volume and/or pressure loaded, right ventricle associated with CHD and/or PAH.
PRIMEx - A Study of 2 Doses of Oral CXA-10 in Pulmonary Arterial Hypertension (PAH)
PAHThis is a multicenter double-blind, placebo-controlled study to evaluate the safety, efficacy and pharmacokinetics of 2 doses of CXA-10 on stable background therapy in 96 subjects 18 to 80 years of age with PAH.
A Study of Pulmonary Arterial Hypertension Participants Treated With Macitentan or Selexipag
Pulmonary Arterial HypertensionThe purpose of the study is to evaluate the change from baseline to 12 months after study enrollment in the number of the following non-invasive risk criteria: World Health Organization Functional Class (WHO/FC), 6-minute walk distance (6MWD), Brain Natriuretic Peptide (BNP) or N-terminal pro-brain Natriuretic Peptide (NT-proBNP).
Long-term Study of AIR001 in Subjects With WHO Group 1 Pulmonary Arterial Hypertension Who Completed...
Pulmonary Arterial HypertensionThe AIR001-CS05 study evaluated the safety and efficacy (effectiveness) of AIR001 over 16 weeks in subjects who have PAH. The purpose of the AIR001-CS06 study is to evaluate the intermediate / long-term safety of AIR001 in subjects who have completed the AIR001-CS05 study. Assessments to evaluate the effectiveness of the study drug will include measurements of exercise ability and evaluations of PAH disease symptoms.
Revatio Portal-Pulmonary Arterial Hypertension Trial
Portopulmonary HypertensionThe investigators propose the first prospective, double blind, randomized controlled trial of treatment for pulmonary arterial hypertension (PAH) related to underlying portal hypertension. Specifically the investigators will evaluate the potential efficacy and safety of sildenafil (Revatio) in a 16 week blinded, multicentre study.
Safety Study of PLX-PAD Cells to Treat Pulmonary Arterial Hypertension (PAH)
Pulmonary Arterial HypertensionThe purpose of this clinical study is to assess the safety of PLX-PAD to treat pulmonary arterial hypertension (PAH). PLX-PAD is a cell-based product made of allogeneic Mesenchymal-like Adherent Stromal Cells (ASCs), derived from human full-term placentas following an elective caesarean section. This year-long study will evaluate the safety of three different dose levels of PLX-PAD, each given as a single intravenous infusion. This study will also evaluate effects that PLX-PAD may have on PAH, such as changes in the ability to exercise and on other tests used to measure the disease severity.
Extension to QTI571A2301 to Evaluate the Long-term Safety, Tolerability and Efficacy of Imatinib...
Pulmonary Arterial HypertensionThis is a multinational, multi center extension study. This study will provide data on the long-term safety, tolerability, and efficacy of imatinib in the treatment of severe pulmonary arterial hypertension.
Therapy of Pulmonary Arterial Hypertension (PAH) - Treatment With Sildenafil in Eisenmenger Patients...
Pulmonary Arterial Hypertension (PAH)Eisenmenger's syndrome presents as a severe clinical picture of polymorbidity that constitutes a great burden at the individual as well as the familial and social level. The combination of critically increased pulmonary vascular resistance, progressive pressure load of the right ventricle and disturbance of pulmonary gas exchange result in long-term polymorbidity. The objective of this study is to provide evidence of improvement of patients exercise tolerance as well as general conditions by treatment with oral sildenafil as a specific pulmonary vasodilator.
Rapid Switch From Flolan to Remodulin in the Outpatient Clinic
HypertensionPulmonaryThe purpose of this 8-week study is to compare the effects of switching from therapy with epoprostenol or Flolan to IV Remodulin. This study will also assess the effect that changing to Remodulin will have on patient satisfaction with their treatment and impact on quality of life.
CXA-10 Study in Subjects With Pulmonary Arterial Hypertension
PAHThis is a Phase 2, multicenter, open-label extension (OLE) of study CXA-10-301, to evaluate the long term safety and efficacy of daily dosing of CXA-10.