10mg macitentan tablets, taken once daily (QD), by mouth (PO), for the treatment period lasting 16 weeks.

Pulmonary Hypertension

Sickle Cell Disease

macitentan

Boston University School of Medicine

Macitentan in Pulmonary Hypertension of Sickle Cell Disease

The Safety and Efficacy of Macitentan for Treatment of Pulmonary Hypertension in Sickle Cell Disease

Assess the rate of adverse and serious adverse events in SCD patients taking macitentan

Assess the change in the following hemodynamic parameters, by right heart catheterization, from baseline after 16 weeks of 10 mg daily of macitentan:
Right atrial pressure (RAP) Right ventricular pressure (RVP) Systolic pulmonary artery pressure (SPAP) Diastolic pulmonary artery pressure (PADP)

Assess the change in the following hemodynamic parameters, by right heart catheterization, from baseline after 16 weeks of 10 mg daily of macitentan:
Cardiac output (CO)

Assess the change in the following hemodynamic parameters, by right heart catheterization, from baseline after 16 weeks of 10 mg daily of macitentan:
Cardiac index (CI)

Assess the change in the following hemodynamic parameters, by right heart catheterization, from baseline after 16 weeks of 10 mg daily of macitentan:
Systemic vascular resistance (SVR) Right ventricular function assessed by pressure-volume loop measurements

Assess the change in the following hemodynamic parameters, by right heart catheterization, from baseline after 16 weeks of 10 mg daily of macitentan:
Right ventricular function assessed by pressure-volume loop measurements

Assess the change from baseline in 6MWT after 16 weeks of 10 mg daily macitentan

Assess the change from baseline of Borg Dyspnea Index (BDI) after 16 weeks of 10 mg daily macitentan

Assess the change from baseline in NYHA/WHO functional classification after 16 weeks of 10 mg daily macitentan

Assess the change from baseline in cardiac biomarkers (e.g., BNP, NT-pro-BNP) after 16 weeks of 10 mg daily macitentan

Boston University

Actelion

Please see the Study Description for complete information.

Immunodeficiency

St. Jude Children's Research Hospital

Peripheral Blood Stem Cells Obtained From Normal Volunteers for Studying Retroviral Vector Mediated Gene Transfer Into Primitive Hematopoietic Cells and Vector Mediated Transgene Expression in Mature Hematopoietic Lineages

hemoglobinopathies/ból głowy

Macitentan in Pulmonary Hypertension of Sickle Cell Disease

Peripheral Blood Stem Cells Obtained From Normal Volunteers for Studying Retroviral Vector Mediated Gene Transfer Into Primitive Hematopoietic Cells and Vector Mediated Transgene Expression in Mature Hematopoietic Lineages

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