(15681) Open-label, individual dose titration study to evaluate safety, tolerability and pharmacokinetics of riociguat in children from 6 to less than 18 years of age with pulmonary arterial hypertension (PAH)

Pulmonary arterial hypertension (PAH)

Incidence of adverse events and serious adverse events, Recording of vital signs, Left-hand x-ray, Pharmacokinetics/Pharmacodynamics analyses

6-Minute Walking Distance (6MWD)., WHO functional class., N-terminal prohormone brain-type natriuretic peptide., Quality of Life scores (parent questionnaire and in children able to understand questions): Child Health-related Questionnaire (SF-10) and PedsQL Generic Core scales self-report, Echocardiographic parameters including: o pulmonary arterial systolic pressure (PASP), o tricuspid annular plane systolic excursion (TAPSE), o pericardial effusion, o left ventricular eccentricity index, o estimated right atrial pressure o right ventricular pressure by tricuspide regurgitant jet velocity, o acceleration time of pulmonary flow o right heart dimensions and o computing of cardiac output, Time to clinical worsening defined as: o hospitalization for right heart failure, o death, o lung transplantation, o Pott’s anastomosis and atrioseptostomy o worsening of PAH symptoms, which must include either an increase in WHO functional class, OR both appearance/worsening symptoms of right heart failure AND need for additional PAH therapy., Taste and texture of the pediatric formulation(s) must be assessed by use of a questionnaire.

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Germany, Italy