7 results
Primary: To determine trends in nasopharyngeal colonization with total vaccine- and non-vaccine serotypes of S. pneumoniae in healthy 11-month-old and 24-month-old infants who have been immunized according to the Dutch National Immunization Program…
Primary• To determine the safety profile of oral topotecan in subjects with cancer andmildly moderately or severely impaired renal function (Groups C, D and E) compared to subjects with cancerwhose renal function is normal and who have received…
The primary objective of this study is to assess the pharmacokinetics (PK), pharmacodynamics (PD), safety, andefficacy of ALXN1210 in pediatric patients with paroxysmal nocturnal hemoglobinuria (PNH).
Part 1 - To determine the efficacy of oral BCX9930 monotherapy administered for 24 weeks, compared to continued complement component 5 (C5) inhibitor therapy, in subjects with paroxysmal nocturnal hemoglobinuria (PNH) with an inadequate response to…
To assess the efficacy of ravulizumab versus placebo in the treatment of adult and adolescent participants with HSCT-TMA.
PrimaryTo assess the efficacy of ravulizumab in the treatment of participants with TMASecondaryTo characterize TMA responseTo assess impact on hemoglobin levels To evaluate change in kidney function To assess duration of Complete TMA Response and…
The aim of this study is to determine whether LNP023 is effective and safe for the treatment of PNH. LNP023 is compared to the Standard of Care (SOC) anti-C5 antibody treatment. The primary objectives are to:• Demonstrate superiority of LNP023…