11 results
The primary objective is to evaluate the EndoRotor®*s ability to safely remove non-viable/necrotic tissue for direct endoscopic necrosectomy in patients with walled off pancreatic necrosis
Primary: To evaluate the efficacy of tisagenlecleucel therapy as measured by overall response rate by investigator assessment.Secondary: Duration of response, event free survival, relapse free survival, overall survival, safety, kinetics,…
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).
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…
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.
To evaluate the efficacy of ravulizumab compared with placebo to reduceproteinuria in adult participants with LN or IgAN.
Primary: - To evaluate the efficacy of tisagenlecleucel therapy as measured by the overall survival (OS)- To evaluate the efficacy of tisagenlecleucel therapy as measured by the 5- year disease-free survival (DFS) without censoring for new…
Main objective:To assess the efficacy of ravulizumab compared with placebo in the treatment of gMG based on the improvement in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) profile.Secondary objectives:• To assess the efficacy of…
To compare tisagenlecleucel treatment strategy to SOC treatment strategy with respect to delaying the composite event of disease progression / stable disease at or after the week 12 assessment; or death at any time.