9 results
The aim of this prospective study is 1) to evaluate the diagnostic efficacy of DBE in comparison to MRE as surveillance technique of the small-bowel in PJS patients, 2) to analyze patient burden and quality of life with DBE and MRE, and 3) to…
To evaluate the efficacy of ravulizumab compared with placebo to reduceproteinuria in adult participants with LN or IgAN.
To assess the efficacy of ravulizumab versus placebo in the treatment of adult and adolescent participants with HSCT-TMA.
- A. What are the SARS-CoV-2 viral load kinetics during and after treatment with neutralizing monoclonal antibodies and other antiviral agents against SARS-CoV-2?- B. What is the risk of SARS-CoV-2 infection after prophylactic use of monoclonal…
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…
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…
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).