18 results
Main Objective:Assess the efficacy of eculizumab as compared with placebo in the treatment of refractory gMG based on the improvement in the MG specific Activities of Daily Living profile (MG-ADL).Secondary Objectives: - Safety and tolerability of…
The objective of the study is to evaluate the safety and efficacy of eculizumab to prevent AMR in sensitized recipients of living donor kidney transplants requiring desensitization therapy.
Primary: to evaluate and compare the efficacy of:1) PDL treatment followed by topical sirolimus application after Er:Yag laser ablation of the stratum corneum in PWS 2) PDL treatment followed by topical sirolimus application without Er:Yag laser…
In our study we aim to find proof-of-concept that influencing the underlying protein dysfunction can result in clinically relevant improvement of epilepsy.
To investigate the safety and efficacy of a programmed introduction of a sirolimus based calcineurin inhibitor free maintenance immunosuppressive regime three months after renal transplantation in recipients of a non-heart-beating donor kidney graft…
The primary objective of the trial is to study the efficacccy of sirolimus in paraneoplastisch neurological syndromes associated with anti-Hu antibodies (Hu-PNS)Secondary objectives are to correlate clinical improvement with anti-Hu antibody titers…
Primary:To identify the recommended dose of sorafenib and of sirolimus for combination therapy in subsequent phase II trials Secondary:1. to determine the safety profile of the combination therapy of sorafenib with sirolimus.2. to determine, if…
The primary objective of this study is to assess the noninferiority of ALXN1210 compared to eculizumab in adult patients with PNH who have never been treated with a complement inhibitor. Noninferiority will be claimed if after 26 weeks of treatment…
The aim of our study is to investigate the effect of sirolimus on the progression of intestinal adenomas in patients with FAP and to assess the safety of this treatment.
The primary objectives of this study are to establish the efficacy and safety of APL-2 compared to eculizumab in patients with PNH who continue to have Hb levels <10.5 g/dL despite treatment with eculizumab.
This study aims to investigate the biological efficacy and safety of eculizumab in patients with aneurysmal SAH.
To evaluate the safety and tolerability of crovalimab compared witheculizumab
The primary objectiveTo evaluate the efficacy of eculizumab in the treatment of pediatric refractory generalized myasthenia gravis (gMG) based on change from Baseline in the Quantitative Myasthenia Gravis score for disease severity (QMG).The…
Primary Objective: The primary objective for this study is to evaluate the efficacy of ABP 959 compared with that of eculizumab based on control of intravascular hemolysis.Secondary Objective: The secondary objective is to assess the safety,…
To evaluate the efficacy of crovalimab compared to eculizumab
Efficacy of ALXN1210Sub-study: to Evaluate Patient Preference for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)
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…