15 results
Primary: To determine whether or not consistent exposures can be achieved in neonates and preterm infants with presumed GERD receiving oral doses of pantoprazole. Secondary: PK and PD assessment after single dose and steady state. Determination fo…
The primary objective of this study is to provide access to SPD503 following participation in SPD503-315 or SPD503-316. The primary outcome of this study is to evaluate the long-term safety of SPD503. The evaluation of safety will be based on the…
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.
The primary objective of this study is to evaluate the long-term maintenance of efficacy of SPD503 in childrenand adolescents (6-17 years) with attention-deficit/hyperactivity disorder (ADHD) who respond to an initial open-label,short-term treatment…
Efficacy of ALXN1210Sub-study: to Evaluate Patient Preference for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)
This study aims to investigate the biological efficacy and safety of eculizumab in patients with aneurysmal SAH.
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 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.
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 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…
To evaluate the efficacy of crovalimab compared to eculizumab
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 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,…