4 results
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…
Primary objective: To determine the reduction of number of patients who receive blood transfusion perioperatively in the group pre-treated with erythropoietin and iron supplement compared to the control patients.
Obtain safety and efficacy data for the investigational drug AMB-05X in the treatment of tenosynovial giant cell tumor (TGCT)
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).