5 results
Rationale:The aim of this study is to investigate the pharmacodynamics (PD), pharmacokinetics (PK), safety, tolerability, immunogenicity, and clinical efficacy of efgartigimod coformulated with recombinant human hyaluronidase PH20 (rHuPH20)…
Primary objective- To evaluate the effects of ACT-541468 on objective simulated driving performance, i.e., the standard deviation of the lateral position (SDLP), after single- and multiple dose administrations (i.e., on Day 1 and Day 4) in the…
The primary objective of the randomized part of the protocol is to investigate if a conditioning regimen containing one alkylator (Bu) combined with two antimetabolites (Clo and Flu) results in superior 2-year acute grade III to IV-free, chronic non…
Primary:To evaluate the long-term safety and tolerability of efgartigimod PH20 SC in participants with Generalized Myasthenia Gravis (gMG).Secondary:• To evaluate the impact of efgartigimod PH20 SC on disease severity• To evaluate the effect of…
Stratum 1:To show that a non total body irradiation (TBI) containing conditioning (Flu/Thio/ivBu) results in a non inferior survival as compared to conditioning with TBI/Etoposide in children older than 4 years after HSCT from a Human leucocyte…