6 results
Evaluation of the effect of salvage therapy with R-DHAP followed by reduced-intensity conditioning and allogeneic stem cell transplantation from a sibling or unrelated donor
The purpose of this study is to compare the effect of AT1001 (migalastat hydrochloride) versus placebo on GL-3 in the kidney.
The primary objectives of this study are: * To characterize the effects of 150 mg and 450 mg of AT1001 administered 2 hours before administration of agalsidase on the safety and plasma pharmacokinetics of agalsidase in subjects with Fabry Disease*…
To determine the dose-response relationship of VAY736 for key efficacy and safety parameters.
Part 1 - To determine effects of different ianalumab doses on ALT normalization at Week 24 in patients with AIH who are incomplete responders or intolerant to standard therapy.Part 2 - To confirm the efficacy (biochemical and histological remission…
The purpose of this study is to assess the efficacy and safety of ianalumab (VAY736) compared to placebo in addition to second-line eltrombopag in adults with primary immune thrombocytopenia