6 results
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*…
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
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…
To determine the dose-response relationship of VAY736 for key efficacy and safety parameters.
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