3 results
The overall objective of this study is to evaluate the efficacy and safety of neoGAA in treatment-naïve patients with LOPD as compared to alglucosidase alfa, when this is administered biweekly for a period of 49 weeks. Also, there is an open-label…
The primary outcome parameter is the Gout Activity Score (GAS)after 6 months of rasburicase treatment. Secondary outcome parameters are the serum uric acid concentration, the clinical evaluation of tophus load, patient reported outcomes: MD-HAQ, GAQ…
ObjectivesPrimary · In Part 1 of the study is to determine the maximum tolerated doses (MTDs) and/or recommended Phase 2 dose (RP2D) of CC-220 as monotherapy (MonoT), in combination with dexamethasone (DEX) (DoubleT), in combination with DEX and…