4 results
MAIN OBJECTIVEDose escalation phase :- To determine the MTD and/or RDE of alpelisib (BYL719) in combination with everolimus, and the MTD and/or RDE of alpelisib in combination with everolimus and exemestaneDose expansion part:- To describe safety…
To evaluate the safety and efficacy of PRX-102 compared to agalsidase beta in Fabry disease patients with impaired renal function.
To evaluate the safety and efficacy of PRX-102 in patients with Fabry disease currently treated with agalsidase alfa
To evaluate the ongoing safety, tolerability, and efficacy parameters of pegunigalsidase alfa in adult Fabry patients who have successfully completed studies PB-102-F20 and PB-102-F30, or completed at least 48 months in study PB-102-F03.