3 results
The objective of this pivotal study is to assess the PK and PD as well as safety and tolerability of RP103 compared to Cystagon® in patients with nephropathic cystinosis. Results of this Phase 3 study will be used to support the registration…
To evaluate the efficacy of VX-661 in combination with ivacaftor through 24 weeks of treatment in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.
The primary objective of this study is to assess the efficacy, measured by OS, of capivasertib + docetaxel versus placebo + docetaxel, with both groups receiving continuous ADT. The primary endpoint is OS in patients who have mCRPC and have received…