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…
The objective of this pivotal study is to assess the long-term safety, tolerability, pharmacokinetics and pharmacodynamics of RP103 in pediatric and adult patients with nephropathic cystinosis. Results of this Phase 3 study will be used to support…
Primary:• To evaluate the clinical benefit rate (CBR) of ixabepilone Secondary:• To evaluated progression free survival (PFS)• To evaluate overall survival (OS)• To evaluate objective response rate (ORR) defined as Complete Response (CR) and Partial…