2 results
Approved WMORecruiting
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…
Approved WMORecruiting
The scientific rationale for the study is summarized above and further detailed in Section 3 and the Investigator Brochure (IB). This Phase 2 randomized, double-blind clinical trial is designed to assess the safety and efficacy of BMS-986165 in…