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 primary purpose of this study is to assess the dose response of several doses of tildacerfont in controlling hormone levels and reducing testicular tumors over 12 weeks by comparing the tildacerfont hormonal control response to a placebo, a *…