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 WMOCompleted
Comparison of steady-state cysteamine-trough WBC cystine levels between Cystagon® and RP103 over 3 months for each treatment period.