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
To measure the effectiveness of intrathecal methylprednisolone and lidocaine on reducing PHN. Measurement of mthylprednisolone concentrations in cerebrospinal fluid (Part I only).