3 results
Approved WMOCompleted
Comparison of steady-state cysteamine-trough WBC cystine levels between Cystagon® and RP103 over 3 months for each treatment period.
Approved WMOCompleted
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…
Approved WMOCompleted
Primary: To evaluate the efficacy of tisagenlecleucel therapy as measured by overall response rate by investigator assessment.Secondary: Duration of response, event free survival, relapse free survival, overall survival, safety, kinetics,…