5 results
Comparison of steady-state cysteamine-trough WBC cystine levels between Cystagon® and RP103 over 3 months for each treatment period.
To determine the antileukemic activity of combination chemotherapy including bortezomib as reinduction therapy in childhood relapsed/refractory ALL.
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…
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…
Primary objectives: Improvement of CR2 rates after induction with ALL R3 with bortezomib versus withoutbortezomib in HR relapsed ALL patientsSecondary objectives:Improvement of Event Free Survival (EFS) and overall survival (OS) ratesImprovement of…