4 results
Comparison of steady-state cysteamine-trough WBC cystine levels between Cystagon® and RP103 over 3 months for each treatment period.
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: 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,…
Olaparib + AZD1775 - this treatment arm was closed.Study objectives are defined for the following patient populations:* *Breast cancer susceptible gene mutation (BRCAm)* = patients from stratum A· *Homologous Recombination Repair gene mutation (HRRm…