3 results
Objectives:Primary Objectives:1. To evaluate the efficacy of BAN2401 compared to placebo by establishing the ED90 (as defined in theprotocol) for BAN2401 on the derived Composite Clinical Score at 12 months of treatment in subjects withEarly…
The primary endpoint is overall survival (OS) in patients with low hENT1 expression. Ifthe hENT1 expression data are available for analysis prior to randomizing 250 patientsthen an interim analysis for sample size re-estimation may be performed.
- To assess the pharmacokinetic profile of cysteamine base after single dose administration of the new sustained-release cysteamine bitartrate (PO-001) to healthy volunteers, in comparison with Cystagon® and Procysbi®.- To collect safety/…