5 results
Supported by the observation from the Phase I studies in healthy volunteers, doses within a range of 50 to 500 mg are safe and well tolerated. This Phase II trial will be performed to:a. obtain proof of concept of BI 44370 TAb. perform dose finding…
We propose a phase II study (intervention) in patients with severe generalized recessive dystrophic EB receiving reduced toxicity conditioning chemotherapy followe by cord blood transplantation with co-infusion of mesenchymal stromal cell units.…
Main objective:The main objective is to evaluate the efficacy of two intensified consolidation strategies in very-high risk neuroblastoma (VHR-NBL) patients in terms of event-free survival from randomisation date. Thisevaluation will follow a…
Objectives The main objective of the FAIR-PARK II trial is to demonstrate an effect of DFP on the course of PD (including both disease-modifying and symptomatic effects). The trial's overall objective can be summarized as follows: to…
Studying the effect of deferiprone on the motor function of patients with Pelizaeus-Merzbacher disease.