7 results
Stratum 1:To show that a non total body irradiation (TBI) containing conditioning (Flu/Thio/ivBu) results in a non inferior survival as compared to conditioning with TBI/Etoposide in children older than 4 years after HSCT from a Human leucocyte…
To assess the efficacy and safety of 2 dose levels of NTRA-2112 on intestinal malabsorption in preterm infants as compared to placebo.The sub-study aims to determine whether NTRA-2112 in own mother's milk, donor human milk or preterm formula…
This is an open, non comparative, multicentre phase II trial, to evaluate the efficacy and feasibility of a newsequential combination of HD-MTX-AraC-based chemoimmunotherapy, followed by R-ICE regimen, and byhigh-dose chemotherapy supported by ASCT.
To evaluate the long-term safety and tolerability of elexacaftor(ELX)/tezacaftor (TEZ)/ivacaftor (IVA)
To evaluate the effect of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) on glucose tolerance in CF subjects with impaired glucose tolerance (IGT) or CF related diabetes (CFRD)
To evaluate the efficacy of VX-121/TEZ/D-IVA in CF subjects who are homozygous forF508del, heterozygous for F508del and a gating (F/G) or residual function (F/RF) mutation, orhave at least 1 other TCR CFTR mutation and no F508del mutation
To evaluate the efficacy and pharmacodynamics (PD) of ELX/TEZ/IVA