8 results
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 test whether the proportion of patients in remission during the last 6 months of follow up is higher for a T2T strategy compared to a T2S stop strategy.
To evaluate the long-term safety and tolerability of elexacaftor(ELX)/tezacaftor (TEZ)/ivacaftor (IVA)
To evaluate the efficacy and pharmacodynamics (PD) of ELX/TEZ/IVA
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…
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
Primair eindpunt:Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis (CF) Who Are Homozygous or Heterozygous for the F508del Mutation.Secundair eindpunt:Evaluate the long-term efficacy of VX-…
The GO TEST OVERTURE study aims to demonstrate the superiority of the Treat to Target management strategy over a Treat to Avoid Symptoms approach in terms of clinical remission of gout symptoms.