7 results
Primary:* To evalaute the efficacy of three doses of norUDCA vs. placebo for the treatment of PSC* To identify efficacious norUDCA dose(s) for the treatment of PSC for further evaluation in phase III. Secondary:* To study safety and tolerability (…
1) To assess whether galactosemia women with ovarian failure are able to produce estradiol after exogenous (normally bioactive) FSH and LH gift 2) To characterize FSH glycan structures
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)
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-…
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 long-term safety and tolerability of elexacaftor(ELX)/tezacaftor (TEZ)/ivacaftor (IVA)
To evaluate the efficacy and pharmacodynamics (PD) of ELX/TEZ/IVA