5 results
Primary Objective:To evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation (F/F)Secondary Objectives:* To evaluate the…
Primary Objective:To evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) andivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and aminimal function mutation (F/MF subjects)Secondary…
To assess the performance of the EndoRotor, as compared to conventional endoscopic techniques, for direct endoscopic necrosectomy (DEN) of walled off necrosis (WON)
Primary objectives: 1. To compare the efficacy of vamorolone administered orally at daily doses of 6.0 mg/kg over a 24-week treatment period vs. placebo in ambulant boys ages 4 to <7 years with DMD; and2. To evaluate the safety and…
The study is divided in two phases.The primary objective of phase I of the study is to evaluate the safety and toxicity of the infusion of ex vivo expanded RNK001 NK cells, both with and without sc IL-2 following immunosuppressive conditioning…