3 results
Approved WMOPending
Primary Objective:To evaluate the efficacy of ELX/TEZ/IVA in subjects 6 through 11 years of age with CF, heterozygous for F508del and a MF mutation (F/MF)Secondary Objectives:• To evaluate the PD of ELX/TEZ/IVA• To evaluate the safety of ELX/TEZ/IVA
Approved WMOCompleted
The goal of this study is to investigate the efficacy and cost-utility of Oncokompas among patients with incurable cancer compared to care-as-usual. The main hypothesis is that use of Oncokompas will be superior to care as usual to improve patient…
Approved WMORecruiting
Assess the potential efficay of L-serine dietary supplementation in patients children with a GRIN2B LoS mutation.