Trial design
DISEASE PROGRESSION
This is a phase III, multicenter, randomized, double-blind, placebo-controlled, parallel-group study. Eligible patients are randomly assigned to one of two treatment arms: TUDCA or placebo. The treatment is administered by oral route, as an add-on to the standard therapy with riluzole, at the dose of 50 mg twice daily (100 mg daily).Patient randomization takes place following a lead-in period of 12 weeks (3 months), during which three assessments are conducted at 6-week intervals. Throughout the subsequent 18-month double-blind phase, clinical assessments are performed every 3 months. This design enables the measurement of disease progression both before and after the initiation of treatment (either active or placebo). The investigational medicinal product is administered orally at the dose of 1 g twice daily (2 g daily) for 18 months in addition to riluzole. Patients are included in the trial only after providing written informed consent. The trial is conducted in 25 clinical centers located in Belgium, France, Italy, Germany, Netherlands, Ireland, and the UK.