Study objectives
ALS
DISEASE PROGRESSION, SECONDARY
The primary objective of the TUDCA-ALS study is to assess the efficacy of TUDCA, as an add-on therapy to riluzole, in slowing down the progression of ALS throughout the 18-month treatment period, compared to the lead-in phase. The secondary objectives of this study are as follows: (1) to assess the efficacy of TUDCA in slowing disease progression and functional impairment in patients with ALS, as measured by the survival time, the ALSFR-R, the ALS Assessment Questionnaire-40 (ALSAQ-40) and EuroQol 5-Dimension 5L questionnaire (EQ-5D-5L), forced vital capacity (FVC), and the Medical Research Council (MRC) sum-score for muscle force, and (2) to evaluate the long-term safety and tolerability of TUDCA.The exploratory objective of this study is to investigate the effect of TUDCA on biomarkers associated with disease progression, such as neurofilaments in cerebrospinal (CSF) fluid and serum, serum expression of matrix metalloproteinase-9 (MMP-9) in serum, and plasma creatinine levels.This document describes in detail the predetermined statistical analysis plan (SAP) for the TUDCA-ALS trial.