Primary Outcomes

Secondary Outcomes

Primary Outcomes

Secondary Outcomes

Primary Outcomes

Description: Efficacy by percent change in biomarker in the CSF at week 12 from baseline: (a) Efficacy biomarkers (b) Target biomarkers (c) Efficacy/target biomarkers

Description: Safety: 1. adverse event frequency and severity, changes in vital signs, clinical laboratory values. 2. Serious adverse event frequency

Description: Tolerability: The ability to complete the first 2 weeks of active treatment in the study

Secondary Outcomes

Description: Progressive change in ALSFRS-R of each patient determined from the following data points:1) symptom onset, 2) baseline, 3) end of week 2 examination, 4) end of week 6 examination and 5) end of week 12 examination.

Description: Progressive change in Forced Vital Capacity (FVC) from the following data points: 1) symptom onset, 2) baseline, 3) end of week 2 examination, 4) end of week 6 examination and 5) end of week 12 examination.

Description: Progressive change in Forced Vital Capacity (FVC) from the following data points: 1) symptom onset, 2) baseline, 3) end of week 2 examination, 4) end of week 6 examination and 5) end of week 12 examination.

Description: Progressive muscle strength change measured by HHD (handheld dynamometry testing score) from the following data points: 1) symptom onset, 2) baseline, 3) end of week 2 examination, 4) end of week 6 examination and 5) end of week 12 examination.

Description: Percentage changes in Biomarkers in blood between baseline and 1) the end of week 1, 2) end of week 2, 3) end of week 6 and 4) end of week 12. Comparing the changes encompassing the entire cohort of 10 subjects.

Description: Report all death as mortality rate

Other Outcomes

Description: Secondary analyses may consider a comparison of slopes (change in the rate of decline) for any hint of disease modification using placebo outcomes in patients matched for baseline features from a large database of recent clinical trials by NEALS showing stable rates of decline as historical controls.

Description: Secondary analysis to allow a-priori stratification of patients by their symptoms if available predominantly lower motor neuron predominantly upper motor neuron predominantly bulbar

Primary Outcomes

Description: miRNA expression pattern in ALS patients compared to control patients.

Secondary Outcomes

Description: Evolution of miRNA expression level in blood and CSF of ALS patients

Description: Difference in diffusivity parameters of MRI between ALS subjects and control groups

Primary Outcomes

Description: To evaluate the effect of VPA+Li on progression of ALS by measuring functionnal changes from baseline in ALSFRS-R.

Secondary Outcomes

Description: Changes from baseline in ALSAQ-5, a brief self-administered quality of life scale.

Description: DTI biomarkers in corticospinal tract are measured in 6 regions bilaterally