Usnoflast Neuromuscular Investigation for Treatment Efficacy in Amyotrophic Lateral Sclerosis
Diagnosis of probable or definite Amyotrophic lateral sclerosis, according to the revised version of the El Escorial World Federation of Neurology criteria
Time since onset of first symptom of Amyotrophic lateral sclerosis ≤24 months. Date of Amyotrophic lateral sclerosis symptom onset. For the purposes of this study, the date of symptom onset will be defined as the date the subject first had symptoms of their disease, i.e., limb weakness, dysarthria, dysphagia, shortness of breath, or fasciculations, from the screening visit.
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised score of ≥35 at screening.
Slow vital capacity: ≥60% of predicted capacity at the screening visit.
Presence of unstable psychiatric disease, cognitive impairment, dementia, or substance abuse that would impair the ability of the subject to provide informed consent, in the opinion of the investigator.
Serious illness (e.g., pneumonia, septicemia) within 4 weeks of the screening visit; infection requiring hospitalization or treatment with intravenous antibiotics, antivirals, or antifungals within 4 weeks of screening; chronic bacterial infection (such as tuberculosis) deemed unacceptable as per the judgment of the investigator.
Active herpes zoster infection within 2 months prior to the screening visit.
Any medical condition that promotes suicidal attempt or behavior within 6 months prior to the screening visit and in the opinion of the investigator might interfere with subject's participation in the study or is a risk for a suicide attempt.
Parallel
50 mg Usnoflast
Drug50 mg Usnoflast (50 mg Usnoflast capsules and matching placebo of 25 mg capsule)
75 mg Usnoflast
Drug75 mg Usnoflast (25 mg + 50 mg Usnoflast capsules)
Placebo
DrugMatching placebo of 25 mg and 50 mg
Efficacy of Usnoflast versus placebo assessed using the revised ALSFRS-R total score
Change in disease progression from baseline through Week 36 as measured by ALSFRS-R total score
Efficacy of Usnoflast versus placebo assessed using the survival
Change in disease progression from baseline through Week 36 as measured by survival Survival is defined based on the time of death or permanent-assisted ventilation (PAV). PAV is defined as the use of invasive or noninvasive mechanical ventilation for \>22 hours daily for \>7 consecutive days
Effect of Usnoflast versus placebo on survival in Open label extension phase
Time from baseline to the occurrence of death or Permanent-assisted ventilation (\>22 hours daily for \>7 days)
Number of participants with treatment emergent adverse events in open label extension
Time from baseline
Effect of Usnoflast versus placebo on survival
Time from baseline to the occurrence of death or Permanent-assisted ventilation (\>22 hours daily for \>7 days)
Evaluate the effect of Usnoflast versus placebo on Slow vital capacity
Change in Slow vital capacity from baseline to Week 36
Evaluate the effect of Usnoflast versus placebo on serum levels of Neurofilament light chain protein
Change in serum levels of Neurofilament light chain protein from baseline to Week 36
Evaluate the effect of Usnoflast versus placebo on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised total score and various functional items/domains of the ALSFRS-R total score
To determine the rate of disease progression using the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised total score and scores of various functional items/domains of the ALSFRS-R total score from baseline to Week 36
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