CuATSM Compared With Placebo for Treatment of ALS/MND

Patients will be randomized 1:1 to CuATSM or placebo for 6 x 28-day cycles (24 weeks) of treatment. Study drug is administered orally, once a day in fasted state (before breakfast). Assessments for safety (physical examination, vital signs, hematology, serum chemistry adverse events) will be conducted at baseline and following each cycle of treatment. Assessments for efficacy (Revised ALS Functional Rating Scale [ASLFRS-R] score, and Edinburgh Cognitive and Behavioral Amyotrophic Lateral Sclerosis Screen [ECAS] score, and seated slow vital capacity [SVC]) will be conducted at baseline and following 2, 4 and 6 cycles of treatment. Analysis of covariance (ANCOVA) will be used to compare efficacy endpoints between CuATSM and placebo groups.

Inclusion Criteria:
- signed informed consent
- familial or sporadic ALS/MNS by Awaji-shima Consensus Recommendations
- not taking riluzole or on stable dose of riluzole for 4 weeks prior to screening visit
- no prior exposure to agents other than riluzole for treatment of ALS
- adequate bone marrow reserve, renal and liver function
- women of childbearing potential must have a negative pregnancy test and be
non-lactating
- women and men with partners of childbearing potential must take effective
contraception while on treatment
Exclusion Criteria:
- presence of a gastrointestinal disorder (eg, malabsorption) that might jeopardize
intestinal absorption of study drug
- inability to perform seated SVC
- known immune compromising illness or treatment
- drug abuse or alcoholism
- clinically significant or active cardiovascular disease
- acute or chronic infection
- diagnosis of malignancy within 2 years prior to screening
- dementia that may affect patient understanding and/or compliance with study
requirements and procedures
- current use of strong inducers or inhibitors of CYPs 2C19 and 2D6
- current us of medications (other than riluzole) that are metabolized predominantly by
CYP 1A2