Phase 1 Dose Escalation and PK Study of Cu(II)ATSM in ALS/MND

Inclusion Criteria: * Signed informed consent prior to initiation of any study-specific procedures; * Familial or sporadic ALS/MND defined as clinically possible, probable, or definite by Awaji-shima Consensus Recommendations; * First ALS/MND symptoms occurred no more than 2 years prior to screening visit; * Seated FVC ≥ 70% and SNP ≥ 50% of predicted value; * Not taking riluzole or on a stable dose of riluzole for at least 4 weeks prior to screening visit (participants are not allowed to start taking riluzole during the study); * Age between 18 and 75 years at time of informed consent; * Patient has a competent caregiver who can and will be responsible for administration of study drug; * Adequate bone marrow reserve, renal and liver function: * absolute neutrophil count ≥ 1500/µL * lymphocyte count \< 48% * platelet count ≥ 150,000/µL * hemoglobin ≥ 11 g/dL * creatinine clearance ≥ 60 mL/min (Cockroft \& Gault formula) * ALT and/or AST ≤ 2 x ULN * total bilirubin ≤ 1.5 x ULN * serum albumin ≥ 2.8 g/dL * Women and men with partners of childbearing potential must take effective contraception while on study and women of childbearing potential must have a negative pregnancy test and be non-lactating at screening Exclusion Criteria: * Inability to swallow oral medications or presence of GI disorder deemed to jeopardize intestinal absorption of Cu(II)ATSM * Dependence of mechanical ventilation (non-invasive or invasive) for any part of day or night * Exposure t…