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

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis Read More

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 to any other investigational agent within 3 months or two investigational agents within 6 months prior to screening visit
• Active GI disease (except gastrointestingal reflux disease) within 30 days of screening visit
• Known immune compromising illness or treatment

• Presence of any of the following clinical conditions

  • drug abuse or alcoholism
  • unstable cardiac, pulmonary, renal, hepatic, endocrine or hematologic disorder
  • active infectious disease
  • AIDS or AIDS-related complex
  • current malignancy
  • unstable psychiatric illness, defined as psychosis or untreated major depression within 90 days of screening visit
  • neuromuscular disease other than ALS/MND
• Dementia that may affect either outcome measures or patient understanding and/or compliance with study requirements and procedures
• Use of anticoagulants at therapeutic doses within 7 days prior to screening visit
• Current use of strong inducers or inhibitors of CYPs 2C19 and 2D6