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

• Status: Completed
• Trial end date: 2020-01-30
• Target enrollment: 0
• Participant gender: All

Summary

Multicenter, open-label , single and multiple dose-escalation and pharmacokinetic study

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Collaborative Medicinal Development Pty Limited
• Treatments: Copper

Criteria

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