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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02870634
• Date of registration: 10/08/2016
• Prospective Registration: Yes
• Primary sponsor: Collaborative Medicinal Development Pty Limited
• Public title: Phase 1 Dose Escalation and PK Study of Cu(II)ATSM in ALS/MND
• Scientific title: A Phase 1 Single and Multiple Dose Escalation and Pharmacokinetic Study of Cu(II)ATSM Administered Orally to Patients With Amyotrophic Lateral Sclerosis/Motor Neuron Disease
• Date of first enrolment: November 16, 2016
• Target sample size: 50
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02870634
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

Australia

Contacts

• Name: Dominic Rowe, MD
• Affiliation: Macquarie University

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Motor Neuron Disease

Intervention(s)

Drug: Cu(II)ATSM

Primary Outcome(s)

recommended phase 2 dose as determined by the number of participants at each dose level with dose limiting toxicities [Time Frame: 24 months]

Secondary Outcome(s)

Peak Cu(II)ATSM plasma concentration following administration of a single dose based on blood draws taken at 1, 2, 4, 8 and 24 hours after dosing [Time Frame: 12 months]

Treatment-related change in disease severity by ALS Functional Rating Scale - Revised (ALSFRS-R) [Time Frame: 24 months]

Area under the Cu(II)ATSM plasma concentration versus time curve (AUC) following administration of a single dose based on blood draws taken at 1, 2, 4, 8 and 24 hours after dosing [Time Frame: 12 months]

Treatment-related change in quality of life by ALSSQOL-R score [Time Frame: 24 months]

Treatment-related change in respiratory function by seated forced vital capacity (FCV) [Time Frame: 24 months]

Treatment-related change in disease severity by transcranial magnetic stimulation (TMS) response [Time Frame: 24 months]

Treatment-related change in cognitive function by Edinburgh Cognitive and Behavioral Assessment (ECAS) score [Time Frame: 24 months]

Treatment-related change in respiratory function by sniff nasal pressure (SNP) test [Time Frame: 24 months]

Secondary ID(s)

CMD-2016-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors