ALS Treatment Extension Study

• Register: ClinicalTrials.gov
• Last refreshed on: 28 February 2022
• Main ID: NCT03136809
• Date of registration: 28/04/2017
• Prospective Registration: Yes
• Primary sponsor: Collaborative Medicinal Development Pty Limited
• Public title: ALS Treatment Extension Study
• Scientific title: A Treatment Continuation Study for Patients With Amyotrophic Lateral Sclerosis/Motor Neuron Disease Who Have Successfully Completed Study CMD-2016-001
• Date of first enrolment: January 18, 2018
• Target sample size: 28
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT03136809
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

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 and
treatment

- Documented completion of protocol-specific assessments following completion of six
28-day treatment cycles in study CMD-2016-001

- Principal Investigator considers the patient would benefit from continued treatment
with Cu(II)ATSM

- Not taking riluzole or on the same (or lower) dose used during the CMD-2016-001 study

- Adequate bone marrow reserve, renal and liver function

- Women and men with partners of childbearing potential must take effective
contraception while on study treatment

Exclusion Criteria:

- Inability to swallow oral medications or presence of a gastrointestinal disorder
(e.g.,malabsorption) deemed to jeopardize intestinal absorption of study drug

- Dependence on mechanical ventilation (invasive or non-invasive) for any part of day or
night, where dependence is defined as being unable to lie flat (supine) without it,
unable to sleep without it, or daytime use

- Dementia that may affect either outcome measures or patient understanding and/or
compliance with study requirements and procedures

- 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

Intervention(s)

Drug: Cu(II)ATSM

Primary Outcome(s)

Tolerance of extended treatment [Time Frame: 24 months]

Secondary Outcome(s)

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

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

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

Secondary ID(s)

CMD-2017-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors