Treatment extension study for ALS/MND patients who participated in phase 1 study CMD-2016-001, completed assessments following six 28-day cycles of treatment, and whom the Investigator considers would benefit from continued CuATSM treatment.
Treatment extension study for ALS/MND patients who participated in phase 1 study CMD-2016-001, completed assessments following six 28-day cycles of treatment, and whom the Investigator considers would benefit from continued CuATSM treatment. The same safety assessments as in the CMD-2016-001 study will be conducted after each cycle of treatment and the same efficacy assessments as in the CMD-2016-001 study will be conducted after every 3 cycles of treatment. Treatment will continue until the first to occur of (1) Investigator considers the patient is no longer deriving benefit from CuATSM treatment, (2) patient develops dependence on mechanical ventilation where dependence is defined as being unable to lie flat (supine) without it, unable to sleep without it, or daytime use, or (3) the patient withdraws consent.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
28
copper-containing synthetic small molecule
Macquarie University
Sydney, New South Wales, Australia
Tolerance of extended treatment
safety
Time frame: 24 months
Treatment-related change in disease severity by ALS Functional Rating Scale - Revised (ALSFRS-R)
efficacy
Time frame: 24 months
Treatment-related change in cognitive function by Edinburgh Cognitive and Behavioral Assessment (ECAS) score
efficacy
Time frame: 24 months
Treatment-related change in respiratory function by seated forced vital capacity (FVC)
efficacy
Time frame: 24 months
Treatment-related change in quality of life by ALSSQOL-R score
efficacy
Time frame: 24 months
Treatment-related change in respiratory function by sniff nasal pressure (SNP) test
efficacy
Time frame: 24 months
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