Amyotrophic Lateral Sclerosis
Conditions
Brief summary
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.
Detailed description
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.
Interventions
DRUGCu(II)ATSM
copper-containing synthetic small molecule
Sponsors
Collaborative Medicinal Development Pty Limited
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Multicenter, open-label treatment extension study
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Tolerance of extended treatment | 24 months | safety |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Treatment-related change in disease severity by ALS Functional Rating Scale - Revised (ALSFRS-R) | 24 months | efficacy |
| Treatment-related change in cognitive function by Edinburgh Cognitive and Behavioral Assessment (ECAS) score | 24 months | efficacy |
| Treatment-related change in respiratory function by seated forced vital capacity (FVC) | 24 months | efficacy |
| Treatment-related change in quality of life by ALSSQOL-R score | 24 months | efficacy |
| Treatment-related change in respiratory function by sniff nasal pressure (SNP) test | 24 months | efficacy |
Countries
Australia
Outcome results
None listed