Amyotrophic Lateral Sclerosis
Conditions
Brief summary
Multicenter, randomized, double-blind, placebo controlled study to assess the tolerabilty and efficacy of CuATSM in patients with ALS/MND. Patients will be randomized 1:1 to CuATSM or placebo for 6 x 28-day cycles (24 weeks) of treatment.
Detailed description
Patients will be randomized 1:1 to CuATSM or placebo for 6 x 28-day cycles (24 weeks) of treatment. Study drug is administered orally, once a day in fasted state (before breakfast). Assessments for safety (physical examination, vital signs, hematology, serum chemistry adverse events) will be conducted at baseline and following each cycle of treatment. Assessments for efficacy (Revised ALS Functional Rating Scale \[ASLFRS-R\] score, and Edinburgh Cognitive and Behavioral Amyotrophic Lateral Sclerosis Screen \[ECAS\] score, and seated slow vital capacity \[SVC\]) will be conducted at baseline and following 2, 4 and 6 cycles of treatment. Analysis of covariance (ANCOVA) will be used to compare efficacy endpoints between CuATSM and placebo groups.
Interventions
DRUGCu(II)ATSM
oral suspension
DRUGPlacebos
oral suspension
Sponsors
Collaborative Medicinal Development Pty Limited
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Masking description
placebo controlled
Intervention model description
ANCOVA will be used to compare efficacy endpoints between CuATSM and placebo groups
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* signed informed consent * familial or sporadic ALS/MNS by Awaji-shima Consensus Recommendations * not taking riluzole or on stable dose of riluzole for 4 weeks prior to screening visit * no prior exposure to agents other than riluzole for treatment of ALS * adequate bone marrow reserve, renal and liver function * women of childbearing potential must have a negative pregnancy test and be non-lactating * women and men with partners of childbearing potential must take effective contraception while on treatment
Exclusion criteria
* presence of a gastrointestinal disorder (eg, malabsorption) that might jeopardize intestinal absorption of study drug * inability to perform seated SVC * known immune compromising illness or treatment * drug abuse or alcoholism * clinically significant or active cardiovascular disease * acute or chronic infection * diagnosis of malignancy within 2 years prior to screening * dementia that may affect patient understanding and/or compliance with study requirements and procedures * current use of strong inducers or inhibitors of CYPs 2C19 and 2D6 * current us of medications (other than riluzole) that are metabolized predominantly by CYP 1A2
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Revised ALS Functional Rating Scale (ALSFRS-R) total score (range: 48 [best] to 0 [worst]) | 24 weeks | assessment of disease severity |
| Edinburgh Cognitive and Behavioral Amyotrophic Lateral Sclerosis Screen (ECAS) total score (range: 136 [best] to 0 [worst]) | 24 weeks | assessment of cognitive function |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| seated slow vital capacity (SVC) | 24 weeks | assessment of respiratory function |
| rate of adverse events | 24 weeks | tolerability assessment |
Countries
Australia
Contacts
Primary ContactKay Noel, PhD
Backup ContactCraig Rosenfeld, MD
Outcome results
None listed