ALS Treatment Extension Study

Study Overview
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Brief Summary
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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
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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.

Official Title
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A Treatment Continuation Study for Patients With Amyotrophic Lateral Sclerosis/Motor Neuron Disease Who Have Successfully Completed Study CMD-2016-001

Conditions
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Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Drug: Cu(II)ATSM


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Multicenter, open-label treatment extension study
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cu(II)ATSM<br>Cu(II)ATSM administered once daily | Drug: Cu(II)ATSM<br>* copper-containing synthetic small molecule<br>* Other names: diacetylbis(N(4)-methylthiosemicarbazonato) copper(II);|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Tolerance of extended treatment | safety | 24 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Treatment-related change in disease severity by ALS Functional Rating Scale - Revised (ALSFRS-R) | efficacy | 24 months | 
| Treatment-related change in cognitive function by Edinburgh Cognitive and Behavioral Assessment (ECAS) score | efficacy | 24 months | 
| Treatment-related change in respiratory function by seated forced vital capacity (FVC) | efficacy | 24 months | 
| Treatment-related change in quality of life by ALSSQOL-R score | efficacy | 24 months | 
| Treatment-related change in respiratory function by sniff nasal pressure (SNP) test | efficacy | 24 months |