ALS Treatment Extension Study
Primary Purpose
Amyotrophic Lateral Sclerosis
Status
Active
Phase
Phase 1
Locations
Australia
Study Type
Interventional
Intervention
Cu(II)ATSM
Sponsored by
About this trial
This is an interventional treatment trial for Amyotrophic Lateral Sclerosis
Eligibility 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
Sites / Locations
- Macquarie University
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Cu(II)ATSM
Arm Description
Cu(II)ATSM administered once daily
Outcomes
Primary Outcome Measures
Tolerance of extended treatment
safety
Secondary Outcome Measures
Treatment-related change in disease severity by ALS Functional Rating Scale - Revised (ALSFRS-R)
efficacy
Treatment-related change in cognitive function by Edinburgh Cognitive and Behavioral Assessment (ECAS) score
efficacy
Treatment-related change in respiratory function by seated forced vital capacity (FVC)
efficacy
Treatment-related change in quality of life by ALSSQOL-R score
efficacy
Treatment-related change in respiratory function by sniff nasal pressure (SNP) test
efficacy
Full Information
NCT ID
NCT03136809
First Posted
April 28, 2017
Last Updated
February 15, 2022
Sponsor
Collaborative Medicinal Development Pty Limited
1. Study Identification
Unique Protocol Identification Number
NCT03136809
Brief Title
ALS Treatment Extension Study
Official Title
A Treatment Continuation Study for Patients With Amyotrophic Lateral Sclerosis/Motor Neuron Disease Who Have Successfully Completed Study CMD-2016-001
Study Type
Interventional
2. Study Status
Record Verification Date
February 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
January 18, 2018 (Actual)
Primary Completion Date
December 31, 2022 (Anticipated)
Study Completion Date
December 31, 2022 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Collaborative Medicinal Development Pty Limited
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Model Description
Multicenter, open-label treatment extension study
Masking
None (Open Label)
Allocation
N/A
Enrollment
28 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Cu(II)ATSM
Arm Type
Experimental
Arm Description
Cu(II)ATSM administered once daily
Intervention Type
Drug
Intervention Name(s)
Cu(II)ATSM
Other Intervention Name(s)
diacetylbis(N(4)-methylthiosemicarbazonato) copper(II)
Intervention Description
copper-containing synthetic small molecule
Primary Outcome Measure Information:
Title
Tolerance of extended treatment
Description
safety
Time Frame
24 months
Secondary Outcome Measure Information:
Title
Treatment-related change in disease severity by ALS Functional Rating Scale - Revised (ALSFRS-R)
Description
efficacy
Time Frame
24 months
Title
Treatment-related change in cognitive function by Edinburgh Cognitive and Behavioral Assessment (ECAS) score
Description
efficacy
Time Frame
24 months
Title
Treatment-related change in respiratory function by seated forced vital capacity (FVC)
Description
efficacy
Time Frame
24 months
Title
Treatment-related change in quality of life by ALSSQOL-R score
Description
efficacy
Time Frame
24 months
Title
Treatment-related change in respiratory function by sniff nasal pressure (SNP) test
Description
efficacy
Time Frame
24 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Dominic Rowe, MD
Organizational Affiliation
Macquarie University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Macquarie University
City
Sydney
State/Province
New South Wales
ZIP/Postal Code
2109
Country
Australia
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
Plan to publish study results in a peer-reviewed journal
Learn more about this trial
ALS Treatment Extension Study
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