Amyotrophic Lateral SclerosisPhase 1/2NCT03136809
ALS Treatment Extension Study
A Phase 1/2 study testing Cu(II)ATSM in people with Amyotrophic Lateral Sclerosis, run by Collaborative Medicinal Development Pty Limited at 1 site in Australia.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Cu(II)ATSM
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 1 siteAustralia
- Study dates
- 18 Jan 2018to 31 Dec 2022
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Treatment Continuation Study for Patients With Amyotrophic Lateral Sclerosis/Motor Neuron Disease Who Have Successfully Completed Study CMD-2016-001
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.
Read the 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.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Cu(II)ATSM
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cu(II)ATSMDrug
Also called diacetylbis(N(4)-methylthiosemicarbazonato) copper(II)
copper-containing synthetic small molecule
| Study group | Role | What they receive |
|---|---|---|
| Cu(II)ATSM | Experimental | Cu(II)ATSM administered once daily Receives: Drug: Cu(II)ATSM |
What is being measured
The primary outcome is the main question the study is designed to answer.
Tolerance of extended treatment
safety
Measured: 24 months
Secondary outcomes (5)
Treatment-related change in disease severity by ALS Functional Rating Scale - Revised (ALSFRS-R)
efficacy
Measured: 24 months
Treatment-related change in cognitive function by Edinburgh Cognitive and Behavioral Assessment (ECAS) score
efficacy
Measured: 24 months
Treatment-related change in respiratory function by seated forced vital capacity (FVC)
efficacy
Measured: 24 months
Treatment-related change in quality of life by ALSSQOL-R score
efficacy
Measured: 24 months
Treatment-related change in respiratory function by sniff nasal pressure (SNP) test
efficacy
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 May 2017
- Study start18 Jan 2018
- Primary completion31 Dec 2022 (estimated)
- Study completion31 Dec 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Multicenter, open-label treatment extension study
- Masking
- None (open label)
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Collaborative Medicinal Development Pty Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Dominic Rowe, MD, Principal investigator, Macquarie University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03136809
- Sponsor's protocol ID
- CMD-2017-001
- Status last verified
- Feb 2022
- First submitted
- 28 Apr 2017
- Last updated
- 17 Feb 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Plan to publish study results in a peer-reviewed journal
Study at a glance
From the registry record, last updated by the sponsor on 17 Feb 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Treatment Continuation Study for Patients With Amyotrophic Lateral Sclerosis/Motor Neuron Disease Who Have Successfully Completed Study CMD-2016-001
- ClinicalTrials.gov ID
- NCT03136809
- Acronym / sponsor ID
- CMD-2017-001
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Collaborative Medicinal Development Pty LimitedIndustry (pharma, biotech, devices)
- Study start
- 18 Jan 2018 (actual)
- Primary completion
- 31 Dec 2022 (estimated)
- Study completion
- 31 Dec 2022 (estimated)
- First posted
- 2 May 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Multicenter, open-label treatment extension study
| Arm | Type | Receives |
|---|---|---|
| Cu(II)ATSM | Experimental | Drug: Cu(II)ATSMCu(II)ATSM administered once daily |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cu(II)ATSMAlso: diacetylbis(N(4)-methylthiosemicarbazonato) copper(II)copper-containing synthetic small molecule | Drug | Cu(II)ATSM |
Primary outcome
Tolerance of extended treatment
Time frame: 24 months
safety
5 secondary outcomes
Treatment-related change in disease severity by ALS Functional Rating Scale - Revised (ALSFRS-R)
Time frame: 24 months
Treatment-related change in cognitive function by Edinburgh Cognitive and Behavioral Assessment (ECAS) score
Time frame: 24 months
Treatment-related change in respiratory function by seated forced vital capacity (FVC)
Time frame: 24 months
Treatment-related change in quality of life by ALSSQOL-R score
Time frame: 24 months
Treatment-related change in respiratory function by sniff nasal pressure (SNP) test
Time frame: 24 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Feb 2022.
- Individual participant dataSharing planned: Yes. Plan to publish study results in a peer-reviewed journal
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Dominic Rowe, MD principal investigator
Macquarie University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Cu(II)ATSM in people with Amyotrophic Lateral Sclerosis, run by Collaborative Medicinal Development Pty Limited at 1 site in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 17 Feb 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Tolerance of extended treatment (time frame: 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Feb 2022.