Amyotrophic Lateral SclerosisPhase 2/3NCT04082832
CuATSM Compared With Placebo for Treatment of ALS/MND
A Phase 2/3 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
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. 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
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 80 participants
- Where
- 1 siteAustralia
- Study dates
- 30 Sep 2019to 30 Dec 2020
- Phase
- Phase 2/3
- 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
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
Australia1 site, 1 recruiting
- Recruiting
Macquarie University
Macquarie, New South Wales
Dominic Rowe, MD
About this study
Official title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study of Cu(II)ATSM in Patients With Amyotrophic Lateral Sclerosis/Motor Neuron Disease
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.
Read the 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.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Cu(II)ATSM, Placebos
- 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
oral suspension
PlacebosDrug
oral suspension
| Study group | Role | What they receive |
|---|---|---|
| Cu(II)ATSM | Active comparator | Cu(II)ATSM Powder for Oral Suspension, 36 mg, to be reconstituted with Diluent (15 mL of sugar-free flavored pharmaceutical syrup) to provide an oral suspension for immediate consumption. Specified dose is 72 mg (2 bottles) taken fasting, before breakfast each day. Receives: Drug: Cu(II)ATSM |
| Placebo Powder for Oral Suspension | Placebo comparator | Placebo Powder for Oral Suspension, to be reconstituted with Diluent (15 mL of sugar-free flavored pharmaceutical syrup) to provide an oral suspension for immediate consumption. Specified dose is 72 mg (2 bottles) taken fasting, before breakfast each day. Receives: Drug: Placebos |
What is being measured
The primary outcome is the main question the study is designed to answer.
Revised ALS Functional Rating Scale (ALSFRS-R) total score (range: 48 [best] to 0 [worst])
assessment of disease severity
Measured: 24 weeks
Edinburgh Cognitive and Behavioral Amyotrophic Lateral Sclerosis Screen (ECAS) total score (range: 136 [best] to 0 [worst])
assessment of cognitive function
Measured: 24 weeks
Secondary outcomes (2)
seated slow vital capacity (SVC)
assessment of respiratory function
Measured: 24 weeks
rate of adverse events
tolerability assessment
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2019
- Study start30 Sep 2019
- Primary completion30 Dec 2020 (estimated)
- Study completion30 Dec 2020 (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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- ANCOVA will be used to compare efficacy endpoints between CuATSM and placebo groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 80 participants (estimated)
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
- NCT04082832
- Sponsor's protocol ID
- CMD-2019-001
- Status last verified
- Nov 2019
- First submitted
- 2 Sep 2019
- Last updated
- 6 Nov 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study of Cu(II)ATSM in Patients With Amyotrophic Lateral Sclerosis/Motor Neuron Disease
- ClinicalTrials.gov ID
- NCT04082832
- Acronym / sponsor ID
- CMD-2019-001
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- Collaborative Medicinal Development Pty LimitedIndustry (pharma, biotech, devices)
- Study start
- 30 Sep 2019 (actual)
- Primary completion
- 30 Dec 2020 (estimated)
- Study completion
- 30 Dec 2020 (estimated)
- First posted
- 9 Sep 2019 (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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- ANCOVA will be used to compare efficacy endpoints between CuATSM and placebo groups
| Arm | Type | Receives |
|---|---|---|
| Cu(II)ATSM | Active comparator | Drug: Cu(II)ATSMCu(II)ATSM Powder for Oral Suspension, 36 mg, to be reconstituted with Diluent (15 mL of sugar-free flavored pharmaceutical syrup) to provide an oral suspension for immediate consumption. Specified dose is 72 mg (2 bottles) taken fasting, before breakfast each day. |
| Placebo Powder for Oral Suspension | Placebo comparator | Drug: PlacebosPlacebo Powder for Oral Suspension, to be reconstituted with Diluent (15 mL of sugar-free flavored pharmaceutical syrup) to provide an oral suspension for immediate consumption. Specified dose is 72 mg (2 bottles) taken fasting, before breakfast each day. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (10 listed)
- 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
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)ATSMoral suspension | Drug | Cu(II)ATSM |
| Placebosoral suspension | Drug | Placebo Powder for Oral Suspension |
Primary outcome
Revised ALS Functional Rating Scale (ALSFRS-R) total score (range: 48 [best] to 0 [worst])
Time frame: 24 weeks
assessment of disease severity
Edinburgh Cognitive and Behavioral Amyotrophic Lateral Sclerosis Screen (ECAS) total score (range: 136 [best] to 0 [worst])
Time frame: 24 weeks
assessment of cognitive function
2 secondary outcomes
seated slow vital capacity (SVC)
Time frame: 24 weeks
rate of adverse events
Time frame: 24 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Nov 2019.
- Individual participant dataSharing planned: No
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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 2/3 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 7 inclusion and 10 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 6 Nov 2019. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Contact Kay Noel, PhD ((415) 444 9600, Kay.Noel@colMedDev.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Revised ALS Functional Rating Scale (ALSFRS-R) total score (range: 48 [best] to 0 [worst]) (time frame: 24 weeks). The study lists 2 primary outcomes in all. 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 6 Nov 2019.