CuATSM Compared With Placebo for Treatment of ALS/MND
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ClinicalTrials.gov Identifier: NCT04082832 |
Recruitment Status : Unknown
Verified November 2019 by Collaborative Medicinal Development Pty Limited.
Recruitment status was: Recruiting
First Posted : September 9, 2019
Last Update Posted : November 6, 2019
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Condition or disease | Intervention/treatment | Phase |
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Amyotrophic Lateral Sclerosis | Drug: Cu(II)ATSM Drug: Placebos | Phase 2 Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 80 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Intervention Model Description: | ANCOVA will be used to compare efficacy endpoints between CuATSM and placebo groups |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Masking Description: | placebo controlled |
Primary Purpose: | Treatment |
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 |
Actual Study Start Date : | September 30, 2019 |
Estimated Primary Completion Date : | December 30, 2020 |
Estimated Study Completion Date : | December 30, 2020 |

Arm | Intervention/treatment |
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Active Comparator: Cu(II)ATSM
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.
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Drug: Cu(II)ATSM
oral suspension |
Placebo Comparator: Placebo Powder for Oral Suspension
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.
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Drug: Placebos
oral suspension |
- 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
- seated slow vital capacity (SVC) [ Time Frame: 24 weeks ]assessment of respiratory function
- rate of adverse events [ Time Frame: 24 weeks ]tolerability assessment

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years to 75 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts 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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04082832
Contact: Kay Noel, PhD | (415) 444 9600 | Kay.Noel@colMedDev.com | |
Contact: Craig Rosenfeld, MD | (415) 444 9600 | CraigR@ColMedDev.com |
Australia, New South Wales | |
Macquarie University | Recruiting |
Macquarie, New South Wales, Australia | |
Contact: Dominic Rowe, MD |
Principal Investigator: | Dominic Rowe, MD | Macquarie University |
Responsible Party: | Collaborative Medicinal Development Pty Limited |
ClinicalTrials.gov Identifier: | NCT04082832 |
Other Study ID Numbers: |
CMD-2019-001 |
First Posted: | September 9, 2019 Key Record Dates |
Last Update Posted: | November 6, 2019 |
Last Verified: | November 2019 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Motor Neuron Disease Amyotrophic Lateral Sclerosis Sclerosis Pathologic Processes Neurodegenerative Diseases Nervous System Diseases |
Neuromuscular Diseases Spinal Cord Diseases Central Nervous System Diseases TDP-43 Proteinopathies Proteostasis Deficiencies Metabolic Diseases |