Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04082832




Registration number
NCT04082832
Ethics application status
Date submitted
2/09/2019
Date registered
9/09/2019
Date last updated
6/11/2019

Titles & IDs
Public title
CuATSM Compared With Placebo for Treatment of ALS/MND
Scientific title
A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study of Cu(II)ATSM in Patients With Amyotrophic Lateral Sclerosis/Motor Neuron Disease
Secondary ID [1] 0 0
CMD-2019-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Amyotrophic Lateral Sclerosis 0 0
Condition category
Condition code
Neurological 0 0 0 0
Neurodegenerative diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Cu(II)ATSM
Treatment: Drugs - Placebos

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.

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.


Treatment: Drugs: Cu(II)ATSM
oral suspension

Treatment: Drugs: Placebos
oral suspension
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Revised ALS Functional Rating Scale (ALSFRS-R) total score (range: 48 [best] to 0 [worst])
Timepoint [1] 0 0
24 weeks
Primary outcome [2] 0 0
Edinburgh Cognitive and Behavioral Amyotrophic Lateral Sclerosis Screen (ECAS) total score (range: 136 [best] to 0 [worst])
Timepoint [2] 0 0
24 weeks
Secondary outcome [1] 0 0
seated slow vital capacity (SVC)
Timepoint [1] 0 0
24 weeks
Secondary outcome [2] 0 0
rate of adverse events
Timepoint [2] 0 0
24 weeks

Eligibility
Key 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
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2/Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Unknown status
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/09/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/12/2020
Actual
Sample size
Target
80
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Macquarie University - Macquarie
Recruitment postcode(s) [1] 0 0
- Macquarie

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Collaborative Medicinal Development Pty Limited
Address
Country

Ethics approval
Ethics application status

Summary
Brief 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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04082832
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Dominic Rowe, MD
Address 0 0
Macquarie University
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Kay Noel, PhD
Address 0 0
Country 0 0
Phone 0 0
(415) 444 9600
Fax 0 0
Email 0 0
Kay.Noel@colMedDev.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04082832