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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/NCT02870634
Registration number
NCT02870634
Ethics application status
Date submitted
10/08/2016
Date registered
17/08/2016
Date last updated
17/03/2020
Titles & IDs
Public title
Phase 1 Dose Escalation and PK Study of Cu(II)ATSM in ALS/MND
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Scientific title
A Phase 1 Single and Multiple Dose Escalation and Pharmacokinetic Study of Cu(II)ATSM Administered Orally to Patients With Amyotrophic Lateral Sclerosis/Motor Neuron Disease
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Secondary ID [1]
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CMD-2016-001
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Amyotrophic Lateral Sclerosis
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Motor Neuron Disease
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Condition category
Condition code
Neurological
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Neurodegenerative diseases
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Cu(II)ATSM
Experimental: Cu(II)ATSM - Cu(II)ATSM capsules, administered orally once daily
Treatment: Drugs: Cu(II)ATSM
copper-containing synthetic small molecule
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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recommended phase 2 dose as determined by the number of participants at each dose level with dose limiting toxicities
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Assessment method [1]
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Timepoint [1]
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24 months
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Secondary outcome [1]
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Treatment-related change in disease severity by ALS Functional Rating Scale - Revised (ALSFRS-R)
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Assessment method [1]
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Timepoint [1]
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24 months
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Secondary outcome [2]
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Treatment-related change in cognitive function by Edinburgh Cognitive and Behavioral Assessment (ECAS) score
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Assessment method [2]
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Timepoint [2]
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24 months
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Secondary outcome [3]
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Treatment-related change in respiratory function by seated forced vital capacity (FCV)
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Assessment method [3]
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Timepoint [3]
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24 months
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Secondary outcome [4]
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Treatment-related change in quality of life by ALSSQOL-R score
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Assessment method [4]
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Timepoint [4]
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24 months
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Secondary outcome [5]
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Treatment-related change in disease severity by transcranial magnetic stimulation (TMS) response
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Assessment method [5]
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Timepoint [5]
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24 months
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Secondary outcome [6]
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Peak Cu(II)ATSM plasma concentration following administration of a single dose based on blood draws taken at 1, 2, 4, 8 and 24 hours after dosing
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Assessment method [6]
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Timepoint [6]
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12 months
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Secondary outcome [7]
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Area under the Cu(II)ATSM plasma concentration versus time curve (AUC) following administration of a single dose based on blood draws taken at 1, 2, 4, 8 and 24 hours after dosing
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Assessment method [7]
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Timepoint [7]
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12 months
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Secondary outcome [8]
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Treatment-related change in respiratory function by sniff nasal pressure (SNP) test
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Assessment method [8]
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Timepoint [8]
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24 months
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Eligibility
Key inclusion criteria
- Signed informed consent prior to initiation of any study-specific procedures;
- Familial or sporadic ALS/MND defined as clinically possible, probable, or definite by
Awaji-shima Consensus Recommendations;
- First ALS/MND symptoms occurred no more than 2 years prior to screening visit;
- Seated FVC = 70% and SNP = 50% of predicted value;
- Not taking riluzole or on a stable dose of riluzole for at least 4 weeks prior to
screening visit (participants are not allowed to start taking riluzole during the
study);
- Age between 18 and 75 years at time of informed consent;
- Patient has a competent caregiver who can and will be responsible for administration
of study drug;
- Adequate bone marrow reserve, renal and liver function:
- absolute neutrophil count = 1500/µL
- lymphocyte count < 48%
- platelet count = 150,000/µL
- hemoglobin = 11 g/dL
- creatinine clearance = 60 mL/min (Cockroft & Gault formula)
- ALT and/or AST = 2 x ULN
- total bilirubin = 1.5 x ULN
- serum albumin = 2.8 g/dL
- Women and men with partners of childbearing potential must take effective
contraception while on study and women of childbearing potential must have a negative
pregnancy test and be non-lactating at screening
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Minimum age
18
Years
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Maximum age
75
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Inability to swallow oral medications or presence of GI disorder deemed to jeopardize
intestinal absorption of Cu(II)ATSM
- Dependence of mechanical ventilation (non-invasive or invasive) for any part of day or
night
- Exposure to any other investigational agent within 3 months or two investigational
agents within 6 months prior to screening visit
- Active GI disease (except gastrointestingal reflux disease) within 30 days of
screening visit
- Known immune compromising illness or treatment
- Presence of any of the following clinical conditions
- drug abuse or alcoholism
- unstable cardiac, pulmonary, renal, hepatic, endocrine or hematologic disorder
- active infectious disease
- AIDS or AIDS-related complex
- current malignancy
- unstable psychiatric illness, defined as psychosis or untreated major depression
within 90 days of screening visit
- neuromuscular disease other than ALS/MND
- Dementia that may affect either outcome measures or patient understanding and/or
compliance with study requirements and procedures
- Use of anticoagulants at therapeutic doses within 7 days prior to screening visit
- Current use of strong inducers or inhibitors of CYPs 2C19 and 2D6
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 1
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
16/11/2016
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
30/01/2020
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Sample size
Target
50
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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Macquarie University - Sydenham
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Recruitment hospital [2]
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Calvary Health Care Bethlehem - Caulfield
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Recruitment postcode(s) [1]
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2109 - Sydenham
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Recruitment postcode(s) [2]
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3162 - Caulfield
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Collaborative Medicinal Development Pty Limited
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
Multicenter, open-label , single and multiple dose-escalation and pharmacokinetic study
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02870634
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Dominic Rowe, MD
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Address
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Macquarie University
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02870634
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