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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02870634




Trial ID
NCT02870634
Ethics application status
Date submitted
10/08/2016
Date registered
12/08/2016
Date last updated
6/03/2017

Titles & IDs
Public title
Phase 1 Dose Escalation and PK Study of Cu(II)ATSM in ALS/MND
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
Secondary ID [1] 0 0
CMD-2016-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Amyotrophic Lateral Sclerosis 0 0
Motor Neuron Disease 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

Experimental: Cu(II)ATSM - Cu(II)ATSM capsules, administered orally once daily


Treatment: Drugs: Cu(II)ATSM
copper-containing synthetic small molecule

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

Outcomes
Primary outcome [1] 0 0
recommended phase 2 dose as determined by the number of participants at each dose level with dose limiting toxicities
Timepoint [1] 0 0
24 months
Secondary outcome [1] 0 0
Treatment-related change in disease severity by ALS Functional Rating Scale - Revised (ALSFRS-R)
Timepoint [1] 0 0
24 months
Secondary outcome [2] 0 0
Treatment-related change in cognitive function by Edinburgh Cognitive and Behavioral Assessment (ECAS) score
Timepoint [2] 0 0
24 months
Secondary outcome [3] 0 0
Treatment-related change in respiratory function by seated forced vital capacity (FCV)
Timepoint [3] 0 0
24 months
Secondary outcome [4] 0 0
Treatment-related change in quality of life by ALSSQOL-R score
Timepoint [4] 0 0
24 months
Secondary outcome [5] 0 0
Treatment-related change in disease severity by transcranial magnetic stimulation (TMS) response
Timepoint [5] 0 0
24 months
Secondary outcome [6] 0 0
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
Timepoint [6] 0 0
12 months
Secondary outcome [7] 0 0
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
Timepoint [7] 0 0
12 months
Secondary outcome [8] 0 0
Treatment-related change in respiratory function by sniff nasal pressure (SNP) test
Timepoint [8] 0 0
24 months

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

Study design
Purpose of the study
Treatment
Allocation to intervention
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Macquarie University - Sydenham
Recruitment hospital [2] 0 0
Calvary Health Care Bethlehem - Caulfield
Recruitment postcode(s) [1] 0 0
2109 - Sydenham
Recruitment postcode(s) [2] 0 0
3162 - Caulfield

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, open-label , single and multiple dose-escalation and pharmacokinetic study
Trial website
https://clinicaltrials.gov/show/NCT02870634
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 9602
Fax 0 0
Email 0 0
kay.noel@ColMedDev.com
Contact person for scientific queries