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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT02870634
Ethics application status
Date submitted
10/08/2016
Date registered
17/08/2016

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
Sex
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
Not applicable
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
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
16/11/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
30/01/2020
Sample size
Target
50
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
Trial website
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
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Undecided
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT02870634