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Trial registered on ANZCTR


Registration number
ACTRN12621000864820
Ethics application status
Approved
Date submitted
19/05/2021
Date registered
5/07/2021
Date last updated
19/08/2021
Date data sharing statement initially provided
5/07/2021
Type of registration
Prospectively registered

Titles & IDs
Public title
3K3A-APC for Treatment of Amyotrophic Lateral Sclerosis (ALS)
Scientific title
A Phase 2 Open Label Trial of 3K3A-APC in Amyotrophic Lateral Sclerosis
Secondary ID [1] 304245 0
Nil known
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Amyotrophic Lateral Sclerosis 321946 0
Motor Neuron Disease 321947 0
Condition category
Condition code
Neurological 319678 319678 0 0
Neurodegenerative diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
3K3A-APC with intravenous dosing of five doses of either 15mg or 30mg at 12 hourly interval. The first 8 patients will receive 15mg dose, and the next 8 patients will receive 30mg dose.
Intervention code [1] 320578 0
Treatment: Drugs
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 327558 0
Composite safety assessment by clinical laboratory testing, physical examination and the collection of adverse events. Known adverse reactions include headache, nausea, vomiting and vertigo, which will be assessed via self-reporting of adverse events. Any clinically significant abnormal findings in clinical laboratory testing (full blood count, biochemistry, coagulation, iron study, CSF analysis and ECG) and physical examinations will also be recorded as adverse events, and their relationship to the investigational drug will be assessed.
Timepoint [1] 327558 0
Screening, then daily from Day 1 through Day 10, and Day 15
Primary outcome [2] 327559 0
Microglial activation in the motor cortex in two (2) dose cohorts, utilising serial [18F]FEMPA PET imaging
Timepoint [2] 327559 0
1 Day before intervention and 4 days after intervention
Secondary outcome [1] 395655 0
Exploratory outcome: MRI scan of the brain utilising diffusion kurtosis to determine whether the blood brain barrier integrity can be measured in ALS by Magnetic Resonance Imaging, and whether 3K3A-APC is able to repair it
Timepoint [1] 395655 0
1 Day before intervention and 4 days after intervention
Secondary outcome [2] 395656 0
Exploratory outcome: biomic measurements of serum, plasma, urine and CSF, consisting of a novel method to determine whether monocyte activation in the peripheral blood can act as a surrogate marker of microglial activation state in ALS, as well as changes in cytokine, chemokine, neurofilament, soluble CD14 and kynurenine levels
Timepoint [2] 395656 0
Screening, then daily from Day 1 through Day 10, and Day 15

Eligibility
Key inclusion criteria
1. Patients must have clinically definite ALS (Awaji Criteria)
2. Male or female age 18 years and less than 75 years at time of ALS study
3. Symptom onset less than 36 months before screening
4. Diagnosis of ALS less than 24 months before screening
5. Clinically definite Upper Motor Neuron signs
Minimum age
18 Years
Maximum age
75 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Current treatment with anticoagulants (e.g., warfarin, novel oral anticoagulants, heparin) that might preclude safe completion of the lumbar puncture
2. Condition that precludes the safe performance of routine lumbar puncture, such as prohibitive lumbar spinal disease, bleeding diathesis, or clinically significant coagulopathy or thrombocytopenia
3. Use of investigational drugs or devices within 60 days prior to Baseline (dietary supplements taken outside of a clinical trial are not exclusionary, e.g., coenzyme Q10)
4. Prolonged prothrombin time or activated partial thromboplastin time >2xULN
5. Severe hypertension or hypotension
6. Glomerular filtration rate (GFR) <35 mL/min
7. Forced vital capacity (FVC) at screening of <50% of predicted
8. Prior exposure to any exogenous form of APC
9. Inability to lie flat for procedures (MRI, PET, LP)
10. Pregnant or lactating during the study period

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised 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
Who is / are masked / blinded?



Intervention assignment
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
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
Recruitment hospital [1] 19470 0
Macquarie University Hospital - Macquarie Park
Recruitment postcode(s) [1] 34061 0
2109 - Macquarie Park

Funding & Sponsors
Funding source category [1] 308621 0
University
Name [1] 308621 0
Macquarie University
Address [1] 308621 0
Suite 204/2 Technology Place
Macquarie University, NSW 2109, Australia
Country [1] 308621 0
Australia
Primary sponsor type
University
Name
Macquarie University
Address
Suite 204/2 Technology Place
Macquarie University, NSW 2109, Australia
Country
Australia
Secondary sponsor category [1] 309490 0
None
Name [1] 309490 0
N/A
Address [1] 309490 0
N/A
Country [1] 309490 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 308553 0
Macquarie University Human Research Ethics Committee (Medical Sciences)
Ethics committee address [1] 308553 0
16 Wally's Walk
Macquarie University, NSW 2109, Australia
Ethics committee country [1] 308553 0
Australia
Date submitted for ethics approval [1] 308553 0
17/05/2021
Approval date [1] 308553 0
29/07/2021
Ethics approval number [1] 308553 0
520211010530509

Summary
Brief summary
This Phase 2 open label trial seeks to investigate whether a novel therapy named 3K3A-APC is safe and potentially effective in patients with Amyotrophic Lateral Sclerosis (ALS). A total of 16 patients with ALS will be enrolled into 2 dose cohorts with five doses of 15mg or 30mg doses given 12 hours apart in each cohort. The primary study outcomes are to ensure the safety and tolerability of 3K3AAPC in ALS patients, and to determine whether 3K3A-APC is able to reduce the pathological changes that might possibly cause ALS.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 111114 0
Prof Dominic Rowe
Address 111114 0
Suite 204/2 Technology Place
Macquarie University, NSW 2109, Australia
Country 111114 0
Australia
Phone 111114 0
+61 2 9812 3720
Fax 111114 0
+61 2 9812 3722
Email 111114 0
dominic.rowe@mq.edu.au
Contact person for public queries
Name 111115 0
Mr Richard Gan
Address 111115 0
Suite 204/2 Technology Place
Macquarie University, NSW 2109, Australia
Country 111115 0
Australia
Phone 111115 0
+61 2 9812 3739
Fax 111115 0
+61 2 9812 3722
Email 111115 0
richard.gan@mq.edu.au
Contact person for scientific queries
Name 111116 0
Prof Dominic Rowe
Address 111116 0
Suite 204/2 Technology Place
Macquarie University, NSW 2109, Australia
Country 111116 0
Australia
Phone 111116 0
+61 2 9812 3720
Fax 111116 0
+61 2 9812 3722
Email 111116 0
dominic.rowe@mq.edu.au

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
What supporting documents are/will be available?
Study protocol
Informed consent form
Ethical approval
How or where can supporting documents be obtained?
Type [1] 11692 0
Study protocol
Citation [1] 11692 0
Link [1] 11692 0
Email [1] 11692 0
Other [1] 11692 0
Available upon request
Attachment [1] 11692 0
Type [2] 11693 0
Informed consent form
Citation [2] 11693 0
Link [2] 11693 0
Email [2] 11693 0
Other [2] 11693 0
Available upon request
Attachment [2] 11693 0
Type [3] 11694 0
Ethical approval
Citation [3] 11694 0
Link [3] 11694 0
Email [3] 11694 0
Other [3] 11694 0
Available upon request
Attachment [3] 11694 0
Summary results
No Results