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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05039268




Registration number
NCT05039268
Ethics application status
Date submitted
23/08/2021
Date registered
9/09/2021

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] 0 0
ZZ-3K3A-202
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 - 3K3A-APC Protein

Active comparator: 15mg Dose Group - Participants will receive a fixed dose regimen of five doses of 15mg.

Active comparator: 30mg Dose Group - Participants will receive a fixed dose regimen of five doses of 30mg.


Treatment: Drugs: 3K3A-APC Protein
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] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants Who Had Any Serious Adverse Events or Any Adverse Events With Severity Higher Than "Moderate".
Timepoint [1] 0 0
15 Days
Primary outcome [2] 0 0
Percentage of Change in PERSI Score in the Motor Cortex Before and After Dosing
Timepoint [2] 0 0
7 Days
Secondary outcome [1] 0 0
Diffusion Kurtosis Using MRI Scan
Timepoint [1] 0 0
7 Days
Secondary outcome [2] 0 0
Monocyte Activation
Timepoint [2] 0 0
7 Days
Secondary outcome [3] 0 0
Cytokine Level
Timepoint [3] 0 0
7 Days
Secondary outcome [4] 0 0
Chemokine Level
Timepoint [4] 0 0
7 Days
Secondary outcome [5] 0 0
Neurofilament Level
Timepoint [5] 0 0
7 Days
Secondary outcome [6] 0 0
Soluble CD14 Level
Timepoint [6] 0 0
7 Days
Secondary outcome [7] 0 0
Kynurenine Level
Timepoint [7] 0 0
7 Days

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
Sex
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 2
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
25/11/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
12/09/2022
Sample size
Target
Accrual to date
Final
16
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Macquarie University - Macquarie Park
Recruitment postcode(s) [1] 0 0
2113 - Macquarie Park

Funding & Sponsors
Primary sponsor type
Other
Name
Macquarie University, Australia
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
ZZ Biotech, LLC
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Dominic Rowe, PhD, MD
Address 0 0
Macquarie University, Australia
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)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT05039268