Amyotrophic Lateral SclerosisPhase 2NCT05039268
3K3A-APC for Treatment of Amyotrophic Lateral Sclerosis (ALS)
A Phase 2 study testing 3K3A-APC Protein in people with Amyotrophic Lateral Sclerosis, run by Macquarie University, Australia at 1 site in Australia.
Trial summary
Phase 2 open label trial to investigate the safety and potentially efficacy of 3K3A-APC in patients with Amyotrophic Lateral Sclerosis (ALS). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- 3K3A-APC Protein
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 1 siteAustralia
- Study dates
- 25 Nov 2021to 12 Sep 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients must have clinically definite ALS (Awaji Criteria)
- Male or female age 18 years and less than 75 years at time of ALS study
- Symptom onset less than 36 months before screening
- Diagnosis of ALS less than 24 months before screening
- Clinically definite Upper Motor Neuron signs
Who cannot join
- Current treatment with anticoagulants (e.g., warfarin, novel oral anticoagulants, heparin) that might preclude safe completion of the lumbar puncture
- Condition that precludes the safe performance of routine lumbar puncture, such as prohibitive lumbar spinal disease, bleeding diathesis, or clinically significant coagulopathy or thrombocytopenia
- 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)
- Prolonged prothrombin time or activated partial thromboplastin time >2xULN
- Severe hypertension or hypotension
- Glomerular filtration rate (GFR) <35 mL/min
Show all 10Show fewer
- Forced vital capacity (FVC) at screening of <50% of predicted
- Prior exposure to any exogenous form of APC
- Inability to lie flat for procedures (MRI, PET, LP)
- Pregnant or lactating during the study period
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 2 Open Label Trial of 3K3A-APC in Amyotrophic Lateral Sclerosis
Phase 2 open label trial to investigate the safety and potentially efficacy of 3K3A-APC in patients with Amyotrophic Lateral Sclerosis (ALS).
Read the detailed description
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.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- 3K3A-APC Protein
- Keywords
- ALS, Amyotrophic Lateral Sclerosis, MND, Motor Neurone Disease, 3K3A-APC
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
3K3A-APC ProteinDrug
Also called 3K3A-APC
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.
| Study group | Role | What they receive |
|---|---|---|
| 15mg Dose Group | Active comparator | Participants will receive a fixed dose regimen of five doses of 15mg. Receives: Drug: 3K3A-APC Protein |
| 30mg Dose Group | Active comparator | Participants will receive a fixed dose regimen of five doses of 30mg. Receives: Drug: 3K3A-APC Protein |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Who Had Any Serious Adverse Events or Any Adverse Events With Severity Higher Than "Moderate".
Number of participants who had any serious adverse events or any adverse events with severity higher than "moderate", as determined by the Principal Investigator, using the composite safety assessment including clinical laboratory testing (full blood count, biochemistry, coagulation, iron study, CSF analysis and ECG), physical examination and self-reporting of adverse events. All clinical significant findings in the composite safety assessment were reported as adverse events.
Measured: 15 Days
Percentage of Change in PERSI Score in the Motor Cortex Before and After Dosing
PERSI (Parametric Estimation of Reference Signal Intensity) score is the measurement of microglial activation in the motor cortex utilising serial [18F]FEMPA PET imaging. The percentage of change in PERSI score before and after dosing in the two (2) dose cohorts is calculated.
Measured: 7 Days
Secondary outcomes (7)
Diffusion Kurtosis Using MRI Scan
Change in diffusion kurtosis using MRI scan of the brain 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
Measured: 7 Days
Monocyte Activation
Change in the level of monocyte activation in the peripheral blood utilising a novel method.
Measured: 7 Days
Cytokine Level
Change in cytokine level in serum, plasma and CSF.
Measured: 7 Days
Chemokine Level
Change in chemokine level in serum, plasma and CSF.
Measured: 7 Days
Neurofilament Level
Change in neurofilament level in serum, plasma and CSF.
Measured: 7 Days
Soluble CD14 Level
Change in soluble CD14 level in serum, plasma and CSF.
Measured: 7 Days
Kynurenine Level
Change in kynurenine level in serum, plasma and CSF.
Measured: 7 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2021
- Study start25 Nov 2021
- Primary completion12 Sep 2022
- Study completion12 Sep 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Macquarie University, Australia
- Sponsor type
- University, hospital or other
- Collaborators
- ZZ Biotech, LLC
- Responsible party
- Sponsor
- Investigators
- Dominic Rowe, PhD, MD, Principal investigator, Macquarie University, Australia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05039268
- Sponsor's protocol ID
- ZZ-3K3A-202
- Status last verified
- Sep 2022
- First submitted
- 23 Aug 2021
- Last updated
- 11 Oct 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Open Label Trial of 3K3A-APC in Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT05039268
- Acronym / sponsor ID
- ZZ-3K3A-202
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS, Amyotrophic Lateral Sclerosis, MND, Motor Neurone Disease, 3K3A-APC
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Macquarie University, AustraliaUniversity, hospital or other
- Collaborators
- ZZ Biotech, LLC
- Study start
- 25 Nov 2021 (actual)
- Primary completion
- 12 Sep 2022 (actual)
- Study completion
- 12 Sep 2022 (actual)
- First posted
- 9 Sep 2021 (actual)
- Results
- Posted 11 Oct 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Phase 2 open label trial to investigate the safety and potentially efficacy of 3K3A-APC in patients with Amyotrophic Lateral Sclerosis (ALS).
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 15mg Dose Group | Active comparator | Drug: 3K3A-APC ProteinParticipants will receive a fixed dose regimen of five doses of 15mg. |
| 30mg Dose Group | Active comparator | Drug: 3K3A-APC ProteinParticipants will receive a fixed dose regimen of five doses of 30mg. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Patients must have clinically definite ALS (Awaji Criteria)
- Male or female age 18 years and less than 75 years at time of ALS study
- Symptom onset less than 36 months before screening
- Diagnosis of ALS less than 24 months before screening
- Clinically definite Upper Motor Neuron signs
Key exclusion criteria (10 listed)
- Current treatment with anticoagulants (e.g., warfarin, novel oral anticoagulants, heparin) that might preclude safe completion of the lumbar puncture
- Condition that precludes the safe performance of routine lumbar puncture, such as prohibitive lumbar spinal disease, bleeding diathesis, or clinically significant coagulopathy or thrombocytopenia
- 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)
- Prolonged prothrombin time or activated partial thromboplastin time >2xULN
- Severe hypertension or hypotension
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 3K3A-APC ProteinAlso: 3K3A-APC3K3A-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. | Drug | 15mg Dose Group, 30mg Dose Group |
Primary outcome
Number of Participants Who Had Any Serious Adverse Events or Any Adverse Events With Severity Higher Than "Moderate".
Time frame: 15 Days
Number of participants who had any serious adverse events or any adverse events with severity higher than "moderate", as determined by the Principal Investigator, using the composite safety assessment including clinical laboratory testing (full blood count, biochemistry, coagulation, iron study, CSF analysis and ECG), physical examination and self-reporting of adverse events. All clinical significant findings in the composite safety assessment were reported as adverse events.
Percentage of Change in PERSI Score in the Motor Cortex Before and After Dosing
Time frame: 7 Days
PERSI (Parametric Estimation of Reference Signal Intensity) score is the measurement of microglial activation in the motor cortex utilising serial [18F]FEMPA PET imaging. The percentage of change in PERSI score before and after dosing in the two (2) dose cohorts is calculated.
7 secondary outcomes
Diffusion Kurtosis Using MRI Scan
Time frame: 7 Days
Monocyte Activation
Time frame: 7 Days
Cytokine Level
Time frame: 7 Days
Chemokine Level
Time frame: 7 Days
Neurofilament Level
Time frame: 7 Days
Soluble CD14 Level
Time frame: 7 Days
Kynurenine Level
Time frame: 7 Days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Oct 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Dominic Rowe, PhD, MD principal investigator
Macquarie University, Australia
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing 3K3A-APC Protein in people with Amyotrophic Lateral Sclerosis, run by Macquarie University, Australia at 1 site in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 11 Oct 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants Who Had Any Serious Adverse Events or Any Adverse Events With Severity Higher Than "Moderate". (time frame: 15 Days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Oct 2023.