Trial Outcomes & Findings for 3K3A-APC for Treatment of Amyotrophic Lateral Sclerosis (ALS) (NCT NCT05039268)

Last Updated: 2023-10-11

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

16 participants

Primary outcome timeframe

15 Days

Results posted on

2023-10-11

Participant Flow

Participant milestones

Participant milestones
Measure	15mg Dose Group Participants will receive a fixed dose regimen of five doses of 15mg. 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.	30mg Dose Group Participants will receive a fixed dose regimen of five doses of 30mg. 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.
Overall Study STARTED	8	8
Overall Study COMPLETED	8	8
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

3K3A-APC for Treatment of Amyotrophic Lateral Sclerosis (ALS)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	15mg Dose Group n=8 Participants Participants will receive a fixed dose regimen of five doses of 15mg. 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.	30mg Dose Group n=8 Participants Participants will receive a fixed dose regimen of five doses of 30mg. 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.	Total n=16 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	6 Participants n=5 Participants	5 Participants n=7 Participants	11 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Age, Continuous	59.5 years n=5 Participants	57.1 years n=7 Participants	58.3 years n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants	5 Participants n=7 Participants	11 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	8 Participants n=5 Participants	8 Participants n=7 Participants	16 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	8 Participants n=5 Participants	8 Participants n=7 Participants	16 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment Australia	8 participants n=5 Participants	8 participants n=7 Participants	16 participants n=5 Participants
ALSFRS-R Score	30.875 units on a scale n=5 Participants	37.125 units on a scale n=7 Participants	34.0 units on a scale n=5 Participants

PRIMARY outcome

Timeframe: 15 Days

Population: All dosed participants are included in the analysis.

Outcome measures

Outcome measures
Measure	15mg Dose Group n=8 Participants Participants will receive a fixed dose regimen of five doses of 15mg. 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.	30mg Dose Group n=8 Participants Participants will receive a fixed dose regimen of five doses of 30mg. 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.
Number of Participants Who Had Any Serious Adverse Events or Any Adverse Events With Severity Higher Than "Moderate".	0 Participants	0 Participants

PRIMARY outcome

Timeframe: 7 Days

Population: In total, 8 participants were included in the data analysis, with 5 participants in the 15mg group and 3 participants in the 30mg group.

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.

Outcome measures

Outcome measures
Measure	15mg Dose Group n=5 Participants Participants will receive a fixed dose regimen of five doses of 15mg. 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.	30mg Dose Group n=3 Participants Participants will receive a fixed dose regimen of five doses of 30mg. 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.
Percentage of Change in PERSI Score in the Motor Cortex Before and After Dosing	-0.14 Percentage of change Interval -4.01 to 2.63	0.53 Percentage of change Interval -0.96 to 2.61

Adverse Events

15mg Dose Group

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

30mg Dose Group

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	15mg Dose Group n=8 participants at risk Participants will receive a fixed dose regimen of five doses of 15mg. 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.	30mg Dose Group n=8 participants at risk Participants will receive a fixed dose regimen of five doses of 30mg. 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.
Injury, poisoning and procedural complications Fall	25.0% 2/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.	12.5% 1/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.
Nervous system disorders Headache	0.00% 0/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.	37.5% 3/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.
Gastrointestinal disorders Constipation	0.00% 0/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.	25.0% 2/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.
Investigations Neutrophilia	12.5% 1/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.	0.00% 0/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.
Musculoskeletal and connective tissue disorders Back pain	0.00% 0/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.	12.5% 1/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.
Gastrointestinal disorders Abdominal Pain Upper	0.00% 0/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.	12.5% 1/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.
Gastrointestinal disorders Nausea	0.00% 0/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.	12.5% 1/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.
Investigations Blood calcium increased	12.5% 1/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.	0.00% 0/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.
Investigations Blood bicarbonate increased	12.5% 1/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.	0.00% 0/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.
Cardiac disorders Supraventricular extrasystoles	12.5% 1/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.	0.00% 0/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.
Infections and infestations Herpes virus infection	0.00% 0/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.	12.5% 1/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.
Musculoskeletal and connective tissue disorders Arthralgia	12.5% 1/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.	0.00% 0/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.
General disorders Catheter site extravasation	0.00% 0/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.	12.5% 1/8 • Day-of-consent through end-of-participation, 15 days Adverse events are continuously collected from day-of-consent through end-of-participation for each participant.

Additional Information

Richard Gan

Macquarie University

Phone: +61 2 9812 3739

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place