Clinical trial NCT05039268: 3K3A-APC for Treatment of Amyotrophic Lateral Sclerosis (ALS)
Official title: A Phase 2 Open Label Trial of 3K3A-APC in Amyotrophic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Keywords: ALS
Amyotrophic Lateral Sclerosis
MND
Motor Neurone Disease
3K3A-APC
Lead sponsor: Macquarie University, Australia
Sponsor class: OTHER
Collaborators: name: ZZ Biotech, LLC
First posted: 2021-09-09
Enrollment: 16
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: 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.

Eligibility criteria: 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

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

Primary outcomes:
measure: Number of Participants Who Had Any Serious Adverse Events or Any Adverse Events With Severity Higher Than "Moderate".
timeFrame: 15 Days
measure: Percentage of Change in PERSI Score in the Motor Cortex Before and After Dosing
timeFrame: 7 Days
Secondary outcomes:
measure: Diffusion Kurtosis Using MRI Scan
timeFrame: 7 Days
measure: Monocyte Activation
timeFrame: 7 Days
measure: Cytokine Level
timeFrame: 7 Days
measure: Chemokine Level
timeFrame: 7 Days
measure: Neurofilament Level
timeFrame: 7 Days
measure: Soluble CD14 Level
timeFrame: 7 Days
measure: Kynurenine Level
timeFrame: 7 Days
Other outcomes:
Locations:
city: Macquarie Park
country: Australia
facility: Macquarie University
state: New South Wales
Study officials:
affiliation: Macquarie University, Australia
name: Dominic Rowe, PhD, MD
role: PRINCIPAL_INVESTIGATOR
References: