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

• Register: ClinicalTrials.gov
• Last refreshed on: 10 October 2022
• Main ID: NCT05039268
• Date of registration: 23/08/2021
• Prospective Registration: Yes
• Primary sponsor: Macquarie University, Australia
• 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
• Date of first enrolment: November 25, 2021
• Target sample size: 16
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT05039268
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Australia

Contacts

• Name: Dominic Rowe, PhD, MD
• Affiliation: Macquarie University, Australia

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: 3K3A-APC Protein

Primary Outcome(s)

Safety Assessment [Time Frame: 45 Days]

Microglial Activation in the Motor Cortex [Time Frame: 8 Days]

Secondary Outcome(s)

Neurofilament Level [Time Frame: 45 Days]

Diffusion Kurtosis Using MRI Scan [Time Frame: 8 Days]

Chemokine Level [Time Frame: 45 Days]

Cytokine Level [Time Frame: 45 Days]

Kynurenine Level [Time Frame: 45 Days]

Monocyte Activation [Time Frame: 45 Days]

Soluble CD14 Level [Time Frame: 45 Days]

Secondary ID(s)

ZZ-3K3A-202

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

ZZ Biotech, LLC