Phase 2
N=16
3K3A-APC for Treatment of Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis
Bottom Line
View on ClinicalTrials.gov: NCT05039268 ↗Enrolled (actual)
16
Serious AEs
0.0%
Results posted
Oct 2023
Primary outcome: Primary: Number of Participants Who Had Any Serious Adverse Events or Any Adverse Events With Severity Higher Than "Moderate". — 0; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- 3K3A-APC Protein (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Macquarie University, Australia
- Primary completion
- Sep 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Who Had Any Serious Adverse Events or Any Adverse Events With Severity Higher Than "Moderate". |
0; 0 | — |
| PRIMARY Percentage of Change in PERSI Score in the Motor Cortex Before and After Dosing |
-0.14; 0.53 | — |
| SECONDARY Diffusion Kurtosis Using MRI Scan |
— | — |
| SECONDARY Monocyte Activation |
— | — |
| SECONDARY Cytokine Level |
— | — |
| SECONDARY Chemokine Level |
— | — |
| SECONDARY Neurofilament Level |
— | — |
| SECONDARY Soluble CD14 Level |
— | — |
| SECONDARY Kynurenine Level |
— | — |
Summary
Phase 2 open label trial to investigate the safety and potentially efficacy of 3K3A-APC in patients with Amyotrophic Lateral Sclerosis (ALS).
Eligibility Criteria
Inclusion Criteria
- 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
Exclusion Criteria
- 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
- 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
Data sourced from ClinicalTrials.gov (NCT05039268). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.