CompletedPhase 2ACTRN12621000864820

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

A Phase 2 Open Label Trial of 3K3A-APC in Amyotrophic Lateral Sclerosis

Sponsor

Macquarie University

Enrollment

16 participants

Start Date

Nov 25, 2021

Study Type

Interventional

Conditions

Amyotrophic Lateral Sclerosis Motor Neuron Disease

Summary

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

Sex: Both males and femalesMin Age: 18 YearssMax Age: 75 Yearss

Inclusion Criteria5

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 38 months before screening
Diagnosis of ALS less than 26 months before screening
Clinically definite Upper Motor Neuron signs

Exclusion Criteria10

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

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Interventions

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.