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

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

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ANZCTR

Registry ID

ACTRN12621000864820

Enrollment

Registered

2021-07-05

Start date

2021-11-25

Completion date

2022-08-29

Last updated

2022-10-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

None listed

Brief 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.

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.

Study design

Allocation

Non-randomised trial

Primary purpose

Treatment

Eligibility

Sex/Gender

All

Age

18 Years to 75 Years

Healthy volunteers

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 38 months before screening 4. Diagnosis of ALS less than 26 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

Outcome results