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

NCT ID: NCT05039268

Last Updated: 2023-10-11

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 16 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-11-25
• Study Completion Date: 2022-09-12

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.

Conditions

Amyotrophic Lateral Sclerosis

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

15mg Dose Group

Participants will receive a fixed dose regimen of five doses of 15mg.

Group Type: ACTIVE_COMPARATOR

3K3A-APC Protein

Intervention Type: DRUG

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.

30mg Dose Group

Participants will receive a fixed dose regimen of five doses of 30mg.

Group Type: ACTIVE_COMPARATOR

3K3A-APC Protein

Intervention Type: DRUG

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.

Interventions

3K3A-APC Protein

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.

Intervention Type: DRUG

Other Intervention Names

3K3A-APC

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

ZZ Biotech, LLC

INDUSTRY

Sponsor Role: collaborator

Macquarie University, Australia

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Dominic Rowe, PhD, MD

Role: PRINCIPAL_INVESTIGATOR

Macquarie University, Australia

Locations

Macquarie University

Macquarie Park, New South Wales, Australia

Countries

Australia

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

ZZ-3K3A-202

Identifier Type: -

Identifier Source: org_study_id