Safety and Tolerability Study of CTx1000 In Participants With Amyotrophic Lateral Sclerosis (KOANEWA)

Koanewa: A First in Human, Phase 1b, Open-label, Non-randomised, Single Dose Study to Assess the Safety and Tolerability of CTx1000 in Participants Diagnosed With Amyotrophic Lateral Sclerosis

This clinical study is in participants with Amyotrophic Lateral Sclerosis and is designed to evaluate the safety and tolerability of the gene therapy CTx1000.

Study Overview

• Status: Recruiting
• Conditions: ALS (Amyotrophic Lateral Sclerosis)
• Intervention / Treatment: Drug: AAV9 Gene therapy

Detailed Description

CTx1000 is an investigational gene therapy that encodes a degron for targeted degradation of TDP-43 following a single dose intra cisterna magna (ICM) delivery in participants diagnosed with Amyotrophic Lateral Sclerosis (ALS).

• Study Type: Interventional
• Enrollment (Estimated): 15
• Phase: Phase 1

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Sydney, New South Wales, Australia, 2109
      • Recruiting
      • Macquarie University Hospital
      • Contact: Richard Gan; Phone Number: 61298123739; Email: richard.gan@mq.edu.au

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Diagnosis of ALS in accordance with the revised El Escorial criteria and TRICALS risk score
• An overall disease duration of ≤ 2 years after the participant's first symptoms
• No or low circulating anti-AAV9 antibodies (titre ≤ 1:50
• Stable dosing with a standard of care ALS medication (eg, riluzole and edaravone) and other prescription medications for 30 days prior to Screening
• Not pregnant or breastfeeding, or willing to cease breastfeeding
• All participants must use a barrier method of contraception

Exclusion Criteria:

• Any participants with genetic forms of ALS, including C9ORF72 repeat carriers, except for TARDBP gene variants, as confirmed by previous clinical history genetic testing
• Any history of myocardial infarction or stroke within 6 months prior to Screening, or uncontrolled diabetes (HbA1C > 9%)
• Positive test for cytomegalovirus, hepatitis C antibody (HCV), hepatitis B surface antigen (HBsAg), human immunodeficiency virus (HIV) antibody.
• Inadequate organ function
• Any participant with a current open tracheostomy

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Active drug-CTx1000; All participants will receive only one dose of the study drug	Drug: AAV9 Gene therapy; Single dose gene therapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate the safety and tolerability of CTx1000 in ALS diagnosed participants	Incidence of Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interests (AESIs)	52 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate the long-term safety and tolerability of CTx1000 in ALS diagnosed participants	Incidence of Treatment Emergent Adverse Events (TEAEs), Severe Adverse Events (SAEs) and Adverse Events of Special Interests (AESIs)	3 years
To evaluate the long-term pharmacodynamics and immunogenicity of CTx1000 in ALS diagnosed participants	Changes in pre-dose biomarkers (Neurofilament light) in cerebrospinal fluid and anti-drug antibody (ADA)	3 years
To evaluate the long-term efficacy of CTx1000 in ALS diagnosed participants	Changes (reduction) from pre-dose Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)	3 years

Collaborators and Investigators

• Sponsor: Celosia Therapeutics Pty Ltd
• Collaborators: Novotech (Australia) Pty Limited

Publications and helpful links

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): January 12, 2026
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2030

Study Registration Dates

• First Submitted: December 10, 2025
• First Submitted That Met QC Criteria: February 6, 2026
• First Posted (Actual): February 10, 2026

Study Record Updates

• Last Update Posted (Actual): February 10, 2026
• Last Update Submitted That Met QC Criteria: February 6, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Keywords: gene therapy; motor neuron disease; CTx1000; genetic medicine; Lou Gehrig disease
• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Metabolic Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Nutritional and Metabolic Diseases; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: ALS-AAV9

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No