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

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

Status

Recruiting

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT07401121

Acronym

KOANEWA

Enrollment

Registered

2026-02-10

Start date

2026-01-12

Completion date

2030-12-01

Last updated

2026-02-10

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

Conditions

ALS (Amyotrophic Lateral Sclerosis)

Keywords

CTx1000, gene therapy, motor neuron disease, genetic medicine, Lou Gehrig disease

Brief summary

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

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).

Interventions

DRUGAAV9 Gene therapy

Single dose gene therapy

Study design

Allocation

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to 80 Years

Healthy volunteers

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

Design outcomes

Primary

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

Secondary

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

Countries

Australia

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 11, 2026