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NCT Number: NCT07401121

Safety and Tolerability Study of CTx1000 In Participants 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.

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Key information

Conditions

ALS (Amyotrophic Lateral Sclerosis) Amyotrophic Lateral Sclerosis Central Nervous System Diseases Metabolic Diseases Motor Neuron Disease Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Nutritional and Metabolic Diseases Proteostasis Deficiencies Spinal Cord Diseases TDP-43 Proteinopathies

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Macquarie University Hospital

Sydney, New South Wales, 2109, Australia

Location status: Recruiting

Location contact

Richard Gan

CONTACT

[email protected]

61298123739

About this study

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).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

AAV9 Gene therapy

Drug

Single dose gene therapy

Primary outcomes

  1. To evaluate the safety and tolerability of CTx1000 in ALS diagnosed participants

    Time frame: 52 weeks

    Incidence of Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interests (AESIs)

Secondary outcomes

  1. To evaluate the long-term safety and tolerability of CTx1000 in ALS diagnosed participants

    Time frame: 3 years

    Incidence of Treatment Emergent Adverse Events (TEAEs), Severe Adverse Events (SAEs) and Adverse Events of Special Interests (AESIs)

  2. To evaluate the long-term pharmacodynamics and immunogenicity of CTx1000 in ALS diagnosed participants

    Time frame: 3 years

    Changes in pre-dose biomarkers (Neurofilament light) in cerebrospinal fluid and anti-drug antibody (ADA)

  3. To evaluate the long-term efficacy of CTx1000 in ALS diagnosed participants

    Time frame: 3 years

    Changes (reduction) from pre-dose Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)

Sponsors and collaborators

Lead sponsor

Celosia Therapeutics Pty Ltd

Industry

Collaborators

  • Novotech (Australia) Pty Limited

Registry information

Official study title

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

Acronym: KOANEWA

Important dates

Study start
2026
Primary completion
2027
Study completion
2030
First posted
Feb 10, 2026
Registry last updated
Feb 10, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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