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

NCT07401121 | Recruiting Phase 1

• 1 other identifier: ALS-AAV9
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

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.

Conditions

ALS (Amyotrophic Lateral Sclerosis)

Keywords

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

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

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

  3 years

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

  3 years

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

  3 years

Study Arms (1)

Active drug-CTx1000

EXPERIMENTAL

All participants will receive only one dose of the study drug

Drug: AAV9 Gene therapy

Interventions

AAV9 Gene therapy DRUG

Single dose gene therapy

Active drug-CTx1000

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Celosia Therapeutics Pty Ltd: lead
• Novotech (Australia) Pty Limited: collaborator

Study Sites (1)

Macquarie University Hospital

Sydney, New South Wales, 2109, Australia

RECRUITING

Related Links

• Related Info

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Motor Neuron Disease

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 10, 2025
• First Posted: February 10, 2026
• Study Start: January 12, 2026
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2030
• Last Updated: February 10, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1)