Safety Study of a Gene Therapy for Amyotrophic Lateral Sclerosis
Tests the gene therapy CTx1000 for safety in adults with ALS.
Plain-language title generated from the registry record
Registry title: Safety and Tolerability Study of CTx1000 In Participants With Amyotrophic Lateral Sclerosis
Key facts
- Where
- Sydney, AustraliaSee the site
- Phase
- Phase 1
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 80 years old?
Source: NCT07401121, who can take part
Where in Australia
- Macquarie University HospitalSydney, AustraliaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Celosia Therapeutics Pty Ltd
- Trial code
- NCT07401121
- Record updated
- 10 February 2026
- Original description
This clinical study is in participants with Amyotrophic Lateral Sclerosis and is designed to evaluate the safety and tolerability of the gene therapy CTx1000.Read full original descriptionShow less
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.
Inclusion: 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: Any participants with genetic forms of ALS, including C9ORF72 repeat carriers, except for TARDBP gene variants, as confirmed by previous clinical history genetic testing; Positive test for cytomegalovirus, hepatitis C antibody (HCV), hepatitis B surface antigen (HBsAg), human immunodeficiency virus (HIV) antibody; Any history of myocardial infarction or stroke within 6 months prior to Screening, or uncontrolled diabetes (HbA1C > 9%); Inadequate organ function; Any participant with a current open tracheostomy.