NCT07401121RecruitingPhase 1
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
Source: ClinicalTrials.gov · Last updated 10 Feb 2026
Brief summary (registry, in English)
This clinical study is in participants with Amyotrophic Lateral Sclerosis and is designed to evaluate the safety and tolerability of the gene therapy CTx1000.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 15 (estimated)
- First submitted
- 10 Dec 2025
- Start date
- 12 Jan 2026
- Primary completion date
- 31 Dec 2027
- Lead sponsor
- Celosia Therapeutics Pty Ltd
- Diseases
- ALS (motor neurone disease)
- NCT number
- NCT07401121
What you commit to
- Study type: Interventional · Phase 1
- Planned participants: 15
- From 12 Jan 2026 to the primary completion date, 31 Dec 2027
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Any participants with genetic forms of ALS, including C9ORF72 repeat carriers, except for TARDBP gene variants, as confirmed by previous clinical history genetic testing
Other illness
- Positive test for cytomegalovirus, hepatitis C antibody (HCV), hepatitis B surface antigen (HBsAg), human immunodeficiency virus (HIV) antibody.
Lab values
- Any history of myocardial infarction or stroke within 6 months prior to Screening, or uncontrolled diabetes (HbA1C \> 9%)
Other
- Inadequate organ function
- Any participant with a current open tracheostomy
Who the study is looking for
- 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
Age in the registry: 18 years to 80 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Macquarie University Hospital Coordinator of this site? Confirm its status | Sydney | Australia | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.