CTx1000 gene therapy delivered into the cisterna magna for people with ALS
Official 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
Run by Celosia Therapeutics Pty Ltd · NCT07401121 · Data updated 2026-11-12
This trial will test a single-dose gene therapy called CTx1000 given into the cisterna magna to see if it is safe and tolerable for people with early ALS.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years to 80 Years |
| Sex | All |
| Enrollment | 15 (estimated) |
| Sponsor | Celosia Therapeutics Pty Ltd Industry-sponsored |
| Locations | 1 site (Sydney, New South Wales) |
| Status | Recruiting |
| Started | January 12, 2026 |
| Main results expected | December 2027 |
| Study ends | December 2030 |
| Registry record updated | February 10, 2026 |
| Trial ID | NCT07401121 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are people with ALS within two years of first symptoms, without most genetic ALS variants (except permitted TARDBP variants), with low anti‑AAV9 antibody titers, stable on ALS medications, and able to travel for ICM dosing and follow‑up.
✕Probably not a fit
Patients with known genetic forms of ALS such as C9ORF72 carriers, high anti‑AAV9 antibody levels, advanced disease beyond two years, recent MI or stroke, uncontrolled diabetes, active viral infections, or pregnancy are unlikely to be eligible or to receive benefit.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
CTx1000 is an investigational AAV9-based gene therapy that encodes a degron to promote targeted degradation of pathological TDP‑43 protein. In this Phase 1, single-dose interventional study participants receive one intra‑cisterna magna (ICM) infusion and are followed for safety, tolerability, and immune response. Eligible participants have ALS with symptom duration of ≤2 years, low circulating anti‑AAV9 antibody titers, and no common genetic ALS forms except certain TARDBP variants. Standard safety exclusions include recent myocardial infarction or stroke, uncontrolled diabetes, active viral infections, and pregnancy, and participants must use effective contraception.
Why it matters
Potential benefit: If successful, CTx1000 could reduce pathological TDP‑43 levels and potentially slow disease progression in people with ALS.
How similar studies have gone: AAV9 delivery has established clinical precedent in spinal muscular atrophy, but using an AAV9‑delivered degron to target TDP‑43 in ALS is novel and unproven in humans.
Who can join: the full rules
Show full inclusion / exclusion criteria
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
Where it's running
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Macquarie University Hospital
Sydney, New South Wales, Australia Recruiting
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.