ALS (Amyotrophic Lateral Sclerosis)Phase 1KOANEWANCT07401121
Safety and Tolerability Study of CTx1000 In Participants With Amyotrophic Lateral Sclerosis
A Phase 1 study testing AAV9 Gene therapy in people with ALS (Amyotrophic Lateral Sclerosis), run by Celosia Therapeutics Pty Ltd at 1 site in Australia.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- ALS (Amyotrophic Lateral Sclerosis)
- What is being tested?
- AAV9 Gene therapy
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteAustralia
- Study dates
- 12 Jan 2026to Dec 2030
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Australia1 site, 1 recruiting
- Recruiting
Macquarie University Hospital
Sydney, New South Wales, 2109
Richard Gan, 61298123739, richard.gan@mq.edu.au
About this study
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
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 the detailed description
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).
- Conditions
- ALS (Amyotrophic Lateral Sclerosis)
- Being tested
- AAV9 Gene therapy
- Keywords
- CTx1000, gene therapy, motor neuron disease, genetic medicine, Lou Gehrig disease
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AAV9 Gene therapyDrug
Single dose gene therapy
| Study group | Role | What they receive |
|---|---|---|
| Active drug-CTx1000 | Experimental | All participants will receive only one dose of the study drug Receives: Drug: AAV9 Gene therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 52 weeks
Secondary outcomes (3)
To evaluate the long-term safety and tolerability of CTx1000 in ALS diagnosed participants
Incidence of Treatment Emergent Adverse Events (TEAEs), Severe Adverse Events (SAEs) and Adverse Events of Special Interests (AESIs)
Measured: 3 years
To evaluate the long-term pharmacodynamics and immunogenicity of CTx1000 in ALS diagnosed participants
Changes in pre-dose biomarkers (Neurofilament light) in cerebrospinal fluid and anti-drug antibody (ADA)
Measured: 3 years
To evaluate the long-term efficacy of CTx1000 in ALS diagnosed participants
Changes (reduction) from pre-dose Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2026
- Study start12 Jan 2026
- Primary completionDec 2027 (estimated)
- Study completionDec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ALS (Amyotrophic Lateral Sclerosis)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Celosia Therapeutics Pty Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Novotech (Australia) Pty Limited
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07401121
- Sponsor's protocol ID
- ALS-AAV9
- Acronym
- KOANEWA
- Status last verified
- Feb 2026
- First submitted
- 10 Dec 2025
- Last updated
- 10 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07401121
- Acronym / sponsor ID
- KOANEWA / ALS-AAV9
- Conditions
- ALS (Amyotrophic Lateral Sclerosis)
- Keywords
- CTx1000, gene therapy, motor neuron disease, genetic medicine, Lou Gehrig disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Celosia Therapeutics Pty LtdIndustry (pharma, biotech, devices)
- Collaborators
- Novotech (Australia) Pty Limited
- Study start
- 12 Jan 2026 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Dec 2030 (estimated)
- First posted
- 10 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This clinical study is in participants with Amyotrophic Lateral Sclerosis and is designed to evaluate the safety and tolerability of the gene therapy CTx1000.
Detailed description
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Active drug-CTx1000 | Experimental | Drug: AAV9 Gene therapyAll participants will receive only one dose of the study drug |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AAV9 Gene therapySingle dose gene therapy | Drug | Active drug-CTx1000 |
Primary outcome
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)
3 secondary outcomes
To evaluate the long-term safety and tolerability of CTx1000 in ALS diagnosed participants
Time frame: 3 years
To evaluate the long-term pharmacodynamics and immunogenicity of CTx1000 in ALS diagnosed participants
Time frame: 3 years
To evaluate the long-term efficacy of CTx1000 in ALS diagnosed participants
Time frame: 3 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Feb 2026.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing AAV9 Gene therapy in people with ALS (Amyotrophic Lateral Sclerosis), run by Celosia Therapeutics Pty Ltd at 1 site in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 10 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate the safety and tolerability of CTx1000 in ALS diagnosed participants (time frame: 52 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Feb 2026.