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

Safety and Tolerability Study of CTx1000 In Participants With Amyotrophic Lateral Sclerosis (NCT07401121) is a Phase 1 interventional studying ALS (Amyotrophic Lateral Sclerosis), sponsored by Celosia Therapeutics Pty Ltd. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

About This Trial

This clinical study is in participants with Amyotrophic Lateral Sclerosis and is designed to evaluate the safety and tolerability of the gene therapy CTx1000.

What Stage of Research Is This?

Phase 1 trials test a new treatment for the first time in humans, focusing on safety, dosing, and how the body processes the drug. For ALS (Amyotrophic Lateral Sclerosis), a Phase 1 study typically enrolls a small number of participants — often healthy volunteers or patients who have exhausted standard treatment options. Phase 1 results determine whether a treatment moves into larger Phase 2 efficacy studies.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

With a target enrollment of 15 participants, this is a small study — typical of early-phase research, rare-disease trials, or pilot studies designed to generate preliminary signal before a larger study is launched.

Who May Be Eligible (Plain English)

Who May Qualify: - 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 Should NOT Join This Trial: - 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 weakened immune system virus (HIV) antibody. - Inyour organs (liver, kidneys, etc.) are working well enough based on blood tests - Any participant with a current open tracheostomy Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language

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

Treatments Being Tested

DRUG

AAV9 Gene therapy

Single dose gene therapy

Locations (1)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

Macquarie University Hospital

Sydney, New South Wales, Australia

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT07401121), the sponsor (Celosia Therapeutics Pty Ltd), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT07401121 clinical trial studying?

This clinical study is in participants with Amyotrophic Lateral Sclerosis and is designed to evaluate the safety and tolerability of the gene therapy CTx1000. The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT07401121?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT07401121?

Contact information for this trial may be available directly on the ClinicalTrials.gov record. Click "View on ClinicalTrials.gov" in the sidebar for the official source. Always discuss any potential trial with your doctor before contacting the study site.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT07401121. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT07401121. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.