RecruitingPhase 1NCT07401121

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

Sponsor

Celosia Therapeutics Pty Ltd

Enrollment

15 participants

Start Date

Jan 12, 2026

Study Type

INTERVENTIONAL

Conditions

ALS (Amyotrophic Lateral Sclerosis)

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.

Eligibility

Min Age: 18 YearsMax Age: 80 Years

Inclusion Criteria6

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 Criteria5

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

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