NCT07401121

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

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 15 (estimated)
• Sponsor: Celosia Therapeutics Pty Ltd · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

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.

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)

Interventions

Type	Name	Description
DRUG	AAV9 Gene therapy	Single dose gene therapy

Timeline

• Start date: 2026-01-12
• Primary completion: 2027-12-01
• Completion: 2030-12-01
• First posted: 2026-02-10
• Last updated: 2026-02-10

Locations

1 site across 1 country: Australia