Safety and Tolerability Study of CTx1000 In Participants With Amyotrophic Lateral Sclerosis (KOANEWA)

Celosia Therapeutics Pty Ltd

Status and phase

Enrolling

Phase 1

Conditions

ALS (Amyotrophic Lateral Sclerosis)

Treatments

Drug: AAV9 Gene therapy

Study type

Interventional

Funder types

Industry

Identifiers

NCT07401121

ALS-AAV9

Details and patient eligibility

About

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

Full 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).

Enrollment

15 estimated patients

Sex

All

Ages

18 to 80 years old

Volunteers

No Healthy Volunteers

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