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Keep this study
Lead
Celosia Therapeutics Pty Ltd
With
Novotech (Australia) Pty Limited
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).
Age
18–80
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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
You may not be if
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
Clareo Health | Safety and Tolerability Study of CTx1000 In Participants With Amyotrophic Lateral Sclerosis
