This clinical study is in participants with Amyotrophic Lateral Sclerosis and is designed to evaluate the safety and tolerability of the gene therapy CTx1000.
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
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
Sequential
AAV9 Gene therapy
DrugSingle dose gene therapy
To evaluate the safety and tolerability of CTx1000 in ALS diagnosed participants
Incidence of Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interests (AESIs)
To evaluate the long-term safety and tolerability of CTx1000 in ALS diagnosed participants
Incidence of Treatment Emergent Adverse Events (TEAEs), Severe Adverse Events (SAEs) and Adverse Events of Special Interests (AESIs)
To evaluate the long-term pharmacodynamics and immunogenicity of CTx1000 in ALS diagnosed participants
Changes in pre-dose biomarkers (Neurofilament light) in cerebrospinal fluid and anti-drug antibody (ADA)
To evaluate the long-term efficacy of CTx1000 in ALS diagnosed participants
Changes (reduction) from pre-dose Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)
Click on the lead sponsor to view all of their trials.
Celosia Therapeutics Pty Ltd
Lead sponsor
Novotech (Australia) Pty Limited
Collaborator
