Safety and Tolerability Study of CTx1000 In Participants With Amyotrophic Lateral Sclerosis
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: AAV9 Gene therapy.
- Who it may be relevant to
- Registry conditions: ALS (Amyotrophic Lateral Sclerosis). Basic parameters: 18 years — 80 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Australia
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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
Overview
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).
Interventions
- Drug AAV9 Gene therapy
Single dose gene therapy
Primary outcome measures
- To evaluate the safety and tolerability of CTx1000 in ALS diagnosed participants [Time frame: 52 weeks]
Secondary outcome measures (3)
- To evaluate the long-term safety and tolerability of CTx1000 in ALS diagnosed participants [Time frame: 3 years]
- To evaluate the long-term pharmacodynamics and immunogenicity of CTx1000 in ALS diagnosed participants [Time frame: 3 years]
- To evaluate the long-term efficacy of CTx1000 in ALS diagnosed participants [Time frame: 3 years]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Australia · 1 center
- Macquarie University Hospital — Sydney
Identifiers
NCT: NCT07401121 · ALS-AAV9