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Tests treatment safety and results for ALS (Amyotrophic Lateral Sclerosis)
Official title Safety and Tolerability Study of CTx1000 In Participants With Amyotrophic Lateral Sclerosis
ClinicalTrials.gov ID: NCT07401121
What this study is testing
What is AAV9 Gene therapy?
AAV9 Gene therapy is an investigational medicine, being studied as a potential treatment for als (amyotrophic lateral sclerosis).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This clinical study is in participants with Amyotrophic Lateral Sclerosis and is designed to evaluate the safety and tolerability of the gene therapy CTx1000.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 80
You may be able to join 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
You likely can't join if
- Any participants with genetic forms of ALS, including C9ORF72 repeat carriers, except for TARDBP gene variants, as confirmed by previous clinical...
- 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
See the full eligibility 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
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Sydney, New South Wales, Australia
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 80 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Sydney, New South Wales, Australia. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.