VTx-002 for ALS
This study is testing an investigational gene therapy called VTx-002 for people with Amyotrophic Lateral Sclerosis (ALS). Before receiving VTx-002, you will be given a steroid medicine called Methylprednisolone to help prevent reactions. The main goal is to see how safe VTx-002 is and what side effects people experience over five years. Researchers will also look at how well people tolerate the treatment. This is a first-in-human study, meaning it's the first time VTx-002 is being given to people. To join, you must be at least 18 years old and have a diagnosis of ALS. The study is currently unclear on its recruitment status.
- Study design
- This is a Phase 1/2, open-label study with 12 planned participants. It will test two different doses of VTx-002.
- What's involved
- You will receive a single injection of VTx-002. There will be 12 visits to the study center in the first year, including an overnight stay, plus 4 remote visits. From year 2-5, there will be 8 additional visits, either in-person or remote.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for over 5 years after treatment to monitor for side effects.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
At a glance
Conditions
NCT07287397
Where you'd take part
This study runs at 11 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Herbert Irving Comprehensive Cancer Center
New York, New Yorkstudy coordinator listed
Not yet recruiting
Kings College Hospital
London, United Kingdomstudy coordinator listed
Not yet recruiting
Mayo Clinic in Florida
Jacksonville, Floridastudy coordinator listed
Recruiting
Royal Hallamshire Hospital
Sheffield, United Kingdomstudy coordinator listed
Recruiting
Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital
Boston, Massachusettsstudy coordinator listed
Recruiting
St Joseph's Hospital and medical Center - Barrow Neurological Institute
Phoenix, Arizonastudy coordinator listed
Not yet recruiting
UMC Utrecht
Utrecht, Netherlandsstudy coordinator listed
Recruiting
University of California San Diego Medical Center
San Diego, Californiastudy coordinator listed
Recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
What this trial measures
- The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)Over 5 years
Assessed by reviewing the nature, incidence, severity, relatedness, seriousness and outcome of treatment emergent adverse events.
- The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)Over 5 years
Assessed by reviewing laboratory values
- The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)Over 5 years
Assessed by reviewing Magnetic Resonance Imaging (MRI) findings.
- The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)over 5 years
Assessed by review of Treatment Induced Peripheral Neuropathy Assessment Scale (TNAS) \*TNAS a brief participant-reported questionnaire used to measure the severity and progression of peripheral neuropathy. Each item is rated on a scale of 0 to 10, where 0 means no symptom and 10 means the symptom is as bad as it can possibly be.
- The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)Over 5 years
Assessed by reviewing cellular responses by analyzing blood samples.
- The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)Over 5 years
Assessed by reviewing scores of the Columbia Suicide Severity Rating Scale (C-SSRS) \*C-SSRS is a widely used, evidence-based suicide risk assessment tool that helps identify whether someone is at risk for suicide and gauge the level of support needed. A low score is associated with a lower risk level in this brief questionnaire.
- The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)At month 6 and month 12
Assessed by performing 4 formal interim analyses 1. 6 months after Gene Therapy Group 1 receive the single dose treatment 2. 6 months after Gene Therapy Group 2 receive the single dose treatment 3. 12 months after Gene Therapy Group 1 receive the single dose treatment 4. 12 months after Gene Therapy Group 2 receive the single dose treatment