A Phase I/II, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects with Fabry Disease.
This research study is investigating a new gene therapy called ST-920 for men with Fabry disease. Fabry disease is a rare condition where the body can't produce enough of an important enzyme. The ST-920 treatment aims to deliver a healthy gene to help the body make this missing enzyme. This is an early-stage study (Phase I/II), meaning it's one of the first times this treatment is being given to people. The main goal is to find out if ST-920 is safe and how well people tolerate it. Researchers will also be looking for early signs that the treatment is working as intended, by checking enzyme levels and how the treatment affects the body over one year. The study will involve careful monitoring of participants' health.
At a glance
What is this study about?
Fabry disease is a genetic condition that often runs in families. It happens when your body doesn't make enough of an important enzyme called alpha-galactosidase A (α-Gal A). This missing enzyme leads to a build-up of a fatty substance in different parts of your body, which can cause serious problems with your heart, kidneys, and nervous system over time. Current treatments can help manage the symptoms, but they don't fix the underlying genetic problem.
This study is trying a new approach called 'gene therapy' using a treatment called ST-920. Gene therapy aims to correct the genetic problem by delivering a healthy copy of the gene that makes the α-Gal A enzyme. The idea is that if your body can start making this enzyme again, it could prevent the fatty build-up and improve your health. This is a very early study, called a Phase I/II trial, which means it's among the first times this treatment is being given to people. So, the main focus is on making sure the treatment is safe and understanding how the body reacts to it.
Throughout the study, doctors will closely monitor participants. They will be looking for any side effects and checking various health markers like blood test results, heart function, and kidney function. They will also measure the levels of the enzyme and the fatty substances to see if the treatment is having the desired effect. The goal is to gather information that will help decide if ST-920 could be a promising new treatment for Fabry disease in the future.
Key takeaways
- This is an early-stage study (Phase I/II) of a new gene therapy for Fabry disease.
- The treatment, ST-920, aims to help the body make a missing enzyme.
- The main focus is to check how safe the treatment is for adult men with Fabry disease.
- Participants will receive a single dose and be closely monitored for one year.
- Potential benefits include a new treatment approach, but risks are also present.
- You have the right to leave the study at any point.
Who may be eligible?
This study is specifically designed for adult men who have Fabry disease. To join, you must be at least 18 years old.
The research team will also check other health factors to make sure it's safe for you to take part and that the study results are clear. This includes reviewing your medical history and current health status.
Because this is an early study of a new treatment, there are strict rules about who can join. The study doctors will go through all the requirements with you to see if you are a good fit.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you an adult aged 18 or over?
- Are you a male?
- Have you been diagnosed with Fabry disease?
- Are you able to attend regular clinic appointments for one year?
- Are you able to understand and agree to the study requirements?
What does participation involve?
If you join this study, you will receive a single dose of the ST-920 gene therapy. Your health will be very carefully monitored for one year after receiving the treatment. This will involve regular visits to the clinic.
During these visits, you can expect to have various assessments. These will include blood tests to check your general health, enzyme levels, and other substances related to Fabry disease. You will also have heart checks like an electrocardiogram (ECG) and an echocardiogram (ECHO), which look at your heart's electrical activity and structure. Kidney function will be assessed, and you might have MRI scans of your liver to check for any changes. Your vital signs, like blood pressure and pulse, will be taken regularly.
The purpose of all these tests is to closely watch for any side effects and to see how the treatment is affecting your body. The study will last for a total of one year, with continued follow-up after the treatment.
Potential risks and benefits
Locations (2)
- —UnverifiedItaly
- —UnverifiedGermany
Common questions
What is gene therapy?
Gene therapy is a treatment that aims to fix a health problem by delivering a healthy gene into the body to replace or help a faulty one.
Why is this study only for men?
Fabry disease is an X-linked condition, meaning it affects men differently and often more severely than women. This early study focuses on men first.
What is a Phase I/II study?
This means it's an early-stage human trial primarily focused on checking the safety of the new treatment and finding the right dose, rather than solely on how well it works.
Will I know if the treatment is working?
The study will measure changes in enzyme levels and other health markers, which can give early indications, but the main goal is safety at this stage.
How long will I be in the study?
You will receive a single dose of the treatment and then be closely monitored for a period of one year.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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