A Gaucher Disease Gene Therapy Trial With FLT201

To join this study, you must be at least 18 years old and have been diagnosed with Gaucher disease type 1. You also need to have been on a stable treatment for your Gaucher disease (either ERT or SRT) for at least two years without breaks. Your blood tests, like your haemoglobin and platelet levels, should be steady and within a healthy range when the study starts.

There are also some reasons why you wouldn't be able to join. For example, if you have a different type of Gaucher disease (Type 2 or 3), you won't be eligible. You also can't take part if you're pregnant or breastfeeding, or if you've had certain major surgeries in the past, like a bone marrow transplant or any gene therapy. Some other medical conditions or specific past treatments would also prevent you from joining, as the researchers need to make sure the study is safe for everyone involved.

If you join this study, you would first go through a screening process to make sure you meet all the requirements. If you're eligible, you would receive the FLT201 gene therapy. After getting the treatment, a key part of the study involves gradually stopping your current ERT or SRT treatment, under close medical supervision. You will have regular visits to the clinic for blood tests, physical examinations, and other assessments to check your health and how the treatment is working. The study is particularly interested in how your body responds to stopping your usual medication after receiving FLT201. The total duration of your participation in the study, including follow-up visits, would be discussed with you by the study team.