Active not recruitingPHASE1, PHASE2INTERVENTIONAL

Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

This study, called GALILEO-2, is a long-term follow-up for individuals in the UK who have Type 1 Gaucher disease and previously took part in a clinical trial where they received a treatment named FLT201. The main aim is to carefully monitor the health of these participants for five years after their treatment. This helps researchers understand the longer-term effects of FLT201, even if some individuals needed to restart or begin other treatments for their Gaucher disease. It's a crucial step in learning more about how new treatments for Gaucher disease work over time.

At a glance

Status

Active not recruiting

Phase

PHASE1, PHASE2

Sponsor

Spur Therapeutics

Enrolment target

Start

13 May 2024

Estimated completion

01 May 2029

Gaucher Disease, Type 1

What is this study about?

This study, known as GALILEO-2, is a long-term monitoring project for people with Type 1 Gaucher disease who have previously been involved in a clinical trial testing a treatment called FLT201. Think of it as a check-in to see how they are doing over an extended period after receiving the experimental treatment. The study aims to follow these individuals for five years.

Gaucher disease is a rare inherited condition. In Type 1 Gaucher disease, certain fatty substances build up in organs like the spleen, liver, and bone marrow, which can cause various health problems. Treatments aim to manage these symptoms and prevent further damage. FLT201 is a new type of treatment being investigated for this condition.

By following participants for a longer time, researchers can gather important information about the lasting effects of FLT201. This includes understanding its long-term safety and whether it continues to influence the course of the disease over several years. This kind of follow-up is really important for fully understanding a new treatment and helping future patients.

Key takeaways

This study is a long-term follow-up for people with Type 1 Gaucher disease.
Participants previously received the experimental treatment FLT201 in an earlier trial.
The study will monitor participants' health for five years.
It aims to understand the long-term effects and safety of FLT201.
This research helps inform future treatments for Gaucher disease.

Who may be eligible?

This study is open to adults who have Type 1 Gaucher disease and previously took part in another clinical trial where they received the FLT201 treatment.

Even if you've had to restart or begin other common treatments for Gaucher disease (like Enzyme Replacement Therapy or Substrate Reduction Therapy) after your FLT201 treatment, you could still be eligible.

To join, you must be able to understand the study information and provide your full permission to take part. You also need to be able to comply with all the study requirements.

Quick self-check

Are you an adult aged 18 or over?
Do you have Type 1 Gaucher disease?
Did you previously take part in a clinical trial where you received FLT201?
Are you able to provide your informed consent?
Are you able to follow the study requirements?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you join this study, you will be monitored for about five years after your previous FLT201 treatment. This will involve regular visits to a clinic at various times, though the exact schedule will be explained in detail. During these visits, the study team will carry out health assessments, which may include physical examinations, blood tests, and scans, to check on your overall health and how your Gaucher disease is progressing. You won't be receiving FLT201 treatment again as part of this specific study; instead, the focus is on long-term observation. All information gathered will help the researchers understand the long-term effects of FLT201.

Potential risks and benefits

The main benefit of taking part is contributing valuable long-term information that could help others with Type 1 Gaucher disease in the future. It allows researchers to understand the lasting effects of FLT201, which is crucial for developing and approving new treatments. As this is a follow-up study, you won't be exposed to new experimental drugs. The main risks are generally low, involving the time commitment for visits and standard procedures like blood tests, which rarely have minor side effects such as bruising. You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (4)

Lysosomal Rare Disorders Research and Treatment Center
Fairfax, United States
Hospital de Clinicas de Porto Alegre (HCPA)
Porto Alegre, Brazil
Hospital Quironsalud Zaragoza
Zaragoza, Spain
Salford Royal Hospital
Salford, United Kingdom

Common questions

What is Gaucher disease Type 1?

Gaucher disease Type 1 is a rare inherited condition where a specific fatty substance builds up in cells, leading to problems in organs like the spleen, liver, and bones.

What is FLT201?

FLT201 is an experimental treatment that was previously being tested for Type 1 Gaucher disease in an earlier clinical trial.

Why is this follow-up study important?

This study is important because it helps researchers understand the long-term effects and safety of FLT201, which is vital for developing new treatments for Gaucher disease.

Will I receive the FLT201 treatment again in this study?

No, this study is only for monitoring. You will not receive FLT201 treatment as part of this follow-up study.

Can I leave the study at any time?

Yes, you are completely free to leave the study at any point without it affecting your usual medical care.