GALILEO-3

FLT201: AN INVESTIGATIONAL ONE-TIME THERAPY

THE GALILEO-3 GAUCHER CLINICAL Trial

If you’ve been living with Gaucher disease type 1 (GD1), you’re all too familiar with its effects. If you’re taking one of the currently approved therapies for GD1, you may have a lifetime of treatments ahead of you, and even with treatment, you may still have symptoms.

The GALILEO-3 clinical trial is studying an investigational one-time gene therapy that has been designed to continuously deliver the enzyme missing in people with Gaucher disease.

YOU MAY BE HERE BECAUSE …

You’ve lived with Gaucher disease type 1 for most of your life.
You’re looking for an alternative to your current treatment.
You’re looking for an alternative treatment option for your loved one.
You’re interested in the possibility of a one-time treatment for Gaucher.
You understand that being part of a study may help the Gaucher community.
You’re interested in contributing to valuable research that may lead to an alternative treatment option.
You want access to investigational treatments that are not yet available to the general public.

Are you living with Gaucher disease type 1?
See if you qualify for the GALILEO-3 clinical trial.

DO I QUALIFY?

ABOUT THE GALILEO-3 GAUCHER TRIAL

WHY THE STUDY IS IMPORTANT

Clinical trials play an essential role in the development of new treatment options. The GALILEO-3 clinical trial seeks to gather data about FLT201, an investigational one-time gene therapy for adults with Gaucher disease type 1.

PURPOSE OF THE STUDY

GALILEO-3 builds on earlier research that studied the safety of FLT201 in patients with Gaucher disease type 1. This trial will continue to assess the safety and efficacy of the investigational therapy and determine how well it works in a larger group of people. FLT201 is designed to provide a more stable form of the enzyme, which could potentially improve disease management and reduce the need for ongoing treatments. The findings could support the advancement of care and may provide an alternative treatment option for the Gaucher community.

DO I QUALIFY?

You may be eligible to participate in
the GALILEO-3 trial if:

	You are diagnosed with Gaucher disease type 1.
	You are on ERT or SRT and have been for at least 2 years.
	You have not had a total splenectomy.
	You are 18 years or older.
	You are not pregnant or nursing.

FIND A SITE

WHAT CAN PARTICIPANTS EXPECT?

GALILEO-3: Investigation of a one-time gene therapy

1 CONSENT:
Prior to taking part in the study, you will have an in-depth conversation with the clinical study team to review the details of the study to ensure you understand and agree to all aspects of trial participation. You will be informed of the potential risks and benefits of participating so you can determine if the study is right for you.
2 SCREENING:
During the screening period, you will receive various health assessments to ensure you meet the requirements to join the study, including a discussion of the potential use of immunosuppressive treatment as part of the study.
3 INFUSION:
Under the care of the clinical study team, you will receive a one-time intravenous (IV) infusion of the study treatment. While the infusion takes only 1-2 hours, there are additional tests and assessments done before and after as part of the clinical trial.
4 TRANSITION OFF CURRENT TREATMENT:
At around four weeks after the infusion, your doctor will advise the right time to stop taking your usual treatment. This allows us to assess how well FLT201 works on its own.
5 FOLLOW-UP:
Your medical team will monitor how you are doing on an ongoing basis for five years by asking how you are feeling and conducting blood tests, imaging, scans, and questionnaires. These check-ins are more frequent at first and then decrease in frequency over time.

US TRIAL LOCATIONS*

More information and additional enrolling trial sites can be found at clinicaltrials.gov.

*Additional sites coming soon.

ARE YOU A PHYSICIAN TREATING PEOPLE WITH GAUCHER DISEASE?

	If you would like to know more about our FLT201 program, please reach out to clinicaltrials@spurtherapeutics.com.
	Do you have a patient interested in participating in the clinical trial? Contact the closest clinical trial site to learn more about referring your patient.

FAQ

What is a clinical trial?

A clinical trial is a research study that investigates a medical treatment or therapy in people. The findings of a trial will be used to determine how safe and effective a treatment is and how it can be best utilized.

How long will my participation last?

Your treatment team will closely monitor you during the first year after you receive the investigational therapy. During the first three months, visits are required more frequently and after this, the visits become less frequent. All participants who received the therapy will be followed for up to 5 years.

Would I have to travel to the site to be assessed?

Yes. There are a number of essential tests that must be completed at the trial center, however some can be done at your home. You will not be responsible for travel costs, and additional support for travel is available upon request; however, it is important to consider how participating in the study may fit into your lifestyle and other commitments.

What are my options if I do not see a trial site near me, but want to be considered for the study?

We continue to add trial sites to the list so check back again for a center that might work for you. If this study doesn’t work for you, there may be other opportunities to be involved in research. Talk to your current doctor about your interest in the trial as they can also contact the trial sponsor, Spur Therapeutics, to inquire about treatment options.

Are there any costs for participating in the trial?

The investigational therapy, medical appointments and assessments, and any travel required to participate in the study are provided at no cost to you.

What else should I know about participation?

Participation in a clinical trial has both potential benefits and risks, the full details of these will be provided by a doctor at the clinical trial site.

FLT201 is an investigational one-time gene therapy, at this stage in its development not everything is known about it, which is important to understand before enrolling.

There may not be a way to reverse whatever effect FLT201 may have, whether it is a positive effect, a neutral effect (no effect), or a negative effect. Participating in this study may mean you could be excluded from future gene therapy trials or treatments. Talk to your doctor about any concerns you may have.

This website is intended for US audiences only and is to provide GALILEO-3 clinical study information only and relates to an un-approved medicinal product or therapy use. The information included in this site should not be construed as promotion or recommendation of any approved or un-approved product, nor replace the advice of a healthcare professional.