Image

A Gaucher Disease Gene Therapy Trial With FLT201

Participate in a study for Gaucher Disease gene therapy.

Recruiting
18 years and older
All
Phase 3

**Gaucher Disease Gene Therapy Trial**

This study is for adults with Gaucher disease Type 1, a condition affecting how your body breaks down a certain fatty substance. Currently, patients in the study must have been on regular enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least 2 years. The study will test a new treatment called FLT201 to see if it's safe and works well when ERT/SRT is stopped.

Key Points:

  • The study is a Phase 3 trial, meaning it's in the later stages of research and involves multiple locations.
  • Participants must be at least 18 years old, have Gaucher disease Type 1, and have stable blood levels.
  • Those who have had certain transplants, or who are pregnant, are not eligible.

This study aims to provide a new treatment option for Gaucher disease Type 1 through gene therapy, which involves using genes to treat or prevent disease. Participants will help researchers understand if FLT201 can replace current therapies safely and effectively.

Study details
    Gaucher Disease Type 1

NCT07223944

Spur Therapeutics

17 October 2026

Step 1 Get in touch with the nearest study center
We have submitted the contact information you provided to the research team at {{SITE_NAME}}. A copy of the message has been sent to your email for your records.
Would you like to be notified about other trials? Sign up for Patient Notification Services.
Sign up

Send a message

Enter your contact details to connect with study team


Email

View email

Phone

Phone country flag

View phone
Investigator Avatar

Primary Contact

  Other languages supported:

First name*
Last name*
Email*
Phone number*
Race*
Are you a healthy volunteer
Ethnicity*
Are you a healthy volunteer
Other language

FAQs

Learn more about clinical trials

What is a clinical trial?

A clinical trial is a study designed to test specific interventions or treatments' effectiveness and safety, paving the way for new, innovative healthcare solutions.

Why should I take part in a clinical trial?

Participating in a clinical trial provides early access to potentially effective treatments and directly contributes to the healthcare advancements that benefit us all.

How long does a clinical trial take place?

The duration of clinical trials varies. Some trials last weeks, some years, depending on the phase and intention of the trial.

Do I get compensated for taking part in clinical trials?

Compensation varies per trial. Some offer payment or reimbursement for time and travel, while others may not.

How safe are clinical trials?

Clinical trials follow strict ethical guidelines and protocols to safeguard participants' health. They are closely monitored and safety reviewed regularly.
Add a private note
  • abc Select a piece of text.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.