Image

A Study of Patients With Fabry Disease (US Specific)

Join a study for Fabry Disease patients in the US.

Recruiting
18 years and older
All
Phase N/A

This study is for people with Fabry disease, a genetic disorder affecting how your body processes fats. It aims to understand how well different treatments work, focusing mainly on migalastat, a type of medicine. The study will include at least 450 people worldwide, with three groups: those treated with migalastat, those treated with enzyme replacement therapy (ERT), and those not yet treated. Participants will be observed for up to 5 years to check the effectiveness and safety of these treatments and their impact on quality of life.

  • The study will last for 5 years, and participants will have regular follow-ups.
  • Participants must be 18 or older, have specific genetic markers, and not be part of another trial.
  • Participants must understand and agree to the study details.

If you have Fabry disease and are considering joining, talk to your doctor about whether this study is right for you. Participation involves regular check-ins and possibly changing or starting a new treatment for your condition.

Study details
    Fabry Disease

NCT06906367

Amicus Therapeutics

7 March 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

Investigator Avatar

Primary Contact

  Other languages supported:

First name*
Last name*
Email*
Phone number*
Race
Ethnicity
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.