Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

Completed

Conditions studied: Fabry Disease

In brief

The objective of this study is to increase the understanding surrounding the choices presented to patients and families impacted by Fabry disease.

Key facts

Study ID

NCT04804566

Run by

Amicus Therapeutics

People needed

44

Starts

2023-03-01

Expected to finish

2023-11-01

Last updated by the study team

2024-04-03

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Participant must be a person with Fabry disease who is 18 years or older or the parent/legal guardian of a living person with Fabry disease who is under the age of 18 years or who are 18 years and older who are unable to answer for themselves.
• Confirmed diagnosis of Fabry disease with written proof of disease provided
• Must have a genetic mutation that is amenable to oral therapy
• Resident of Germany, the U.K or the U.S.
• Able to read, write and communicate in German, or English.
• Able to grant informed consent
• Willing to participate in a 50 to 60-minute telephone interview, including follow up questions (if necessary) and information regarding adverse events (if necessary).

You may not qualify if…

• Inability to meet any of the inclusion criteria

Where it is running

• Engage Health — Eagan, Minnesota, United States

Full record on ClinicalTrials.gov

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