Status:
COMPLETED
Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients
Lead Sponsor:
Amicus Therapeutics
Collaborating Sponsors:
Engage Health Inc.
Conditions:
Fabry Disease
Eligibility:
All Genders
18+ years
Brief Summary
The objective of this study is to increase the understanding surrounding the choices presented to patients and families impacted by Fabry disease.
Detailed Description
This will be a cross-sectional study conducted in approximately 130 individuals (or representative parents/caregivers of patients) living with Fabry disease. All study participants will complete the R...
Eligibility Criteria
Inclusion
- 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).
Exclusion
- Inability to meet any of the inclusion criteria
Key Trial Info
Start Date :
March 1 2023
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
November 1 2023
Estimated Enrollment :
44 Patients enrolled
Trial Details
Trial ID
NCT04804566
Start Date
March 1 2023
End Date
November 1 2023
Last Update
April 3 2024
Active Locations (1)
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1
Engage Health
Eagan, Minnesota, United States, 55121