Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

Fabry Disease Clinical Trial

Official title:

Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04804566
• Other study ID #: AT-NIS-00002
• Status: Completed
• Start date: March 1, 2023
• Est. completion date: November 1, 2023

Study information

• Verified date: April 2024
• Source: Amicus Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

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 RSVP followed by a structured interview conducted by trained interviewers. It is estimated that each respondent will need up to 60 minutes for the entire process; 10 minutes to complete the RSVP including uploading the proof of Fabry disease diagnosis or verifying membership with Fabry groups, including but not limited to: Fabry Support and Information Group, National Fabry Disease Foundation, MPS Society UK, Morbus Fabry Selbsthilfergruppe, Fabry International Network, or others, and approximately 50 minutes to complete the interview.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 44
• Est. completion date: November 1, 2023
• Est. primary completion date: November 1, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• 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 Criteria:

• Inability to meet any of the inclusion criteria

Related Conditions & MeSH terms

• Fabry Disease

Intervention

Other:
• This is a non-interventional study
This is a non-interventional study

Locations

Country	Name	City	State
United States	Engage Health	Eagan	Minnesota

Sponsors (2)

Lead Sponsor	Collaborator
Amicus Therapeutics	Engage Health Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Patterns and Trends that Provide Evidence and Context for the Treatment Choices and Experiences of Those with Fabry Disease	The goal of the statistical analysis is to uncover patterns and trends that provide both evidence and context for the treatment choices and experiences of patients and families impacted by Fabry disease.; All findings will be summarized in the final report, which will not identify any respondent as described above. At the conclusion of this study, the researchers may publish their findings in a medical / scientific journal.	1-2 months

External Links

•   About Fabry Disease
•   About Fabry Disease
•   Galafold and Fabry Disease
•   Release Date for Galafold
•   Review of Fabry Disease