Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

Amicus Therapeutics44 enrolled

Overview

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

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Fabry Disease

Interventions

This is a non-interventional studyOTHER

This is a non-interventional study

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (1)

Engage Health

Eagan, Minnesota, United States

Outcomes

Primary Outcomes

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.

Time frame: 1-2 months

Data from ClinicalTrials.gov

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