Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

NCT04804566 — Completed, 44 participants, Amicus Therapeutics

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Registry	NCT04804566
Status	Completed
Study type	Observational
Enrollment	44 (Actual)
Sponsor	Amicus Therapeutics (Industry); collaborators: Engage Health Inc.
Conditions	Fabry Disease
Interventions	This is a non-interventional study (Other)
Dates	start 2023-03-01, primary completion 2023-11-01, completion 2023-11-01, first posted 2021-03-18, last updated 2024-04-03
Locations	1 site in United States

-Summary

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

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+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 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.

+Arms and interventions

Arm	Interventions	Description
ERT User- Did Not Switch to Galafold	Other: This is a non-interventional study	ERT users with mutation amenable to Galafold who did not switch
ERT User- Switched and Stayed on Galafold	Other: This is a non-interventional study	ERT users with the mutation amenable to Galafold who switched and stayed on Galafold
No Previous Therapy- Started Galafold and Stayed On	Other: This is a non-interventional study	Those naïve to therapy with the mutation amenable to Galafold who went on and stayed on Galafold
No Previous Therapy- No Current Therapy	Other: This is a non-interventional study	Those who were naïve to therapy with the mutation amenable to Galafold and have never been on any therapy.
ERT Users- Switched and Discontinued Galafold	Other: This is a non-interventional study	Participants who are ERT users with an amenable mutation who switched to and later discontinued Galafold
No Previous Therapy- Started Galafold and Discontinued	Other: This is a non-interventional study	Participants who are naïve to therapy with an amenable mutation, went on Galafold, and discontinued

This is a non-interventional study (Other): This is a non-interventional study

+Outcome measures

Primary outcomes

Patterns and Trends that Provide Evidence and Context for the Treatment Choices and Experiences of Those with Fabry Disease [1-2 months]: 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.

+Eligibility

Sex: All, ages: 18 Years to any

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)

Facility	City	Country	Status
Engage Health	Eagan, Minnesota	United States

+Investigators

Niloofar Nobakht, MD, Principal investigator, Ronald Regan UCLA Medical Center