Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients
NCT ID: NCT04804566
Last Updated: 2024-04-03
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
44 participants
OBSERVATIONAL
2023-03-01
2023-11-01
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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ERT User- Did Not Switch to Galafold
ERT users with mutation amenable to Galafold who did not switch
This is a non-interventional study
This is a non-interventional study
ERT User- Switched and Stayed on Galafold
ERT users with the mutation amenable to Galafold who switched and stayed on Galafold
This is a non-interventional study
This is a non-interventional study
No Previous Therapy- Started Galafold and Stayed On
Those naïve to therapy with the mutation amenable to Galafold who went on and stayed on Galafold
This is a non-interventional study
This is a non-interventional study
No Previous Therapy- No Current Therapy
Those who were naïve to therapy with the mutation amenable to Galafold and have never been on any therapy.
This is a non-interventional study
This is a non-interventional study
ERT Users- Switched and Discontinued Galafold
Participants who are ERT users with an amenable mutation who switched to and later discontinued Galafold
This is a non-interventional study
This is a non-interventional study
No Previous Therapy- Started Galafold and Discontinued
Participants who are naïve to therapy with an amenable mutation, went on Galafold, and discontinued
This is a non-interventional study
This is a non-interventional study
Interventions
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This is a non-interventional study
This is a non-interventional study
Eligibility Criteria
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Inclusion Criteria
* 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).
18 Years
ALL
No
Sponsors
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Engage Health Inc.
INDUSTRY
Amicus Therapeutics
INDUSTRY
Responsible Party
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Principal Investigators
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Niloofar Nobakht, MD
Role: PRINCIPAL_INVESTIGATOR
Ronald Regan UCLA Medical Center
Locations
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Engage Health
Eagan, Minnesota, United States
Countries
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Related Links
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Galafold and Fabry Disease
Release Date for Galafold
Review of Fabry Disease
Other Identifiers
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AT-NIS-00002
Identifier Type: -
Identifier Source: org_study_id
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