Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients
Study Details
Study Description
Brief Summary
The objective of this study is to increase the understanding surrounding the choices presented to patients and families impacted by Fabry disease.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
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.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| ERT User- Did Not Switch to Galafold ERT users with mutation amenable to Galafold who did not switch |
Other: 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 |
Other: 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 |
Other: 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. |
Other: 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 |
Other: 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 |
Other: This is a non-interventional study
This is a non-interventional study
|
Outcome Measures
Primary Outcome Measures
- 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 Criteria
Criteria
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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Engage Health | Eagan | Minnesota | United States | 55121 |
Sponsors and Collaborators
- Amicus Therapeutics
- Engage Health Inc.
Investigators
- Principal Investigator: Niloofar Nobakht, MD, Ronald Regan UCLA Medical Center
Study Documents (Full-Text)
None provided.More Information
Additional Information:
- Galafold and Fabry Disease
- Release Date for Galafold
- Review of Fabry Disease
- About Fabry Disease
- About Fabry Disease
Publications
None provided.- AT-NIS-00002