Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients
1 other identifier
observational
44
1 country
1
Brief Summary
The objective of this study is to increase the understanding surrounding the choices presented to patients and families impacted by Fabry disease.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for all trials
Started Mar 2023
Shorter than P25 for all trials
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 16, 2021
CompletedFirst Posted
Study publicly available on registry
March 18, 2021
CompletedStudy Start
First participant enrolled
March 1, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 1, 2023
CompletedStudy Completion
Last participant's last visit for all outcomes
November 1, 2023
CompletedApril 3, 2024
April 1, 2024
8 months
March 16, 2021
April 2, 2024
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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
Study Arms (6)
ERT User- Did Not Switch to Galafold
ERT users with mutation amenable to Galafold who did not switch
ERT User- Switched and Stayed on Galafold
ERT users with the mutation amenable to Galafold who switched and stayed on Galafold
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
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.
ERT Users- Switched and Discontinued Galafold
Participants who are ERT users with an amenable mutation who switched to and later discontinued Galafold
No Previous Therapy- Started Galafold and Discontinued
Participants who are naïve to therapy with an amenable mutation, went on Galafold, and discontinued
Interventions
This is a non-interventional study
Eligibility Criteria
Purposive sampling techniques will be used which will seek to initially recruit 30 participants (person with Fabry or their parent/legal guardian) from each of four groups noted below: 1. ERT users with an amenable mutation, who did not switch 2. ERT users with an amenable mutation, who switched to Galafold and stayed on 3. Those naïve to therapy with an amenable mutation, who went on Galafold and stayed on 4. Those naïve to therapy with an amenable mutation, who have never been on any therapy. There will also be an attempt to recruit 5 patients from each of the two groups: 1. ERT users with an amenable mutation, who switched and discontinued Galafold, and 2. Those naïve to therapy, with an amenable mutation, who went on Galafold and discontinued. In order to get a geographic sampling, the first four groups will attempt to recruit 30 patients/parents or caregivers of patients with representation from Germany, the UK, and the US.
You may qualify if:
- 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).
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Amicus Therapeuticslead
- Engage Health Inc.collaborator
Study Sites (1)
Engage Health
Eagan, Minnesota, 55121, United States
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Niloofar Nobakht, MD
Ronald Regan UCLA Medical Center
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 16, 2021
First Posted
March 18, 2021
Study Start
March 1, 2023
Primary Completion
November 1, 2023
Study Completion
November 1, 2023
Last Updated
April 3, 2024
Record last verified: 2024-04
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- SAP, ANALYTIC CODE
The pseudonymized answers provided by each respondent will be combined with those of others participating in the study and the pseudonymized database (Excel format) will be locked and provided to Engage Health personnel for analysis ("Study Data"). 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.