Clinical trial · Observational
Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients
NCT04804566CI-TRIAL-00075693completedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
The objective of this study is to increase the understanding surrounding the choices presented to patients and families impacted by Fabry disease.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Fabry Disease | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| This is a non-interventional study | Other | — | UNRESOLVED |
Design
Arms and outcomes
Arms (6)
- label
- ERT User- Did Not Switch to Galafold
- description
- ERT users with mutation amenable to Galafold who did not switch
- interventionNames
- Other: This is a non-interventional study
- label
- ERT User- Switched and Stayed on Galafold
- description
- ERT users with the mutation amenable to Galafold who switched and stayed on Galafold
- interventionNames
- Other: This is a non-interventional study
- label
- No Previous Therapy- Started Galafold and Stayed On
- description
- Those naïve to therapy with the mutation amenable to Galafold who went on and stayed on Galafold
- interventionNames
- Other: This is a non-interventional study
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
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
References
Publications (0)
Data not yet available
No reference posted for this study.