followME Fabry Disease Registry
Summary
The followME registry is an observational, voluntary study to evaluate the effects of various treatments on long-term safety, effectiveness, and quality of life in patients with Fabry disease, with a main focus on a medicine called migalastat. This registry study is strictly observational. Patients will no be asked to attend additional visits, take any additional medicines, or undergo any tests over and above their regular care. All data will be collected from the information that the patients' physicians document during regular visits as well as the tests that are ordered during those visits. Patients will be observed for occurrences of key indicators of safety and effectiveness such as heart, brain, blood vessel and kidney events as well as pain management and overall survival. A comparison of these events will be evaluated over a period of 5 years in patients taking migalasat, patients receiving enzyme replacement therapy and untreated patients with Fabry disease.
Eligibility
Eligible gender: Male, Female, Transgender, Other
Eligible ages: 16 to 100
Inclusion criteria:
1. Diagnosis of Fabry disease made via analysis of alpha-galactosidase enzyme levels in plasma or white blood cells or mutational analysis;
2. Age 16 or older;
3. Able to give informed consent;
4. Willing to comply with the recommended schedule of assessments
Exclusion criteria:
1. Patients who are participating in a clinical trial of any investigational medicinal product or device at the time of consent.
Participate
Fill out the following form if you want to participate in this research
Collection of personal information
Your personal information is collected under
the authority of section 33(c) of the Freedom of Information and Protection of Privacy Act. If
you have any questions about the collection or use of this information, please visit our
Access to Information page.
Additional information
Contact information
Metabolic and Genetic Clinical Research Study Coordinator at 403-955-2631 for Dr Aneal Khan.
Principal investigator:
Aneal Khan
Clinical trial:
No
REB-ID:
HREBA.CTC-23-0024