It is an international and non-interventional registry (observational registry) to assess the effects of different types of treatment used to treat Fabry disease.
Aim of the study
This register is for observation purposes only, which means that you will not be asked to make additional clinic visits or have further laboratory tests carried out. All data collected as part of this registry is obtained from the information in the medical record created by your doctor during your regular medical visits and from the examinations performed during these visits.
Who can take part?
Patients with Fabry disease
Procedure
This register is for observation purposes only, which means that you will not be asked to make additional clinic visits or have further laboratory tests carried out. All data collected as part of this registry is drawn from the information generated by your doctor during your visits as part of your routine care and from the examinations performed during these visits.
As part of this registry, Amicus hopes to obtain information from up to 2000 patients diagnosed with Fabry disease. The information about your Fabry disease is collected for the registry in 2 ways:
1. data collected during your regular appointment with your doctor will be checked by your doctor or his team and made available to Amicus in a secure manner. The data collected will be limited to data required by the registry protocol and collected during your appointment with the physician. No other data is collected. Your data will be protected and will not contain your name and address so that no conclusions can be drawn about you personally and so that you cannot be traced.
2. you will be asked to complete questionnaires to help Amicus determine whether Fabry disease treatments are changing your quality of life. These questionnaires are completed either on a secure website, on a smartphone or on a tablet device. These systems are validated and safe. This means that no one can access your data unless it is specified that this person may have access within the scope of the register. You will receive a unique user name and password to gain access to these systems. Only you, no one else, have access to your questionnaires.
Compensation
You will not be paid for your participation in this register.
Original study name
A PROSPECTIVE, OBSERVATIONAL REGISTRY OF PATIENTS WITH FABRY DISEASE
BASEC number
2018-00394
Sponsors
Amicus Therapeutics UK (Sponsor)