The detection and treatment of diseases has made enormous progress in recent decades. This development is the result of many years of research in which doctors, scientists and patients of different generations are actively involved. Research is largely dependent on data from patients' medical histories. These can include, for example, results from laboratory analyses, information on a therapy or on hereditary predispositions to certain diseases. This information explains how you as a patient can contribute to medical progress and shows how your patient data and rights are protected.
Aim of the study
A study to evaluate the effect of venglustat tablets on neuropathic and abdominal pain in adult participants with Fabry disease
Who can take part?
Patients with Fabry disease who have not been treated for at least 6 months and are untreated.
Procedure
A subsidiary of the sponsor, Sanofi, manages a Fabry disease registry (DIREGC07006) for patients with Fabry disease to help healthcare providers better understand the disease. The purpose of the Fabry Disease Registry is to collect information about the current treatments of patients with Fabry disease in order to gain more knowledge about Fabry disease and to better help patients in the future.
This Fabry disease registry is voluntary and independent of the EFC17045 study.
It would be very useful to perform a combined analysis of your coded data collected in the EFC17045 study with your coded data from the Fabry disease registry (if you are already participating or would like to participate in the registry). This will provide more insight into your Fabry disease and the long-term outcomes after your participation in the EFC17045 study has ended. For this reason, at the end of the EFC17045 study, Sanofi would like the opportunity to transfer your coded study data from the EFC17045 study to the database of the Fabry Disease Registry. The encrypted data that may be transmitted includes your demographic data (e.g. your age) and information about your health (e.g. laboratory/imaging data, genetic data or other) and lifestyle. Once the transfer is complete, Sanofi and its service providers and partners involved in the implementation of the Fabry disease registry will be able to access your encrypted data from the EFC17045 study.
Your consent is voluntary. It is valid indefinitely unless it is revoked (withdrawn). You can withdraw your consent at any time without giving reasons. After revocation, your data will no longer be made available. Your decision has no influence on your medical treatment.
Compensation
None
Original study name
A Randomized, Double-blind, Placebo-controlled, 12-month Phase 3 Study to Evaluate the Effect of Venglustat on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age with Fabry Disease who are Treatment-naïve or Untreated for at L
BASEC number
2023-01446
Sponsors
Sanofi-Aventis (Suisse), Montfleury, Vernier (Sponsor)