Digital Health Platform Customized for Patients With Gaucher Disease

Brief summary (registry, in English)

The entry of digitization into the world in recent years is helping the health care system to operate more efficiently than in the past and has increased the participation of patients and their families in managing their health care. In a rare disease, such as Gaucher disease, patient involvement through digital technology is of great importance. Gaucher patients come for an inspection at the Gaucher unit once every six months. However, medical events, related and unrelated to Gaucher, may occur between these visits, some of which may be urgent. A digital Gaucher platform will allow for the updating of medical events occurring in the patient between these visits and will allow specialists to give up-to-date medical advice to the patient and the local doctor when needed. The Gaucha Digital Platform will provide digital tools (and applications) for self-management, monitoring and regular contact with the Gaucher Unit. The system will have an alert system that will allow accessible communication between the patient and the Gaucher unit. Moreover, patients with Gaucher disease need a lifelong commitment to their care; Enzyme replacement therapy (ERT) and substrate inhibitor therapy (SRT). When patients are monitored only once or twice a year, monitoring adherence to treatment may be a problem. Adherence to the treatment regimen is essential for achieving normalization. The system will have a system of reminders for treatment and a system for monitoring the receipt of treatment. The digital system will include quality of life questionnaires and pain questionnaires that will help to more comprehensively understand the patient's condition. Finally, a Gaucher-adapted digital platform will ensure the collection of all relevant clinical data that is important for the treatment of a rare and multi-systemic disease such as Gaucher disease. A complete database will make it possible to create an anonymous database that will be used to find predictors of response to treatment, complications and commodities associated with Gaucher disease.

What you commit to

• Study type: Observational
• Participants enrolled: 80
• From Feb 2022 to the primary completion date, Feb 2024
• 1 site in 1 country
• Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Who the study is looking for

Age in the registry: 19 years to - · Sex: All

Full criteria on ClinicalTrials.gov

Sites (1)

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