MedTrace Logo

Digital Health Platform Customized for Patients With Gaucher Disease

NCT04997772 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 80

Last updated 2024-04-17

No results posted yet for this study

Summary

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.

Conditions

  • Gaucher Disease
  • Patient Reported Outcome

Interventions

DEVICE

Digital Gaucher Platform

Digital platform specified for Gaucher patients.

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    collaborator INDUSTRY

  • Shoshana Vilk

    lead OTHER

Eligibility

Min Age
19 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-02-01
Primary Completion
2024-02-28
Completion
2024-03-30

Countries

  • Israel

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04997772 on ClinicalTrials.gov