Clinical Trial Details
— Status: Completed
Administrative data
| NCT number |
NCT04997772 |
| Other study ID # |
0380-20-SZMC |
| Secondary ID |
|
| Status |
Completed |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
February 1, 2022 |
| Est. completion date |
March 30, 2024 |
Study information
| Verified date |
April 2024 |
| Source |
Shaare Zedek Medical Center |
| Contact |
n/a |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational
|
Clinical Trial 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.
Description:
With quality outcomes and value, the watchwords for health care in the 21st century,
stakeholders around the globe are looking for innovative, cost-effective ways to deliver
patient-centered, technology-enabled "smart" healthcare, both inside and outside hospital
walls.The Digital transformation in health and care aims at supporting the management of
health and well being while empowering the participation of people and facilitating the
transformation of health and care services to more digitized, person-centered and
community-based care models, thereby enabling better access to healthcare and the
sustainability of health and care systems.
Globally, the investigators see also an increase in the desire of patients themselves to play
an active role in their healthcare management. In rare disease, such as Gaucher Disease (GD),
patient engagement through a combined digital technology may have added importance. It is
recommended for patients with GD to be managed by a multidisciplinary team of experts having
long-term experience with the diverse clinical manifestations and potential GD-related or
unrelated co-morbidities, their follow-up visits at the center of excellence are usually on
an annual or semi-annual basis. However, disease-related and un-related medical events may
occur in-between these visits, some which may be emergent. A digital Gaucher platform will
enable updating medical events occurring to the patient in-between these visits and enable
the specialists to give updated medical consult to the patient and local physician when
needed.
Furthermore, patients with GD need a life-long commitment to their therapy; enzyme
replacement therapy (ERT) and substrate reduction therapy (SRT). The adherence to therapy
regimen is essential to achieve normalization. When patients are followed only once-twice a
year in a specialized clinic following the adherence to therapy might be a problem. A digital
Gaucher-platform will provide digital tools (and applications) for self-management,
adherence, and patient-reported outcome using a Gaucher-dedicated APP. Alert dashboard will
be developed and installed for the health professionals in order to enable accessible
communication between the patient and health professionals and be able to respond in a timely
manner.
Patient-reported outcome (PRO) and Patient-reported outcome measures (PROMs) are increasingly
important in research and clinical practice and to monitor the efficiency of health care
services. The use of PROMs is fundamental to ensure that what matters to patients is captured
in a valid, reliable, responsive, and feasible manner. A Digital Gaucher Platform can collect
GD-specific PROMs.
Finally, a digital Gaucher platform will ensure capturing all relevant clinical data, full
data files, and complete missing data, important endeavor for the treatment of the individual
patients with a rare and multi-system disease such as GD. Data completion is essential for
the development of a database, agnostic to pharma companies or ways of treatment, with
high-quality real-time data. This database can then be used for big data analysis for finding
predictors for GD-related complications and commodities.
