Digital Health Platform Customized for Patients With Gaucher Disease

April 16, 2024 updated by: Shoshana Vilk

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed 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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
80

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Israel
      • Jerusalem, Israel, 9103102
        • Shaare Zedek Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

19 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Sampling Method

Non-Probability Sample

Study Population

Gaucher disease patients above the age of 18 years old.

Description

Inclusion Criteria:

  1. All adults (>18 years, no age maximum) treated patients with GD will be eligible.
  2. Signed informed consent.

Exclusion Criteria:

  1. Patients without access to either computer or mobiles phone.
  2. For this pilot study patients whose communication skills in Hebrew is not good enough to complete the study questionnaires and PROMs [the app platform will use a very simplified language, thus allowing patients with limited Hebrew to use the app] *for future use/study the platform will be translated to other languages, as needed.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
1
Gaucher disease patients > 18 years old.
Device: Digital Gaucher Platform
Digital platform specified for Gaucher patients.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Rate of satisfaction from Gaucher treatment at baseline compared to the end of study
Time Frame: 12 months
GD-PROMs
12 months

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Adherence to treatment based on participant reports in the App
Time Frame: 12 months
participants report in the app
12 months
Achievement of treatment goals based on Gaucher consensus paper
Time Frame: 12 months
Gaucher consensus paper
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Shoshana Vilk

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Genzyme, a Sanofi Company

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
February 1, 2022

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
February 28, 2024

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
March 30, 2024

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 28, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 9, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
August 10, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 17, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 16, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 0380-20-SZMC

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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