Getting Global Rare Disease Insights Through Technology Study (GRIT)

The G.R.I.T. Study: Getting Global Rare Disease Insights Through Technology

This project is a randomized controlled trial to use a mobile health journal, called Zamplo (formerly known as MyHealthJournal or ZoeInsights), to record patient reported outcomes (PROM) in patients with metabolic disorders.

The objective of the study is to assess the feasibility, acceptability and potential effectiveness of the Zamplo.

The primary hypothesis is as follows:

The Zamplo platform will significantly increase patient activation at 6 months post-baseline, defined as an individual's knowledge, skill, and confidence for managing their health and health care.

The primary outcome is as follows:

Patient activation following the use of Zamplo will serve as the primary outcome of interest and will be measured by the Patient Activation Measure (PAM) 13. The PAM 13 shows the degree of the patient's ability to manage their health with confidence by providing a total patient activation score.

Brief Background:

This project is a randomized controlled trial to use a mobile health journal, called Zamplo, to record patient reported outcomes (PROM) in patients with metabolic disorders.

Zamplo is a software as a service (SaaS) digital platform on both iOS and Android platforms that allows real-time entry of patient symptoms and response to medications. It provides the patients with an interface to see their progress, store questions that they will ask at the next clinic visit, record their health data and use their data to engage in their health outcomes. MAGIC Clinic Ltd., which is the largest clinic in Alberta that manages metabolic disorders such as Fabry disease, Pompe disease, and Gaucher disease, will provide access to Zamplo to patients free-of-charge to evaluate its utility in managing the symptoms of their disease.

Brief Study Design:

The study is a two-armed randomized controlled design with 1:1 allocation to treatment (Zamplo app group) or control (usual care) arms, with assessments at four time points: baseline, 1 month, 3 months (primary outcome), 6 months and 12 months follow-up post-baseline. This is an open-label trial.

The investigators intend to recruit 150 participants in this study, with 75 of them being controls.

Inclusion Criteria:

Adult patients with a diagnosis of metabolic disease Access to a smartphone with data connection Willingness to devote 10-15 mins of time in a day to log medications and notes Able to speak and write English sufficiently to complete questionnaires.

Exclusion Criteria:

Insufficient cognitive function to participate in the study The use of any electronic application requires some competency with the software on a cellphone, downloading the application and entering the data. Some patients who are elderly may not be familiar with this technology and would be excluded.

Study Overview

• Status: Recruiting
• Conditions: Mitochondrial Diseases; Fabry Disease; Metabolic Disease; Gaucher Disease; Pompe Disease
• Intervention / Treatment: Other: Zamplo Digital Health Platform

• Study Type: Interventional
• Enrollment (Anticipated): 150
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Desmond Koo; Phone Number: +1 (403) 888-3278; Email: clinicaltrials@calgarymetabolics.hush.com

Study Locations

• Canada
  • Alberta
    • Calgary, Alberta, Canada, T2M0L6
      • Recruiting
      • M.A.G.I.C. Clinic LTD
      • Contact: Desmond Koo; Phone Number: +1 (403) 888-3278; Email: clinicaltrials@calgarymetabolics.hush.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Adult patients with a diagnosis of metabolic disease
• Access to a smartphone/computer with data connection
• Willingness to devote 10-15 mins of time in a day to log medications and notes
• Able to speak and write English sufficiently to complete questionnaires

Exclusion Criteria:

• Insufficient cognitive function to participate in the study
• The use of any electronic application requires some competency with the software on a cellphone, downloading the application and entering the data. Some patients who are elderly may not be familiar with this technology and would be excluded.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: OTHER
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: NONE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: App Group; Patients in this group are provided with a link to download the application and their application usage is tracked by the clinic.	Other: Zamplo Digital Health Platform; This application will be provided to patients and provide them with a method to track various data related to their metabolic disease.
PLACEBO_COMPARATOR: Placebo Group; Patients in this group are not provided with the clinic link to the application.	Other: Zamplo Digital Health Platform; This application will be provided to patients and provide them with a method to track various data related to their metabolic disease.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Patient Activation	Patient activation following the use of Zamplo will serve as the primary outcome of interest and will be measured by the Patient Activation Measure (PAM) 13. The PAM 13 shows the degree of the patient's ability to manage their health with confidence by providing a total patient activation score. The survey will be completed by participants of the two groups at baseline and follow-up time-points.	3 Months post baseline

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Self-Efficacy for Managing Chronic Conditions (PROMIS)	Manage Medications/Treatment: Self-efficacy is defined as confidence in one's ability to successfully perform specific tasks or behaviors. Self Efficacy for Managing Chronic Conditions assesses confidence in one's ability to successfully perform specific tasks or behaviors related to one's health in a variety of situations. Confidence in managing medication schedules of different complexity.	3 Months
Patient Satisfaction	This is a survey question asked during Alberta Health Quality Assessments Q11. "Thinking about all of your personal experiences within the past year with the healthcare services in Alberta that we just reviewed, to what degree are you satisfied or dissatisfied with the services you have received?"	3 Months
Acceptability	The acceptability of Zamplo will be assessed by patients and informal caregivers.	3 Months

Collaborators and Investigators

• Sponsor: M.A.G.I.C. Clinic LTD
• Collaborators: Hanalytics Solutions Inc.

Publications and helpful links

Helpful Links

• Link to request information about the study

Study record dates

Study Major Dates

• Study Start (ACTUAL): August 7, 2020
• Primary Completion (ANTICIPATED): May 15, 2021
• Study Completion (ANTICIPATED): November 30, 2021

Study Registration Dates

• First Submitted: January 21, 2021
• First Submitted That Met QC Criteria: February 12, 2021
• First Posted (ACTUAL): February 17, 2021

Study Record Updates

• Last Update Posted (ACTUAL): April 8, 2021
• Last Update Submitted That Met QC Criteria: April 6, 2021
• Last Verified: April 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Cardiovascular Diseases; Vascular Diseases; Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Disease Attributes; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Cerebral Small Vessel Diseases; Lipidoses; Lipid Metabolism, Inborn Errors; Glycogen Storage Disease; Mitochondrial Diseases; Fabry Disease; Metabolic Diseases; Rare Diseases; Glycogen Storage Disease Type II; Gaucher Disease
• Other Study ID Numbers: GRIT

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