Getting Global Rare Disease Insights Through Technology Study

NCT04758130 | Unknown

• 1 other identifier: GRIT
• Study Type: interventional
• Target: 150
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Metabolic Disease; Fabry Disease; Gaucher Disease; Pompe Disease; Mitochondrial Diseases

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Self-Efficacy for Managing Chronic Conditions (PROMIS)

  3 Months

• Patient Satisfaction

  3 Months

• Acceptability

  3 Months

Study Arms (2)

App Group

EXPERIMENTAL

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

Placebo Group

PLACEBO COMPARATOR

Patients in this group are not provided with the clinic link to the application.

Other: Zamplo Digital Health Platform

Interventions

Zamplo Digital Health Platform OTHER

This application will be provided to patients and provide them with a method to track various data related to their metabolic disease.

App Group; Placebo Group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• M.A.G.I.C. Clinic LTD: lead
• Hanalytics Solutions Inc.: collaborator

Study Sites (1)

M.A.G.I.C. Clinic LTD

Calgary, Alberta, T2M0L6, Canada

RECRUITING

Related Links

• Link to request information about the study

MeSH Terms

Conditions

Metabolic Diseases; Fabry Disease; Gaucher Disease; Glycogen Storage Disease Type II; Mitochondrial Diseases

Condition Hierarchy (Ancestors)

Nutritional and Metabolic Diseases; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Glycogen Storage Disease; Carbohydrate Metabolism, Inborn Errors

Central Study Contacts

Desmond Koo

CONTACT

+1 (403) 888-3278; clinicaltrials@calgarymetabolics.hush.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 21, 2021
• First Posted: February 17, 2021
• Study Start: August 7, 2020
• Primary Completion: May 15, 2021
• Study Completion: November 30, 2021
• Last Updated: April 8, 2021

Record last verified: 2021-04

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1)