Getting Global Rare Disease Insights Through Technology Study

Last updated: April 6, 2021
Sponsor: M.A.G.I.C. Clinic LTD
Overall Status: Active - Recruiting

Phase

N/A

Condition

Fabry Disease

Metabolic Disorders

Neuronal Ceroid Lipofuscinoses (Ncl)

Treatment

N/A

Clinical Study ID

NCT04758130
GRIT
  • Ages > 18
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

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

Exclusion Criteria:

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

Study Design

Total Participants: 150
Study Start date:
August 07, 2020
Estimated Completion Date:
November 30, 2021

Connect with a study center

  • M.A.G.I.C. Clinic LTD

    Calgary, Alberta T2M0L6
    Canada

    Active - Recruiting

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