CLINICAL TRIAL SUPPORT GRANT PROJECT

A Customised Digital Health Intervention to improve fatigue and function in patients with mitochondrial disease

Clinical Trial Support Grants

$225,000 AUD

01/07/2020 → 30/06/2023

Carolyn Sue

Researcher

Prof Carolyn Sue AM

Research Institution

Kolling Institute Royal North Shore Hospital

Project Name

A Customised Digital Health Intervention to improve fatigue and function in patients with mitochondrial disease

Project Overview

Muscle fatigue and weakness is one of the most common problems faced by patients with mitochondrial disease. These problems are typically treated by exercise therapy, but unfortunately, people living with mitochondrial disease often have exercise intolerance and struggle to maintain an effective exercise program. Clinical investigators, Prof Sue and Prof Chow from leading research institutes in Sydney will be recruiting people with mitochondrial disease and symptoms of muscle fatigue and weakness, to participate in a 12 month, randomised controlled trial using a customised interactive mobile phone text-messaging and support service to determine whether this disruptive technology can improve the uptake of exercise therapy. The service will link data from wearable activity monitors (Fitbits) to assess compliance and effect of the prescribed exercise program. We will determine whether this digital health intervention is able to improve compliance and facilitate the delivery of the beneficial effects of a personalised exercise program, prescribed to improve symptoms of muscle fatigue and weakness in patients with mitochondrial disease.
If the trial proves successful, the use of this DHI could have considerable impact if uptake by people with mitochondrial disease and these symptoms is widespread.

Project Details

Aim
The aim of this project is to determine whether interactive digital health interventions (DHI) can increase compliance of a personalised exercise program in patients with mitochondrial disease (MD).

Relevance
MD is an under-researched medical condition that affects over 100,000 Australians. It is the most common group of inherited metabolic disease in the world. MD is a clinically heterogeneous group of disorders caused by mutations in mitochondrial DNA (mDNA) or nuclear DNA (nDNA) [Davis 2011; Gorman 2016]. Myopathy is a common clinical manifestation of MD typified by symptoms of exercise intolerance, muscle fatigue and muscle weakness [Trenell 2006]. In addition, 62% of people with MD report excessive symptomatic fatigue [Gorman 2015]. Aerobic training improves muscle phosphate creatine clearance, which can be a surrogate marker for improvement in mitochondrial function in the muscle studied [Trenell 2006]. Aerobic exercise is one of the few interventions shown to improve oxidative function and muscle metabolism in patients with MD and can lead to improvements in muscle function and muscle strength. However, many patients with MD have exercise intolerance and struggle to maintain an exercise program despite its documented benefits. Tools to improve compliance with exercise therapy by supporting patients to adhere to a customised exercise program are needed. Interactive DHI programs facilitate high levels of adoption of exercise programs and can be applied for widespread implementation. This would potentially improve muscle function and thereby reduce the impact of muscle fatigue in patients with MD.

How will this project be undertaken?
Research teams led by Professor Carolyn Sue from the Kolling Institute and Professor Clara Chow from the Westmead Applied Research Centre will collaborate to build a DHI program to support the application of exercise therapy in patients with MD. We will recruit 60 patients from the mitochondrial clinic at Royal North Shore Hospital to participate in a randomised controlled trial (RCT) to compare the effect of an individualised exercise program with or without the DHI support. The study will be completed over a 3-year period; 6 months to establish the set up, 6 to 12 months of recruitment, a 12 month trial period randomised to exercise treatment prescribed with and without the DHI program and 6 months to analyse and report of the results.
We will also determine the feasibility of scaling the DHI following successful outcomes of the trial. We will employ a 0.5FTE project officer who will assist the investigators to establish and manage data collection, data analysis and database management. Furthermore, the project officer will be responsible for:
1) Governance
2) Trial Management, including ethics applications, protocol and informed consent forms as required.
3) Screening and analysing incoming trial and DHI data
4) Project communication, including publication.