Trial Review

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Trial registered on ANZCTR


Registration number
Help: Registration number
ACTRN12626000943347p
Ethics application status
Help: Ethics application status
Submitted, not yet approved
Date submitted
Help: Date submitted
18/06/2026
Date registered
Help: Date registered
30/07/2026
Date last updated
Help: Date last updated
30/07/2026
Date data sharing statement initially provided
Help: Data sharing statement date
30/07/2026
Type of registration
Help: Type of registration
Prospectively registered

Titles & IDs
Public title
To assess the usability and clinical utility of a Medical Artificial Intelligence System with different consumer groups in the Pompe Disease community
Scientific title
Interventional study of the utility of a newly developed AI system (Mia AI), in providing education and application for Patients, Carers, Physicians, and Researchers on Pompe Disease, a Lysosomal Storage Disease
Additional trial identifiers [1] 317627 0
NIL
Universal Trial Number (UTN)
Trial acronym
Related trial records

Health condition
Health condition(s) studied:
Pompe Disease 342398 0
Condition category
Condition code
Human Genetics and Inherited Disorders 338529 338529 0 0
Other human genetics and inherited disorders
Musculoskeletal 338530 338530 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
Description of the intervention or exposure
Description of intervention or exposure: The intervention under investigation is the Mia AI clinical decision-support platform for Pompe disease. The system is designed to assist with patient identification, disease monitoring, and multidisciplinary care coordination using artificial intelligence tools, including large language models, retrieval augmented generation, natural language processing (NLP), machine learning models, and biomarker analytics.

Participants will be provided with:
Access to a demonstration version of the Mia AI platform (secure web-based interface or local deployment)
Participant Information Sheet and informed consent documentation
System Usability Scale (SUS) questionnaire
Semi-structured interview guide (adapted from established automation and AI assessment methodologies)
Participants will only be able to ask questions to the AI chatbot relevant to their stratification, ie patient/carer, nurse, primary care physician, consultant/researcher. Access to individual patient details such as care plans and biomarkers, will not be available.
Participants will be able to explore the platform freely but will be limited to their stratification as explained earlier. Participants will not have to complete the same study activities, they will be able to ask the chatbot as many questions as time permits.

Participants may ask for assistance while using the platform if needed.
Duration: One 2 hour session

Location/setting: 10 The Esplanade, Perth, Western Australia, 6000; or Perth Children's Hospital; or online
Intervention code [1] 334446 0
Diagnosis / Prognosis
Description of the comparator or control
No control group

This study is an interventional study to understand the participants' perspectives on the use of AI and its usability.
Control group
Uncontrolled

Outcomes
Primary outcome [1] 345808 0
System usability as measured by the System Usability Scale (SUS) score.
Timepoint [1] 345808 0
within three months of first use of the system
Secondary outcome [1] 463381 0
Participant perceptions of the usefulness, acceptability, and trustworthiness of the Mia AI platform. This will be assessed as a composite outcome.
Timepoint [1] 463381 0
Within three months of first use of the system
Secondary outcome [2] 463943 0
Identification of barriers and facilitators to implementation.
Timepoint [2] 463943 0
Within three months of first use of the system
Secondary outcome [3] 463944 0
Qualitative themes relating to stakeholder expectations of AI in Pompe disease (exploratory outcome).
Timepoint [3] 463944 0
Within three months of first use of the system
Secondary outcome [4] 465394 0
Technical performance of AI system (ie data accuracy)
Timepoint [4] 465394 0
1 week before commencing interviews and 1 week after concluding interviews
Secondary outcome [5] 465395 0
Technical performance of AI system (ie hallucination of AI system)
Timepoint [5] 465395 0
1 week before commencing interviews and 1 week after concluding interviews

Eligibility
Key inclusion criteria
Any one at any age with late onset pompe disease, or the associated commmunity of family, carers, researchers, clinicians, or treatment providers
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Healthy volunteers
No
Key exclusion criteria
None

Study design
Purpose of the study
Diagnosis
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Who is / are masked / blinded?



Intervention assignment
Other design features
Phase
Not Applicable
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
1/08/2026
Actual
Date of last participant enrolment
Anticipated
1/09/2026
Actual
Date of last data collection
Anticipated
30/09/2026
Actual
Sample size
Target
30
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
ACT,NSW,NT,QLD,SA,TAS,WA,VIC
Recruitment hospital [1] 29653 0
Perth Children's Hospital - Nedlands
Recruitment postcode(s) [1] 45998 0
6009 - Nedlands

Funding & Sponsors
Funding source category [1] 322169 0
Commercial sector/Industry
Name [1] 322169 0
Integrated Energy Pty Ltd
Country [1] 322169 0
Australia
Funding source category [2] 322170 0
Commercial sector/Industry
Name [2] 322170 0
Medical AI Pty Ltd
Country [2] 322170 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
David Cavanagh, Integrated Energy Pty Ltd
Address
Country
Australia
Secondary sponsor category [1] 325069 0
Commercial sector/Industry
Name [1] 325069 0
Integrated Energy Pty Ltd
Address [1] 325069 0
Country [1] 325069 0
Australia
Secondary sponsor category [2] 325070 0
Commercial sector/Industry
Name [2] 325070 0
Medical AI Pty Ltd
Address [2] 325070 0
Country [2] 325070 0
Australia
Other collaborator category [1] 283995 0
University
Name [1] 283995 0
University of Western Australia
Address [1] 283995 0
Country [1] 283995 0
Australia
Other collaborator category [2] 283996 0
Hospital
Name [2] 283996 0
Perth Childrens Hospital
Address [2] 283996 0
Country [2] 283996 0
Australia
Other collaborator category [3] 283997 0
Individual
Name [3] 283997 0
Professor Jon Watson, University of Western Australia
Address [3] 283997 0
Country [3] 283997 0
Australia
Other collaborator category [4] 283998 0
University
Name [4] 283998 0
Krisha Timbadia, University of Western Australia
Address [4] 283998 0
Country [4] 283998 0
Australia

Ethics approval
Ethics application status
Submitted, not yet approved
Ethics committee name [1] 320702 0
Bellberry Human Research Ethics Committee A
Ethics committee address [1] 320702 0
Ethics committee country [1] 320702 0
Australia
Date submitted for ethics approval [1] 320702 0
20/07/2026
Approval date [1] 320702 0
Ethics approval number [1] 320702 0

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 151334 0
Mr David Cavanagh
Address 151334 0
Integrated Energy Pty Ltd; 10 The Esplanade, Perth, Western Australia 6000
Country 151334 0
Australia
Phone 151334 0
+61 432 201 446
Fax 151334 0
Email 151334 0
[email protected]
Contact person for public queries
Name 151335 0
David Cavanagh
Address 151335 0
Integrated Energy Pty Ltd; 10 The Esplanade, Perth, Western Australia 6000
Country 151335 0
Australia
Phone 151335 0
+61 432 201 446
Fax 151335 0
Email 151335 0
[email protected]
Contact person for scientific queries
Name 151336 0
David Cavanagh
Address 151336 0
Integrated Energy Pty Ltd; 10 The Esplanade, Perth, Western Australia 6000
Country 151336 0
Australia
Phone 151336 0
+61 432 201 446
Fax 151336 0
Email 151336 0
[email protected]

Data sharing statement
Will the study consider sharing individual participant data?
No


What supporting documents are/will be available?

No Supporting Document Provided


Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.