ANZCTR - Registration

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Trial registered on ANZCTR

Registration number

ACTRN12626001072303p

Ethics application status

Not yet submitted

Date submitted

27/02/2026

Date registered

31/08/2026

Date last updated

31/08/2026

Date data sharing statement initially provided

31/08/2026

Type of registration

Prospectively registered

Titles & IDs

Public title

A Digital Health Platform to Support Self-Management of Hypophosphatasia in Adults (HPP Helper)

Scientific title

Exploring the Feasibility of a Co-Designed Digital Health Platform to Support Self-Management of Hypophosphatasia in Adults (HPP Helper)

Additional trial identifiers [1]

N/A

Universal Trial Number (UTN)

Trial acronym

Related trial records

Health condition

Health condition(s) studied:

Hypophosphatasia

Condition category

Condition code

Musculoskeletal

Other muscular and skeletal disorders

Intervention/exposure

Study type

Interventional

Description of the intervention or exposure

Enrolled participants will receive a pre-configured Amazon Echo Show 8 (DVA equipped with an 8-inch interactive HD touchscreen) delivered to their homes via courier. The unit will be shipped with written and visual onboarding materials detailing home Wi-Fi connection, microphone/camera privacy controls, basic touch and voice navigation commands, and contact details for the trial research team to provide dedicated remote setup support. Instructions on the use of the intervention software interface "BuddyLink" (Great Australian Pty Ltd) will also be provided. This interface can be accessed via the DVA, or via a web browser on other internet-enabled devices, as preferred by participants.

Following baseline assessment, intervention content is provided across the 12-week intervention period on two days per week (generally Monday and Thursday). New educational videos, modules, and task prompts trigger visual on-screen notifications on the DVA, alerting participants to their availability. Participants can choose to interact with these immediately, or at a later date, using their preferred device. All newly unlocked modules remain permanently accessible on demand throughout the remainder of the 12 weeks for repeated viewing and ongoing reference. Live group sessions will be scheduled on days and times deemed most convenient for the majority of participants and conducted via Zoom. Recordings of these sessions will be shared via BuddyLink so that those who are unable to attend in person can review the content.

The 12-week curriculum is developed based on current evidence and our previous co-design process with adult HPP consumers. Content includes:
Educational Videos: Delivered by research team members and based on clinical practice guidelines, content covered will include “Understanding HPP” (biology, clinical phenotypes, and reviews of contemporary metabolic bone disease research). “Health Behaviours” (Instruction on resistance training, joint-protection ergonomics, fall mitigation, and pacing strategies designed to avoid stress fractures, guidance on whole-food nutrient density and anti-inflammatory diets, management of calcium and vitamin D intake), “Mental Health” (cognitive and behavioural self-management approaches to addressing diagnostic delay distress, chronic pain interference, illness uncertainty, and social isolation in rare diseases, and access to support services), and “Health Navigation” (guidance on building multidisciplinary care teams, accessing allied health and psychology treatment plans, and transitioning to sustainable community self-management).
Personalised Health Tracking: The optional Digital Health Diary and Dietary Tracker allows participants to regularly record symptoms (e.g., pain, fatigue, mobility), healthcare appointments, and daily food logs. Aggregated personal diary summaries can be exported directly to the participant via email to inform clinical consultations.
Noticeboard and Healthcare Professional Database: Accessible directly through the BuddyLink interface, the HPP Noticeboard functions as a shared repository of curated community resources, including educational content, physical activity options, healthy recipes, services and healthcare (e.g., general practitioners, endocrinologists, rheumatologists, and allied health professionals with adult HPP expertise) recommendations. Participants can nominate noticeboard content via the platform or email. All nominations undergo screening by the research team prior to being published and broadcast to the participant cohort.
Group-Based Peer Interactions and Health Professional Education Sessions: Includes two live 60-minute investigator-facilitated peer connection videoconferences (conducted via Zoom in Week 2 and Week 12) to foster shared lived experiences, alongside an optional, asynchronous, study-moderated peer messaging group (linked via BuddyLink) available throughout the 12-week trial. Participants will also be invited to participate in a structured, live 60-minute clinical education seminar conducted via Zoom in Week 6 (with session recordings archived in Week 7 for on-demand access). The seminar will be delivered by a specialist endocrinologist/metabolic bone expert and cover multidisciplinary clinical management, research directions, and an interactive participant Q&A based on pre-submitted and live questions.
Artificial Intelligence Question-and-Answer Tool: Participants will have on-demand access to "HPP AI," a conversational agent embedded within the DVA. The tool operates using the OpenAI ChatGPT API via an enterprise license, ensuring zero data retention (conversations are neither recorded nor used for model training). The agent is bounded by pre-specified guardrails and an evidence-based HPP knowledge base curated by the study investigators. It is programmed to answer general consumer queries regarding HPP, lifestyle modifications, and trial navigation, while explicitly restricting independent diagnostic claims and redirecting urgent clinical queries to the participant’s treating physician.

Intervention engagement and adherence will be monitored objectively via BuddyLink server logs, tracking accessing of specific modules across the 12 weeks. Acceptable adherence is quantitatively defined as accessing =66% of delivered content. Platform usage data will be paired with automated conversational check-ins assessing content relevance and usefulness. Participants will be clearly instructed that platform materials support their health self-management and do not replace personalised advice from their medical team.

Intervention code [1]

Lifestyle

Intervention code [2]

Behaviour

Intervention code [3]

Treatment: Other

Description of the comparator or control

There will be no control group of participants receiving usual care given that the primary aims for this pilot study relate to exploring feasibility of the intervention.

Control group

Uncontrolled

Outcomes

Primary outcome [1]

Adherence

Timepoint [1]

12 weeks post-commencement of the intervention

Secondary outcome [1]

Change in self-efficacy

Timepoint [1]

12 weeks post-commencement of the intervention

Secondary outcome [2]

Change in mobility and pain (assessed as a composite outcome)

Timepoint [2]

12 weeks post-commencement of the intervention

Secondary outcome [3]

Change in health-related quality of life

Timepoint [3]

12 weeks post-commencement of the intervention

Secondary outcome [4]

System usability

Timepoint [4]

12 weeks post-commencement of the intervention

Secondary outcome [5]

Enablers and barriers to participation (assessed as a composite outcome)

Timepoint [5]

12 weeks post-commencement of the intervention

Secondary outcome [6]

Recruitment

Timepoint [6]

Prior to baseline

Secondary outcome [7]

Retention

Timepoint [7]

12 weeks post-commencement of the intervention

Eligibility

Key inclusion criteria

1. Adult (aged 18 or older) with diagnosed Hypophosphatasia (HPP)
2. Currently living in Australia

Minimum age

18 Years

Maximum age

No limit

Sex

Both males and females

Healthy volunteers

Key exclusion criteria

1. No access to internet service which would preclude use of the DVA system.
2. Currently receiving enzyme replacement therapy with asfotase alfa (Strensiq®); This ensures that the intervention is tested in the population most likely to rely on non-pharmacological strategies for disease management.

Study design

Purpose of the study

Educational / counselling / training

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

Open (masking not used)

Who is / are masked / blinded?

Intervention assignment

Single group

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

2/11/2026

Actual

Date of last participant enrolment

Anticipated

1/02/2027

Actual

Date of last data collection

Anticipated

28/05/2027

Actual

Sample size

Target

Accrual to date

Final

Recruitment in Australia

Recruitment state(s)

ACT,NSW,NT,QLD,SA,TAS,WA,VIC

Funding & Sponsors

Funding source category [1]

Commercial sector/Industry

Name [1]

Alexion Pharmaceuticals, Inc

Address [1]

Country [1]

United States of America

Primary sponsor type

University

Name

Deakin University

Address

Country

Australia

Secondary sponsor category [1]

None

Name [1]

Address [1]

Country [1]

Ethics approval

Ethics application status

Not yet submitted

Ethics committee name [1]

Deakin University Human Research Ethics Committee (DUHREC)

Ethics committee address [1]

https://www.deakin.edu.au/research/support-for-researchers/research-integrity?a=438168

Ethics committee country [1]

Australia

Date submitted for ethics approval [1]

30/09/2026

Approval date [1]

Ethics approval number [1]

Summary

Brief summary

We recently collaborated with seven Australian adults living with HPP, and a carer of an adult living with HPP, to co-design a DVA-delivered health tool aimed at meaningfully supporting self-management of HPP. The participatory project explored the perspectives of those with lived experience of HPP, following the first three steps of the four-step Design Council Double Diamond Framework (Discover, Define, Develop, Deliver).

In the Discover and Define phases, three priorities were identified: providing connection with peers, coordinating care, and supporting self-management. The most important features addressing each priority were then determined using structured prioritisation (i.e. video calls with peers, community noticeboard, health diary/ monitoring, healthcare professional database, education sessions, artificial intelligence-supported question and answer function, and nutrition/mental health support). During the Develop phase, the prioritised features were prototyped and presented for feedback.

To address the final Deliver step of the Double Diamond framework, we will test the feasibility and preliminary effectiveness of using the DVA-delivered self-management program in a 12-week trial of adults living with HPP.

Trial website

Trial related presentations / publications

Public notes

Contacts

Principal investigator

Name

Prof David Scott

Address

Deakin University, Melbourne Burwood Campus, 221 Burwood Highway, Burwood VIC 3125

Country

Australia

Phone

+61392468438

Fax

[email protected]

Contact person for public queries

Name

David Scott

Address

Deakin University, Melbourne Burwood Campus, 221 Burwood Highway, Burwood VIC 3125

Country

Australia

Phone

+61392468438

Fax

[email protected]

Contact person for scientific queries

Name

David Scott

Address

Deakin University, Melbourne Burwood Campus, 221 Burwood Highway, Burwood VIC 3125

Country

Australia

Phone

+61392468438

Fax

[email protected]

Data sharing statement

Will the study consider sharing individual participant data?

Yes

Will there be any conditions when requesting access to individual participant data?

Persons/groups eligible to request access:
• Researchers
Conditions for requesting access:
• Yes, conditions apply:
• Requires review on a case-by-case basis by the trial custodian, sponsor or data sharing committee
• Requires a scientifically sound proposal or protocol
• Requires approval by an ethics committee
• Requires a data sharing agreement between data requester and trial custodian or sponsor

What individual participant data might be shared?

• All de-identified individual participant data

What types of analyses could be done with individual participant data?

• Any type of analysis (i.e. no restrictions on data re-use)

When can requests for individual participant data be made (start and end dates)?

From:
After publication of main results
To:
A finite period of: 7 years

Where can requests to access individual participant data be made, or data be obtained directly?

• Email of trial custodian, sponsor or committee: [email protected]

Are there extra considerations when requesting access to individual participant data?

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.

Trial Review

Documents added manually

Documents added automatically