Using a Speech-Generating Device to Support Communication in Rare Genetic Conditions
A Randomized Cross-over Trial Examining the Efficacy of Implementing a Speech-generating Device for Rare Genetic Conditions
1 other identifier
interventional
38
1 country
1
Brief Summary
Individuals with rare genetic conditions may experience a delay or loss of developmental skills. Many have limited verbal speech. The aim of this clinical trial is to examine how well a speech-generating device supports the communication skills of participants with a rare genetic condition. The speech-generating device is a communication program loaded onto an iPad. This is a crossover trial, meaning that each participant will receive both the treatment (device) and a control (usual care; no device) phase. The order in which each participant receives the device versus the usual care (no device) will depend on which group the participant is assigned to. The changes in communication in each phase will then be compared. During the trial, participants can expect to complete a series of assessments and attend a total of 2 x 1-hour therapy session per week for 6 weeks.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for not_applicable
Started Nov 2025
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 18, 2025
CompletedFirst Posted
Study publicly available on registry
June 26, 2025
CompletedStudy Start
First participant enrolled
November 3, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 1, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
May 1, 2027
May 26, 2026
September 1, 2025
1.5 years
June 18, 2025
May 21, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Changes in the frequency of target communicative act related to the patient-defined outcome determined at the beginning of the trial
Each participant will have specific communicative acts related to a patient-defined outcome determined at the beginning of the trial with each participant and their family. This means that each participant will have an individualised communication outcome which will be targeted during treatment sessions. The change in frequency of these specific communicative acts will be measured. The mean difference of each period (comparing the start to end of the phase) will be determined and compared between the 2 periods.
Baseline, Week 0, Week 6 and Week 12
Secondary Outcomes (5)
Changes in overall communicative acts
Baseline, Week 0, Week 6 and Week 12
Changes in parent-reported communication via the Communication Matrix total score
Baseline, Week 0, Week 6 and Week 12
Changes in family and caregiver impact assessed via the Family Impact of Assistive Technology Scale for Augmentative and Alternative Communication (FIATS-AAC)
Baseline, Week 0, Week 6 and Week 12
Changes in family and caregiver impact assessed via semi-structured interviews
Baseline, Week 6 and Week 12
Changes in Language / Communication Competence assessed via the Low-Verbal Investigatory Survey
Baseline, Week 0, Week 6 and Week 12
Study Arms (2)
Treatment then Control
OTHERThe arm receives the intervention (device), then the control
Control then Treatment
OTHERThe arm receives the control, then the treatment (device)
Interventions
The device is an iPad loaded with a grid-based and speech-generating communication application - a suitable application for the participant is determined at the beginning of the trial. This device is used in all 12 therapy sessions and implemented by a qualified speech pathologist in a natural, play-based setting.
Participants will continue their current model of care. This means that if participants are receiving speech therapy locally, they may continue to do so given that no speech-generating device is introduced or used during this time. Participants will check in with the researching clinician at least once a week via telehealth or phone call - the aim is to stay in contact with families and reduce loss to follow-up. The clinician will also monitor treatment integrity and document any differences that may be present during this phase.
Eligibility Criteria
You may qualify if:
- Is between the ages of 3 and 12 years, inclusive, at the time of enrolment
- Has a diagnosis of a rare genetic disorder
- Passes a visual-motor screening test, therefore being able to tap on an iPad spontaneously or by imitation and has adequate hearing
- Considered "minimally verbal" with less than 50 spontaneous words (or gestalts) at baseline assessments, confirmed with the LVIS.
- Is not currently using a speech-generating device with proficiency (i.e. using the device as a main mode of communication on a daily basis).
- Is English-speaking or consents to therapy being conducted in English (parents will need to be able to complete the parent-reported measures in English)
You may not qualify if:
- Has an additional or dual genetic variation (as this is likely to cause multiple complications and increase variability),
- Is extremely ill or has progressed into a later stage of their disease (i.e. child has clinically significant loss of vision, hearing, fine motor skills, or is unable to adequately attend sessions due to illness),
- This is to ensure treatment is beneficial, reduce harm and reduce attrition rates.
- Lives outside of the state of Victoria (making it difficult for in-person appointments)
- Inability or unwillingness of participant or legally acceptable representative to give written informed consent.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Murdoch Children's Research Institute
Melbourne, Victoria, Australia
Related Publications (4)
Ganz JB, Morin KL, Foster MJ, Vannest KJ, Genc Tosun D, Gregori EV, Gerow SL. High-technology augmentative and alternative communication for individuals with intellectual and developmental disabilities and complex communication needs: a meta-analysis. Augment Altern Commun. 2017 Dec;33(4):224-238. doi: 10.1080/07434618.2017.1373855. Epub 2017 Sep 19.
PMID: 28922953BACKGROUNDNunn K, Williams K, Ouvrier R. The Australian Childhood Dementia Study. Eur Child Adolesc Psychiatry. 2002 Apr;11(2):63-70. doi: 10.1007/s007870200012.
PMID: 12033746BACKGROUNDLight J, McNaughton D. Communicative Competence for Individuals who require Augmentative and Alternative Communication: A New Definition for a New Era of Communication? Augment Altern Commun. 2014 Mar;30(1):1-18. doi: 10.3109/07434618.2014.885080.
PMID: 30952185BACKGROUNDElvidge KL, Christodoulou J, Farrar MA, Tilden D, Maack M, Valeri M, Ellis M, Smith NJC; Childhood Dementia Working Group. The collective burden of childhood dementia: a scoping review. Brain. 2023 Nov 2;146(11):4446-4455. doi: 10.1093/brain/awad242.
PMID: 37471493BACKGROUND
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- RANDOMIZED
- Masking
- SINGLE
- Who Masked
- OUTCOMES ASSESSOR
- Masking Details
- As well as the outcome assessments, research assistants completing coding/rating and integrity/reliability assessments will be blinded.
- Purpose
- SUPPORTIVE CARE
- Intervention Model
- CROSSOVER
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 18, 2025
First Posted
June 26, 2025
Study Start
November 3, 2025
Primary Completion (Estimated)
May 1, 2027
Study Completion (Estimated)
May 1, 2027
Last Updated
May 26, 2026
Record last verified: 2025-09
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, ICF, ANALYTIC CODE
- Time Frame
- 12 months after publication of primary outcome
- Access Criteria
- Interested researchers may request access to the data by submitting a formal data sharing request to the Sponsor. The request will be reviewed by the Sponsor and the Sponsor-Investigator, and any relevant Murdoch Children's Research Institute (MCRI) data sharing committee, considering factors such as scientific merit, data security, and adherence to the approved research objectives. The data may be obtained from MCRI by emailing mctc@mcri.edu.au
The de-identified data set collected for the analysis of this trial will be made available 12 months following analysis and publication of the primary outcome. The following will be made available long-term for use by future researchers from a recognised research institution: * Individual participant data that underlie the results reported after anonymisation/deidentification (text, tables, figures and appendices), * Trial protocol, Participant Information and Consent Form (PICF), * Statistical Analysis Plan (SAP), statistical code. Note that the data sharing of de-identified speech and language data is an Optional Consent for participants.