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.
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.
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)
Crossover
Speech-generating device
DeviceThe 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.
Control
Other interventionParticipants 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.
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.
Changes in overall communicative acts
Changes in overall communicative acts measured through the difference in total number of all communicative acts observed between the two periods. This will be measured by the overall number of communicative acts observed during an assessment session regardless of the relation to the patient-defined outcome. The change in the total score between the start and the end of each period will be calculated for each participant. The mean difference in the change in total score between the two periods will then be calculated.
Changes in parent-reported communication via the Communication Matrix total score
Changes in parent-reported communication skills will be measured using the Communication Matrix total score. For each participant the change in the total score between the start and the end of each period will be calculated. The mean difference in the total score between the two periods will then be calculated. The Communication Matrix allows for the quantification of reported behaviours, e.g. the total score is calculated by scoring each cell in the Matrix (each cell representing a communication behaviour at a particular level of communication). Each cell is scored with 0 (Not used), 1 (Emerging - inconsistently used) or 2 (Mastered - used proficiently at all times). These points are summed to obtain a score between 0 to 160 and a percentage is obtained to represent the proportion of communication behaviours Mastered.
Changes in family and caregiver impact assessed via the Family Impact of Assistive Technology Scale for Augmentative and Alternative Communication (FIATS-AAC)
Changes in parent satisfaction, social impact and the mental health of caregivers will be assessed using the Family Impact of Assistive Technology Scale for Augmentative and Alternative Communication (FIATS-AAC). Each item is scored on a 7-point Likert scale and assigned into one of the 13 domains (Behavior, Caregiver Relief, Contentment, Doing Activities, Education, Energy, Face-to-Face Communication, Family Roles, Finances, Security, Self Reliance, Social Versatility, Supervision). The mean score for each domain (ranging between 1 to 7) are added together to produce the total score (ranging between 13 to 91). A higher domain score suggests a higher (more positive) functional level of that specific domain. The change in scores between the start and the end of each period will be calculated for each participant. The mean difference in the score between the two periods will then be calculated.
Changes in family and caregiver impact assessed via semi-structured interviews
Changes in parent satisfaction, social impact and the mental health of caregivers will be assessed using a semi-structured interview as seen in Vogel et al. (2016), completed at baseline and at the end of each phase. Thematic analysis will be used to identify any themes that are present during these interviews and descriptively compare the differences identified.
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