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Trial registered on ANZCTR


Registration number
ACTRN12618000519257
Ethics application status
Approved
Date submitted
28/03/2018
Date registered
9/04/2018
Date last updated
11/03/2019
Date data sharing statement initially provided
11/03/2019
Type of registration
Prospectively registered

Titles & IDs
Public title
SpeechATAX: Intensive home based biofeedback driven speech treatment for hereditary ataxia
Scientific title
SpeechATAX: A 2-arm, examiner-blinded, wait-list control group randomized controlled trial of intensive home based biofeedback driven speech rehabilitation in hereditary ataxia
Secondary ID [1] 294427 0
Nil
Universal Trial Number (UTN)
U1111-1211-2931
Trial acronym
SpeechATAX
Linked study record
ACTRN12616001583437 - This study is a pilot study to the current study.
ACTRN12616001582448 - This study is a pilot study to the current study.

Health condition
Health condition(s) or problem(s) studied:
Dysarthria 307174 0
Friedreich ataxia 307175 0
Spinocerebellar ataxia 307176 0
Condition category
Condition code
Neurological 306290 306290 0 0
Neurodegenerative diseases
Physical Medicine / Rehabilitation 306291 306291 0 0
Speech therapy
Human Genetics and Inherited Disorders 306381 306381 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment will involve a behavioural intervention designed to improve speech intelligibility and quality (SpeechAtax computer software).

Materials: Patients will be provided with a tablet with treatment software installed, head mounted USB microphone and treatment manual. The treatment manual includes comprehensive instructions for the exercises to complete for each of the 20 days of therapy.

Procedures: The program is designed specifically to target improving overall speech intelligibility as well as vocal control and prosody. It is based on principles of motor learning and neuroplasticity relating to practice conditions and bio-feedback.

The program consists of three stages:

Phase I: Teaching and Establishment of treatment components
The clinician introduces the goals and content of the therapy. Patients learn the skills needed to undertake the program and implementation of the exercises. Using the computer program will be reviewed and practiced together on the first visit.

Phase II: Instatement and Rehabilitation
Therapy is completed in the home by the patient over 20 sessions within one month. Each session consists of exercises aiming to improve vocal quality and control, articulation, prosody and intelligibility. Self-evaluation skills are refined by the use of visual, aural and performance feedback and personalised problem-solving strategies. Clinicians monitor patient progress and provide support during this stage through weekly skype or phone calls.

On each treatment day, the patient is required to complete seven exercises outlined in the SpeechAtax software and treatment manual:
i) Long vowel x 10 repetitions
ii) Loudness exercise: crescendo/decrescendo x 10 repetitions
iii) Pitch glide x 10 repetitions
iv) Emphasis/Stress: Short phrases with target word designed to improve prosody x 15
v) Articulation: words and short phrases to practice over-enunciation
vi) Reading task
vii) Transfer exercise (daily question)

vii) Conversation (introduced on day 16).

The patient is encouraged to make use of the following feedback options throughout the duration of treatment:

A: Visual feedback:
Visual feedback is provided through the real time loudness display.
Patients can monitor the stability or variability of their loudness while speaking. For example, if patients cannot see any colour in the visual display they need to increase the loudness of their speech. Similarly, a stable production is required for the long vowel task, meaning the visual display should remain at a constant level throughout the production.
The feedback given by the interface supports the patient by training self-awareness. Loudness is represented visually, providing an alternative mode of feedback other than listening.

B: Aural (listening) feedback:
Patients are prompted to record parts of their speech each day. They are then required to listen to their recorded sample from the previous day. Listening feedback is designed to enhance self-monitoring and help the speaker identify aspects of their speech that need improvement.

Listening to speech can be upsetting for some patients but it is a vital component of therapy. Delayed aural feedback is important for the development of self-monitoring skills by providing an opportunity to hear their performance, identify what worked and what went wrong and set some goals for the day.

C: Outcome feedback:
Patients are provided with immediate and objective feedback of their performance. Three pieces of information are derived from the recorded samples and compared against the previous days’ scores:
1. Duration, which is important for the long vowel task; 2-3. Loudness and pitch variation, both of which are important for the vowel and connected speech tasks. This information is designed to enhance the patient’s understanding of their performance and to provide a benchmark on which to compare earlier productions.
Summary figures are provided at the end of each day after tasks are completed. The clinician will be able to plot your patients’ progress – highlighting the gains made during therapy.

Phase III: Generalisation and Maintenance
After having completed Phases I and II, the patient can use the materials to consolidate improvements made during Phase II. Maintenance exercises will assist in maintaining intelligibility within a broader context beyond home-practice sessions. Here the patient will be asked to apply their newly acquired skills in specific situations in everyday life. These should preferably be selected together with the patient (or his/her relatives if necessary).


Who: Treatment is coordinated by the treating clinician and completed in the home or clinic by the patient. Treating clinicians are trained speech-language pathologists.

Mode of delivery: The first treatment session is conducted face to face. Each subsequent treatment day is conducted using the computer software. Adherence and progress is monitored weekly by the treating clinician via skype or telephone. Therapy is completed in the home and individually.

Number of times: The intervention will be delivered 20 times over a 30-day period. Patients are required to complete therapy 5 days out of every 7 for the period of the study. Treatment sessions take approximately 45 minutes to complete. 20 x 45 minute sessions 5 times a week for 4 weeks.
Intervention code [1] 300722 0
Rehabilitation
Intervention code [2] 300723 0
Treatment: Other
Comparator / control treatment
A randomized, assessor blinded, delayed entry design. After informed consent, participants will be randomly allocated 1:1 to one of two conditions: Group A - start treatment (SpeechAtax) after four weeks of monitoring/standard care or Group B - delayed start of treatment after 8 weeks of monitoring/standard care.
Control group
Active

Outcomes
Primary outcome [1] 305295 0
Intelligibility: To be rated via expert perceptual judgement (blinded assessors) of the first 30 seconds from the monologue using direct magnitude estimation (DME).
Timepoint [1] 305295 0
Baseline assessment (week 1), post baseline assessment at week 4 (and 8 weeks for group B). Then assessment immediately post treatment [primary timepoint], and again 4 weeks post completion of treatment for both groups.
Secondary outcome [1] 344764 0
Paired comparison ratings of conversation by naïve listeners. Listeners are asked to decide which of two audio-recorded samples (pre- and post-treatment, played in random order) is clearer. Informal objective rating scale will be used.
Timepoint [1] 344764 0
Baseline assessment (week 1), post baseline assessment at week 4 (and 8 weeks for group B). Then assessment immediately post treatment, and again 4 weeks post completion of treatment for both groups.
Secondary outcome [2] 344765 0
Objective measures of speech, including acoustic measures of timing (eg. pause length in reading and monologue tasks), vocal control (eg. fundamental frequency and loudness variation from vowel and monologue), and vocal quality (eg. dysphonia measures derived from sustained vowel). This is a composite secondary outcome.
Timepoint [2] 344765 0
Baseline assessment (week 1), post baseline assessment at week 4 (and 8 weeks for group B). Then assessment immediately post treatment, and again 4 weeks post completion of treatment for both groups.
Secondary outcome [3] 344766 0
Oral motor function derived from Frenchay Dysarthria Profile - 2 which examines muscles and functions relevant for speech including lips, tongue, breathing, palate.
Timepoint [3] 344766 0
Baseline assessment (week 1), post baseline assessment at week 4 (and 8 weeks for group B). Then assessment immediately post treatment, and again 4 weeks post completion of treatment for both groups.
Secondary outcome [4] 344768 0
Speech-related quality of life (Dysarthria Impact Profile, DIP). The DIP is a standardised survey on the psychosocial impact of acquired dysarthria from the speaker’s perspective.
Timepoint [4] 344768 0
Baseline assessment (week 1), post baseline assessment at week 4 (and 8 weeks for group B). Then assessment immediately post treatment, and again 4 weeks post completion of treatment for both groups.
Secondary outcome [5] 344771 0
Communication partner perception (Communicative Effectiveness Survey, CES) The CES is a standardised survey describing domains of communication beyond intelligibility. Disorder-specific patient-reported outcomes will address issues unique to the condition being studied.
Timepoint [5] 344771 0
Baseline assessment (week 1), post baseline assessment at week 4 (and 8 weeks for group B). Then assessment immediately post treatment, and again 4 weeks post completion of treatment for both groups.
Secondary outcome [6] 344773 0
Health utility score (SF-36). The SF-36 will inform economic quality of life outcomes
Timepoint [6] 344773 0
Baseline assessment (week 1), post baseline assessment at week 4 (and 8 weeks for group B). Then assessment immediately post treatment, and again 4 weeks post completion of treatment for both groups.
Secondary outcome [7] 344776 0
Carer survey. An informal survey of carer time will provide data on healthcare-related costs. This survey has been designed specifically for this study.
Timepoint [7] 344776 0
Baseline assessment (week 1), post baseline assessment at week 4 (and 8 weeks for group B). Then assessment immediately post treatment, and again 4 weeks post completion of treatment for both groups.
Secondary outcome [8] 344777 0
Overall disease severity. Scale for the assessment and rating of ataxia (SARA) score
Timepoint [8] 344777 0
Baseline assessment (week 1), post baseline assessment at week 4 (and 8 weeks for group B). Then assessment immediately post treatment, and again 4 weeks post completion of treatment for both groups.

Eligibility
Key inclusion criteria
Genetically confirmed diagnosis of hereditary ataxia including diagnosis of spinocerebellar ataxia (any type) or Friedreich ataxia.

Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
No genetically confirmed diagnosis of spinocerebellar ataxia (any type) or genetically confirmed diagnosis of Friedreich ataxia. People with other progressive neurological disorders including Parkinson's disease, multiple sclerosis and Huntington's disease will not be included in this study.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled 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
Blinded (masking used)
Who is / are masked / blinded?


The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Other
Other design features
The study is a randomised, assessor blinded, delayed entry design with two groups of participants, not a cross-over design.
Phase
Not Applicable
Type of endpoint/s
Efficacy
Statistical methods / analysis
Sample size estimation and power calculation: Using the overall findings in our pilot study, a sample size of 40 participants per arm is needed to detect a geometric mean ratio of 1.25 in the speech intelligibility at 4 weeks of the home-based intervention to the existing care arm with 90% power at the two-sided 5% level of significance assuming an equal coefficient of variation of 0.31 for both arms. A 5-10% relative increase is considered a clinically meaningful difference. We intend to recruit 100 people with FRDA or other hereditary ataxias allowing for 20% attrition – a realistic number given the model of delivery and caseload available at each recruitment site.

The primary outcome speech intelligibility will be base-2 log-transformed before fitting a mixed-model to the measurements at baseline, immediately post-treatment and 4 weeks post treatment, including in the model baseline (log2), arm, week, and arm by week interaction as well as the stratification factors site and ataxia type. The primary hypothesis will be evaluated using the estimate and two-sided 95% CI of the geometric mean ratio of the home-based intervention to existing-care at 4 weeks. The treatment effect at 3 and 6 months will be examined similarly. The treatment effect within and heterogeneity of the treatment effect between ataxia type will be explored. The model provides valid inference if the underlying missing data mechanism is missing at random. The robustness of the results to this missing data assumption will be investigated using a pattern-mixture model. The clinical meaningfulness of the treatment effect on a patient level (ie. treatment responder) will be explored using a generalised mixed-model with a logit link fitted to responder status (yes/no) at 4 data collection points.The estimate and two-sided 95% CI of the odds ratio of being a treatment responder following home-based intervention compared to existing care will be obtained at post treatment. Continuous secondary outcomes will be analysed using a similar model specification as the primary outcome, with (e.g., outcome on ratio scale or skewed) or without appropriate transformations before fitting the model. Binary secondary outcomes will be analysed similarly to the analysis of treatment responders.

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
23/04/2018
Actual
1/06/2018
Date of last participant enrolment
Anticipated
8/01/2021
Actual
Date of last data collection
Anticipated
28/05/2021
Actual
Sample size
Target
100
Accrual to date
30
Final
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 10467 0
Royal Brisbane & Womens Hospital - Herston
Recruitment hospital [2] 10468 0
Monash Medical Centre - Clayton campus - Clayton
Recruitment hospital [3] 10469 0
Caulfield Hospital - Caulfield
Recruitment postcode(s) [1] 22176 0
4029 - Herston
Recruitment postcode(s) [2] 22177 0
3168 - Clayton
Recruitment postcode(s) [3] 22178 0
3162 - Caulfield
Recruitment outside Australia
Country [1] 10237 0
Germany
State/province [1] 10237 0
Baden-Württemberg
Country [2] 10238 0
New Zealand
State/province [2] 10238 0
Auckland Region
Country [3] 21340 0
France
State/province [3] 21340 0
Paris

Funding & Sponsors
Funding source category [1] 299058 0
Government body
Name [1] 299058 0
Australian Government, Department of Health
Country [1] 299058 0
Australia
Primary sponsor type
University
Name
The University of Melbourne
Address
550 Swanston Street, Parkville, 3010 Victoria
Country
Australia
Secondary sponsor category [1] 298293 0
Hospital
Name [1] 298293 0
Universitatsklinikum Tuebingen
Address [1] 298293 0
Geissweg 3, 72076 Tuebingen
Country [1] 298293 0
Germany
Secondary sponsor category [2] 298298 0
Hospital
Name [2] 298298 0
Royal Brisbane and Women's Hospital
Address [2] 298298 0
Cartwright Street, Windsor, QLD, 4030
Country [2] 298298 0
Australia
Secondary sponsor category [3] 298338 0
Charities/Societies/Foundations
Name [3] 298338 0
Friedreich Ataxia Research Association (FARA)
Address [3] 298338 0
PO Box 210 Elsternwick VIC 3185
Country [3] 298338 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 299991 0
The University of Melbourne Psychology Health and Applied Sciences Human Ethics Committee
Ethics committee address [1] 299991 0
Ethics committee country [1] 299991 0
Australia
Date submitted for ethics approval [1] 299991 0
21/01/2018
Approval date [1] 299991 0
02/02/2018
Ethics approval number [1] 299991 0
1339394
Ethics committee name [2] 300024 0
Medical Ethics Board, University Hospital Tübingen, Germany (Az. 003/2015BO2
Ethics committee address [2] 300024 0
Ethics committee country [2] 300024 0
Germany
Date submitted for ethics approval [2] 300024 0
03/03/2015
Approval date [2] 300024 0
09/04/2015
Ethics approval number [2] 300024 0
003/2015BO2

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes
Attachments [1] 2528 2528 0 0
/AnzctrAttachments/374783-2018 amendment SPEECH ATAX.pdf (Ethics approval)

Contacts
Principal investigator
Name 82190 0
A/Prof Adam Vogel
Address 82190 0
The University of Melbourne
550 Swanston Street, Parkville, 3010 Victoria

Country 82190 0
Australia
Phone 82190 0
+61390355334
Fax 82190 0
Email 82190 0
vogela@unimelb.edu.au
Contact person for public queries
Name 82191 0
Hannah Reece
Address 82191 0
The University of Melbourne
550 Swanston Street, Parkville, 3010 Victoria
Country 82191 0
Australia
Phone 82191 0
+613 8344 7687
Fax 82191 0
Email 82191 0
hannah.reece@unimelb.edu.au
Contact person for scientific queries
Name 82192 0
Adam Vogel
Address 82192 0
The University of Melbourne
550 Swanston Street, Parkville, 3010 Victoria
Country 82192 0
Australia
Phone 82192 0
+61390355334
Fax 82192 0
Email 82192 0
vogela@unimelb.edu.au

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




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.