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Trial registered on ANZCTR


Registration number
ACTRN12616001582448
Ethics application status
Approved
Date submitted
26/10/2016
Date registered
16/11/2016
Date last updated
16/11/2016
Type of registration
Retrospectively registered

Titles & IDs
Public title
Speech treatment for spinocerebellar ataxia and Friedreich ataxia
Scientific title
Speech rehabilitation for dysarthria resulting from spinocerebellar ataxia or Friedreich ataxia
Secondary ID [1] 290392 0
None
Universal Trial Number (UTN)
U1111-1189-1034
Trial acronym
SRSCA
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Spinocerebellar ataxia 300716 0
Friedreich ataxia 300717 0
Dysarthria 300718 0
Condition category
Condition code
Neurological 300559 300559 0 0
Neurodegenerative diseases
Physical Medicine / Rehabilitation 300560 300560 0 0
Speech therapy

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment involved a behavioural intervention designed to improve speech intelligibility and quality.

Materials: Patients are provided with a laptop computer with treatment software installed, head mounted USB microphone, treatment manual.

Procedures: The program specifically targets prosody, vocal control and over-enunciation with the aim of improving overall speech intelligibility and confidence when communicating. It is based on principles of motor learning and neuroplasticity relating to practice conditions and bio-feedback.

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

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. Demonstrate and encourage the use of these feedback options.

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.

Therapy is completed in the home by the patient over 20 sessions within one month. Each session consists of excercises 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.

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 with a minimum of 5 years' experience;

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. Treatment is provided 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] 296224 0
Rehabilitation
Intervention code [2] 296391 0
Treatment: Other
Comparator / control treatment
A randomized, assessor blinded, delayed entry design. Participants will be randomly allocated to either one of two conditions: Group A - start after four weeks of monitoring or Group B - delayed start after 8 weeks of monitoring
Control group
Active

Outcomes
Primary outcome [1] 299973 0
Intelligibility as examined by blinded raters using direct magnitude estimation
Timepoint [1] 299973 0
Baseline and at 4 weeks (and 8 weeks for group B) post baseline. Then at 4 weeks post completion of treatment for both groups
Primary outcome [2] 300179 0
Naturalness as examined by blinded raters using direct magnitude estimation
Timepoint [2] 300179 0
Baseline and at 4 weeks (and 8 weeks for group B) post baseline. Then at 4 weeks post completion of treatment for both groups
Secondary outcome [1] 328731 0
Speech-related quality of life using the Dysarthria Impact Profile
Timepoint [1] 328731 0
Baseline and immediately post completion of treatment for both groups
Secondary outcome [2] 328732 0
Overall disease severity using the Scale for the assessment and rating of ataxia (SARA)
Timepoint [2] 328732 0
Baseline and at 4 weeks (and 8 weeks for group B) post baseline. Then at 4 weeks post completion of treatment for both groups
Secondary outcome [3] 329255 0
Acoustic analysis of speech: mean pause length
Timepoint [3] 329255 0
Baseline and at 4 weeks (and 8 weeks for group B) post baseline. Then at 4 weeks post completion of treatment for both groups
Secondary outcome [4] 329256 0
Acoustic analysis of voice: harmonics to noise ratio using praat
Timepoint [4] 329256 0
Baseline and at 4 weeks (and 8 weeks for group B) post baseline. Then at 4 weeks post completion of treatment for both groups
Secondary outcome [5] 329257 0
Acoustic analysis of speech: speech rate calculated by dividing syllables per second
Timepoint [5] 329257 0
Baseline and at 4 weeks (and 8 weeks for group B) post baseline. Then at 4 weeks post completion of treatment for both groups

Eligibility
Key inclusion criteria
Genetically confirmed diagnosis of spinocerebellar ataxia (any type) or genetically confirmed diagnosis of 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

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
The use of the specific tools will depend on the collected data (ie. if data follow quasi-Gaussian distributions, ANOVA tests will be used; otherwise we will use non-parametric tests such as the Mann-Whitney to compare groups). We will study the association strengths of objective characteristics of speech (eg. acoustic measures) with clinical and intelligibility measures using correlation coefficients and mutual information. We will compare densities computed using non-parametric approaches (eg. kernel density estimation) to identify differences between groups.

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
Actual
1/09/2016
Date of last participant enrolment
Anticipated
1/09/2017
Actual
Date of last data collection
Anticipated
1/02/2018
Actual
Sample size
Target
30
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 6867 0
Monash Medical Centre - Clayton campus - Clayton
Recruitment hospital [2] 6868 0
Royal Brisbane & Womens Hospital - Herston
Recruitment postcode(s) [1] 14537 0
3168 - Clayton
Recruitment postcode(s) [2] 14538 0
4029 - Herston
Recruitment outside Australia
Country [1] 8351 0
Germany
State/province [1] 8351 0

Funding & Sponsors
Funding source category [1] 294793 0
Charities/Societies/Foundations
Name [1] 294793 0
Alexander von Humboldt Foundation
Country [1] 294793 0
Germany
Primary sponsor type
University
Name
The University of Melbourne
Address
550 Swanston Street, Parkville, 3010 Victoria
Country
Australia
Secondary sponsor category [1] 293635 0
Hospital
Name [1] 293635 0
Universitatsklinikum Tuebingen
Address [1] 293635 0
Geissweg 3, 72076 Tuebingen
Country [1] 293635 0
Germany
Secondary sponsor category [2] 293636 0
Hospital
Name [2] 293636 0
Royal Brisbane Hospital
Address [2] 293636 0
Cartwright St, Windsor QLD 4030
Country [2] 293636 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 296195 0
Tuebingen University Medical Faculty Ethics Committee
Ethics committee address [1] 296195 0
Ethics committee country [1] 296195 0
Germany
Date submitted for ethics approval [1] 296195 0
03/03/2015
Approval date [1] 296195 0
09/04/2015
Ethics approval number [1] 296195 0
003/2015B02

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

Contacts
Principal investigator
Name 69942 0
Dr Adam Vogel
Address 69942 0
The University of Melbourne
550 Swanston Street, Parkville, 3010 Victoria
Country 69942 0
Australia
Phone 69942 0
+61390355334
Fax 69942 0
Email 69942 0
[email protected]
Contact person for public queries
Name 69943 0
Adam Vogel
Address 69943 0
The University of Melbourne
550 Swanston Street, Parkville, 3010 Victoria
Country 69943 0
Australia
Phone 69943 0
+61390355334
Fax 69943 0
Email 69943 0
[email protected]
Contact person for scientific queries
Name 69944 0
Adam Vogel
Address 69944 0
The University of Melbourne
550 Swanston Street, Parkville, 3010 Victoria
Country 69944 0
Australia
Phone 69944 0
+61390355334
Fax 69944 0
Email 69944 0
[email protected]

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

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