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Trial registered on ANZCTR


Registration number
ACTRN12616000570482
Ethics application status
Approved
Date submitted
27/04/2016
Date registered
3/05/2016
Date last updated
10/07/2017
Type of registration
Prospectively registered

Titles & IDs
Public title
Behavioural Balloon Dilatation in Upper Esophageal Sphincter (UES) Dysfunction
Scientific title
Effects of Behavioural Balloon Dilatation on UES opening diameter and duration and UES pressures during swallowing in participants with UES Dysfunction
Secondary ID [1] 289009 0
Nil known
Universal Trial Number (UTN)
U1111-1180-0414
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Dysphagia 298414 0
UES dysfunction 298415 0
Condition category
Condition code
Physical Medicine / Rehabilitation 298513 298513 0 0
Speech therapy
Oral and Gastrointestinal 298656 298656 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Each participant will undergo 6 assessments and 10 treatment sessions over a period of 4 months at the Rose Centre for Stroke Recovery and Research, University of Canterbury (see attachment “Study procedures” for a diagram detailing procedures that will occur at each session). Each session is expected to last approximately one hour. Some of the assessment and treatment sessions have been combined – these sessions will last approximately 1.5 hours. Each session will be an individual face-to-face session. They will be delivered by a speech language therapist with a minimum of 5 years’ experience.
Each participant will undergo two baseline assessments with the Timed Water Swallow Test (TWST), the Test of Mastication and Swallowing Solids (TOMASS). These are screening tests where the participant will be observed while drinking a fixed amount of water and when eating a cracker. The participant will then undergo High-Resolution Manometry (HRM) and Videofluoroscopic Swallowing Study (VFSS) to document UES function prior to therapy. At the second baseline assessment session, if measures on HRM and the screening tests remain similar, VFSS will be omitted to limit radiation exposure. After the two baseline assessment sessions, a two-week behavioural dilatation treatment program will then commence.
The intervention under investigation is behavioural balloon dilatation. It involves oral insertion of a urethral balloon catheter without sedation to the upper esophageal sphincter (UES). Two balloon dilatation methods will be carried out for each participant:
a. the retrograde method – the balloon catheter is inserted to under the UES before the balloon is inflated with air and pulled through the UES in synchrony with swallowing (20 repetitions)
b. the anterograde method –the balloon portion of the catheter is inflated with air before the participant swallows it down past the UES with the aid of a guidewire (10 repetitions).
The retrograde method will be implemented from the first week of treatment. The anterograde method will be added on only in the second week. Each time a new method of dilatation is taught, the first dilatation will be done with visualisation by VFSS to mark how far the balloon catheter needs to be inserted. This will hence occur twice: once in the first retrograde dilatation attempt in the first week; the other in the first anterograde dilatation attempt in the second week. In subsequent dilatation attempts without VFSS, insertions will follow the markings made and the participant will be instructed to phonate after swallowing the catheter to ensure the catheter has not been inserted into the trachea.
The regimen will hence consist of a total of twenty repetitions of balloon dilatation at each session, two sessions per day, five times per week for the first week. At the end of the first week, HRM will be carried out to chart progress before commencing the second week of treatment. It potentially may yield data that indicates which patients may or may not tolerate anterograde dilatation. The regimen then increases to thirty repetitions of balloon dilatation at each session, two sessions per day, five times per week for the second week. Patients are encouraged to perform self-dilatation of the UES. To rate the tolerability of the treatment program, participants will be asked to rate their comfort level on a visual analogue scale at one week into the treatment phase and again at immediately after treatment has ended.
Initial balloon sizes will be determined by VFSS opening diameters measured from the baseline VFSS sessions. Balloon size will then gradually be increased based on patient comfort levels and intra-balloon pressures generated when the balloon passes the UES. Intra-balloon pressures will be measured throughout the whole dilatation session with a manometer connected to a laptop which will display pressures within the balloon on screen. Dou and colleagues (2012) reported that small increments in balloon size will lead to fewer side effects like bleeding from mucosal trauma. Typically intra-balloon pressures will be monitored to remain less than 20% above baseline pressures. If pressures stay within 20% of baseline pressures, balloon size will be increased by 0.5mm. The maximum balloon diameter will correspond to normative data of maximum UES diameter during swallowing (normative data is available in Leonard and Kendall, 2013). Once this maximum diameter is achieved, therapy will continue at this balloon size until the end. This is to minimise the risk of bleeding from over-expansion.
At the end of the two-week of treatment, the TWST, TOMASS, HRM and VFSS will be repeated to document change in UES opening and manometric pressures from treatment. At two weeks and at three months after treatment has terminated, the TWST, TOMASS and HRM will be repeated to assess short and long-term effects of treatment. Once again, VFSS will be omitted if HRM measures remain stable to minimise radiation exposure.
Functional change in swallowing function will be monitored via scoring on the Functional Oral Intake Scale (FOIS), a 7-point scale based on details of a participant’s feeding status and amount of intake. The Eating Assessment Tool (EAT-10), a short questionnaire that assesses change in patient-reported dysphagia symptoms and severity, and quality of life, will also be administered at each session. Based on previous research indicating that reflux symptoms may increase after oesophageal dilatation, it would be of interest to investigate if laryngopharyngeal reflux, in particular, will be affected after behavioural dilatation. Change in symptoms will be tracked using the Reflux Symptom Index (RSI).
Intervention code [1] 294491 0
Rehabilitation
Intervention code [2] 294492 0
Behaviour
Intervention code [3] 294614 0
Treatment: Other
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 298013 0
Composite primary outcome: To determine if the behavioural balloon dilatation regimen is effective through assessing UES opening diameter and duration via videofluoroscopic swallowing study (VFSS)
Timepoint [1] 298013 0
i) Immediately after end of treatment (immediate outcome)
ii) 2 weeks after treatment (short-term outcome)
iii) 3 months after treatment (longer-term outcome)
Primary outcome [2] 298014 0
Composite primary outcome: To determine if the behavioural balloon dilatation regimen is effective through assessing manometric measurements of the UES (UES resting pressure, nadir relaxation pressure and relaxation duration) on High-Resolution Manometry (HRM).
Timepoint [2] 298014 0
i) mid-treatment (1 week after treatment has commenced, mid-treatment outcome)
ii) Immediately after end of treatment (immediate outcome)
iii) 2 weeks after treatment (short-term outcome)
iv) 3 months after treatment (longer-term outcome
Primary outcome [3] 298152 0
Composite primary outcome: To investigate change in scores on functional swallowing scales - Functional Oral Intake Scale (FOIS) and Eating Assessment Tool (EAT-10)
Timepoint [3] 298152 0
i) Immediately after end of treatment (immediate outcome)
ii) 2 weeks after treatment (short-term outcome)
iii) 3 months after treatment (longer-term outcome)
Secondary outcome [1] 322894 0
Composite secondary outcome: To determine change in reflux symptoms due to behavioural balloon dilatation as assessed by Reflux Symptom Index (RSI)
Timepoint [1] 322894 0
i) Immediately after end of treatment (immediate outcome)
ii) 2 weeks after treatment (short-term outcome)
iii) 3 months after treatment (longer-term outcome)
Secondary outcome [2] 323286 0
Assess tolerability of behavioural balloon dilatation as a treatment program with 100mm visual analogue scale
Timepoint [2] 323286 0
i) After first week of treatment
ii) After second week of treatment
Secondary outcome [3] 323287 0
Determine if UES manometric pressures, as measured on HRM, can indicate which participants can tolerate anterograde balloon dilatation in the second week of treatment
Timepoint [3] 323287 0
one week after treatment

Eligibility
Key inclusion criteria
- Able to give informed consent
- Diagnosis of UES dysfunction evidenced by reduced UES opening and pyriform residue post swallow on VFS and/ or
- Abnormal UES opening pressures on HRM
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Are medically unstable
- Have cognitive or language impairments
- Have abnormalities in the head and neck region
- Have undergone therapy for head and neck cancer including extirpative surgery, chemotherapy and radiation therapy
- Are currently on tracheostomy
- Have known upper oesophageal abnormalities e.g. active inflammation, tumours, perforation (not including reflux)
- Have known nasal abnormalities that precludes use of manometry
- Have known bleeding tendencies
- Are currently undergoing any other forms of swallowing management for UES dysfunction

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Allocation is not concealed
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
N.A.
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Multiple baseline cohort study
Phase
Not Applicable
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis
The sample size was calculated using G*Power program based on published data from the study by Arenaz and colleagues (2015) which listed UES sagittal diameter 1 month pre- and post-intervention (intervention method was conventional balloon dilatation) in a sample size of 4:
Mean change in UES sagittal diameter (post- versus pre-treatment) = 1.48 mm
Standard deviation = 1.50 mm
Tails = Two
Calculated effect size = 0.99
Alpha error probability = 0.05
Power (1- beta error probability) = 0.8
Calculated sample size = 11
A calculated sample size of 11 was obtained. However, given that the sample size was calculated from outcome data for medical UES dilatation and there is a lack of data from behavioural dilatation studies regarding exact change in UES opening size, a smaller effect size is expected for behavioural balloon dilatation. This is due to it being less intrusive as it is not expected to tear muscle. Hence, a more conservative sample size of 20 has been set.

Statistical data analysis of the treatment phase will be conducted using the Statistical Package for the Social Sciences (SPSS) program.

Independent variable: Time since intervention (balloon dilatation)
Dependent variables: UES opening diameter (ratio), UES opening duration (ratio), UES resting pressure (ratio), UES nadir relaxation pressure (ratio), UES relaxation duration (ratio), Timed Water Swallow Test times (ratio), Test of Masticating and Swallowing Solids times (ratio), Functional Oral Intake Scale (FOIS) score (ordinal), Reflux Symptom Index (RSI) score (ordinal), Eating Assessment Tool (EAT-10) score (ordinal)

Data screening for normal distribution and homogeneity of variance will first be carried out using various methods, including box plots, histograms, calculations of skewness and Levene’s test . If the data do not meet the requirements for parametric testing, transformation of the data into a logarithm scale will be undertaken, or non-parametric tests will be used.

Descriptive statistics such as the mean, range and standard deviation of each measure will be listed. The alpha level will be set at 0.05 for this study.

There are six points in time when the dependent variables are measured:
1) First baseline
2) Second baseline
3) Mid treatment (one week after treatment) – only for HRM measures of UES resting pressure, UES nadir relaxation pressure and UES relaxation duration
4) Post treatment (immediate)
5) Post treatment (two weeks after)
6) Post-treatment (three months after)
To assess for changes in the dependent variables that are ratio data over these times, repeated measures ANOVA will be used. For ordinal data i.e. FOIS scores, RSI scores and EAT-10 scores, the mean of these scores will be reported.

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
16/05/2016
Actual
8/08/2016
Date of last participant enrolment
Anticipated
31/05/2018
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
20
Accrual to date
Final
Recruitment outside Australia
Country [1] 7819 0
New Zealand
State/province [1] 7819 0
Canterbury

Funding & Sponsors
Funding source category [1] 293379 0
University
Name [1] 293379 0
University of Canterbury
Country [1] 293379 0
New Zealand
Primary sponsor type
Individual
Name
Seh Ling Kwong
Address
Rose Centre for Stroke Recovery and Research at St George’s Medical Centre
Leinster Chambers, Level 1, Private Bag 4737
249 Papanui Road
Christchurch 8140
Country
New Zealand
Secondary sponsor category [1] 292217 0
Individual
Name [1] 292217 0
Maggie-Lee Huckabee
Address [1] 292217 0
Rose Centre for Stroke Recovery and Research at St George’s Medical Centre
Leinster Chambers, Level 1, Private Bag 4737
249 Papanui Road
Christchurch 8140
Country [1] 292217 0
New Zealand
Secondary sponsor category [2] 292218 0
Individual
Name [2] 292218 0
Phoebe Macrae
Address [2] 292218 0
Rose Centre for Stroke Recovery and Research at St George’s Medical Centre
Leinster Chambers, Level 1, Private Bag 4737
249 Papanui Road
Christchurch 8140
Country [2] 292218 0
New Zealand

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 294836 0
Health and Disability Ethics Committees
Ethics committee address [1] 294836 0
Ministry of Health
Ethics Department
Freyberg Building
Reception – Ground Floor
20 Aitken Street
Wellington 6011
Ethics committee country [1] 294836 0
New Zealand
Date submitted for ethics approval [1] 294836 0
28/04/2016
Approval date [1] 294836 0
27/06/2016
Ethics approval number [1] 294836 0
16/NTA/63

Summary
Brief summary
Medical management of impairment in the UES has traditionally consisted of the surgical option of cricopharyngeal myotomy, with variable efficacy. More conservative options include medical dilatation, botulinum toxin injections and swallowing rehabilitation exercises carried out by the speech-language pathologist. Behavioural balloon dilatation, a conservative approach, was first documented in the 1990s by Japanese researchers. Frequently used in Asian countries as an adjunct to swallowing rehabilitation programs, the protocol for balloon rehabilitation has varied across studies, with a paucity of data available on long-term effects of UES dysfunction and UES dysfunction caused by disorders other than neurological diseases.

This study will evaluate the short-term and longer-term efficacy, safety and tolerability of balloon dilatation as a behavioural treatment approach for patients with UES dysfunction. Twenty patients with UES dysfunction, demonstrated on videofluoroscopic swallowing study (VFSS) and high-resolution manometry (HRM), will be recruited to undergo a two-week intensive behavioural balloon dilatation protocol with a follow-up session at three months after therapy. The protocol is based on the concept of retraining the response of the UES via a series of behavioural therapy sessions, rather than just a single medical, mechanical expansion of the UES with the balloon. A combination of two main methods of self-administered balloon dilatation will be carried out with an inflated urethral balloon catheter: 1) anterograde method, where an inflated balloon is “swallowed” through the UES with aid of a guidewire; 2) retrograde method, where the catheter is inserted below the UES before inflating the balloon, then pulled up through the UES in synchrony with dry swallows. The retrograde method will be implemented from the first day of treatment and the anterograde method will only be added on in the second week of treatment. Dilatation will be performed 20 to 30 times in total within each session, two sessions a day, five days a week for two weeks, before re-evaluation of swallowing function. Initial balloon sizes will be determined based on UES opening size on VFSS. Subsequent increases in size will be guided by pressures that are generated when the balloon passes the UES, paired with patient comfort levels, to minimise the risk of mucosal trauma. Data analysis will compare pre and post- therapy UES opening diameter and duration during swallowing as well as UES resting pressure, nadir relaxation pressure and UES relaxation duration to determine the efficacy of behavioural balloon dilatation in treating UES dysfunction. Change in feeding status is also of interest, to determine if improvements in VFSS and HRM measures lead to functional changes in swallowing. Maintenance effects of therapy will also be examined with data provided at the three-month follow-up.
Trial website
Trial related presentations / publications
Public notes
Attachments [1] 863 863 0 0
/AnzctrAttachments/370206-Study procedures.doc

Contacts
Principal investigator
Name 63882 0
Ms Seh Ling Kwong
Address 63882 0
Rose Centre for Stroke Recovery and Research at St George’s Medical Centre
Leinster Chambers, Level 1, Private Bag 4737
249 Papanui Road
Christchurch 8140
Country 63882 0
New Zealand
Phone 63882 0
+6433692385
Fax 63882 0
Email 63882 0
[email protected]
Contact person for public queries
Name 63883 0
Seh Ling Kwong
Address 63883 0
Rose Centre for Stroke Recovery and Research at St George’s Medical Centre
Leinster Chambers, Level 1, Private Bag 4737
249 Papanui Road
Christchurch 8140
Country 63883 0
New Zealand
Phone 63883 0
+6433692385
Fax 63883 0
Email 63883 0
[email protected]
Contact person for scientific queries
Name 63884 0
Seh Ling Kwong
Address 63884 0
Rose Centre for Stroke Recovery and Research at St George’s Medical Centre
Leinster Chambers, Level 1, Private Bag 4737
249 Papanui Road
Christchurch 8140
Country 63884 0
New Zealand
Phone 63884 0
+6433692385
Fax 63884 0
Email 63884 0
[email protected]

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