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Trial registered on ANZCTR


Registration number
ACTRN12624000568516
Ethics application status
Approved
Date submitted
23/04/2024
Date registered
6/05/2024
Date last updated
6/05/2024
Date data sharing statement initially provided
6/05/2024
Type of registration
Prospectively registered

Titles & IDs
Public title
Cricopharyngeal peroral endoscopic myotomy (C-POEM) as a treatment for dysphagia in Parkinson’s disease: a multicentre randomised controlled trial
Scientific title
Efficacy of cricopharyngeal peroral endoscopic myotomy (C-POEM) as a treatment for dysphagia attributable to impaired cricopharyngeal relaxation in Parkinson’s disease: a multicentre randomised controlled trial
Secondary ID [1] 312004 0
None
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Parkinson's disease 333614 0
dysphagia 333630 0
Condition category
Condition code
Oral and Gastrointestinal 330316 330316 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Neurological 330317 330317 0 0
Parkinson's disease

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Cricopharyngeal peroral endoscopic myotomy (C-POEM) is a minimally invasive endoscopic procedure performed under general anaesthesia by an interventional endoscopist. The procedure is performed in a left lateral position. An initial submucosal bleb is followed by a longitudinal 1-2cm incision into the mucosa in the hypopharyngeal wall . Next, a short (5-6cm) submucosal tunnel is created extending into the proximal oesophageal wall thereby exposing the horizontal fibres of the cricopharyngeus. Full thickness dissection of the cricopharyngeus muscle (3-4cm in length) is then performed using the electric surgical knife. Lastly, the initial mucosal incision is closed with X-Tack Endoscopic HeliX Tacking System or through-the-scope endoscopic clips. The procedure duration is approximately 1 hour and entire procedure is video-captured and report documented in electronic medical record.
Postoperatively, patients receive 24 hours of prophylactic intravenous antibiotics and analgesia on an as required basis. Essential medications (including Parkinson's Disease-specific medications) are permitted with sips of water after recovery.
Intervention code [1] 328472 0
Treatment: Surgery
Comparator / control treatment
Participants will undergo standard-of-care swallow therapy consultations conducted by experienced speech pathologists. Two 30-minutes one-to-one sessions will be provided for each participant at 1-month apart. During the consultation, instructions for the following exercises and manoeuvres will be given based on the observed swallow dysfunction on the Videofluoroscopic Swallow Study (VFSS) performed during screening. Dietary modification if required will consist of viscosity selection guided by VFSS. A printout of written instructions will be provided to the patients at the end of the consultations to reinforce the instructions

Participants in the control group who do not respond to dysphagia therapy at 12 weeks and meet the inclusion criteria (ie: elevated Sydney Swallow Questionnaire score as well as abnormal Upper Oesophageal Sphincter Integrated Relaxation Pressure on post-treatment impedance manometry) will be offered a cross-over to undergo C-POEM
Control group
Active

Outcomes
Primary outcome [1] 338071 0
Change in dysphagia symptoms
Timepoint [1] 338071 0
Baseline and 12 weeks following treatment
Primary outcome [2] 338072 0
Change in upper oesophageal sphincter integrated relaxation pressure
Timepoint [2] 338072 0
Baseline and 12 weeks following treatment
Secondary outcome [1] 434372 0
Change in swallow-related quality of life
Timepoint [1] 434372 0
Baseline and at 12 weeks post treatment
Secondary outcome [2] 434373 0
Change in other manometric abnormalities associated with restrictive outflow obstruction resulting in dysphagia
Timepoint [2] 434373 0
Baseline and at 12 weeks post treatment
Secondary outcome [3] 434374 0
Change in opening characteristics of Upper Oesophageal Sphincter (UOS) on fluroscopy. This will be assessed as a composite outcome incorporating UOS diameter and opening duration.
Timepoint [3] 434374 0
Baseline and at 12 weeks post treatment
Secondary outcome [4] 434375 0
Change in deglutitive aspiration
Timepoint [4] 434375 0
Baseline and at 12 weeks post treatment
Secondary outcome [5] 434376 0
Change in body weight
Timepoint [5] 434376 0
Baseline and at 12 weeks post treatment
Secondary outcome [6] 434377 0
Change in distensibility of the cricopharyngeus muscle
Timepoint [6] 434377 0
immediately pre and post treatment while under anaesthesia (treatment arm only)
Secondary outcome [7] 434602 0
Change in post-swallow residue
Timepoint [7] 434602 0
Baseline and at 12 weeks post treatment

Eligibility
Key inclusion criteria
Diagnosis of Parkinson’s disease
Self-reported symptoms of oropharyngeal dysphagia
Abnormal Sydney Swallow Questionnaire (SSQ) score (>234)
Manometric evidence of failed upper oesophageal sphincter relaxation defined as an abnormally raised Integrated Relaxation Pressure
Minimum age
40 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Deep brain stimulator (DBS) – monopolar diathermy is contraindicated in DBS.
Severe oropharyngeal dysphagia that had led to a previous or current recommendation to stay completely nil-by-mouth.
Significant co-morbidities and/or advanced Parkinson’s Disease where risk of general anaesthesia is deemed unacceptably high.
Hypopharyngeal structural disorders known to cause pharyngeal dysfunction (e.g., Zenker’s diverticulum, radiotherapy-related CP stricture)
Oesophageal disorders known to cause dysphagia (e.g., achalasia cardia, peptic oesophageal stricture, eosinophilic oesophagitis etc).
Insufficient cognitive capability to follow clinicians’ instruction or to provide informed consent (e.g., dementia).

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)
central randomisation by computer
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Permuted blocked randomisation
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Not Applicable
Type of endpoint/s
Efficacy
Statistical methods / analysis
Because change from baseline in SSQ score and Upper Oesophageal Sphincter (UOS) Integrated Relaxation Pressure are co-primary endpoints the larger sample size for either endpoint was used. The estimates based on total published and unpublished C-POEM procedures performed to up to date (n=15) indicate that change in baseline in SSQ requires a larger sample size with preliminary improvement in SSQ score of 373 points from baseline of 871. Assuming improvement of 10% from baseline in the control arm, under 1:1 randomisation scheme a sample size of 52 (26 per study arm) will have 90% power at 2-sided alpha of 0.05.

Primary outcome will be analysed according to Intention-To-Treat principles using analysis of variance to test the null-hypothesis that two groups have equal means post treatment or ANCOVA as appropriate to adjust for significant pre-treatment differences between the study groups if they exist. Both endpoints must be statistically significant at the 2-sided 5% alpha-level.

Mean change from baseline to week 12 in total SSQ scores.
Mean change from baseline to week 12 in per-patient-averaged (UOS) Integrated Relaxation Pressure for 10ml bolus volume or the highest bolus volume tolerated without piecemeal swallowing.

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
13/05/2024
Actual
Date of last participant enrolment
Anticipated
31/12/2025
Actual
Date of last data collection
Anticipated
31/03/2026
Actual
Sample size
Target
52
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 26471 0
St George Hospital - Kogarah
Recruitment postcode(s) [1] 42451 0
2217 - Kogarah
Recruitment outside Australia
Country [1] 26280 0
Hong Kong
State/province [1] 26280 0

Funding & Sponsors
Funding source category [1] 316348 0
Charities/Societies/Foundations
Name [1] 316348 0
Brian and Pearl Bowles Fund
Country [1] 316348 0
Australia
Primary sponsor type
Hospital
Name
St George Hospital
Address
Country
Australia
Secondary sponsor category [1] 318558 0
None
Name [1] 318558 0
Address [1] 318558 0
Country [1] 318558 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 315161 0
South Eastern Sydney Local Health District HREC
Ethics committee address [1] 315161 0
https://www.seslhd.health.nsw.gov.au/services-clinics/directory/research-home/ethics
Ethics committee country [1] 315161 0
Australia
Date submitted for ethics approval [1] 315161 0
07/02/2023
Approval date [1] 315161 0
03/04/2023
Ethics approval number [1] 315161 0
2023/ETH00204

Summary
Brief summary
Failure of the upper oesophageal sphincter (UOS) relaxation is frequently observed in Parkinson's Disease. It contributes to swallowing dysfunction which has enormous impact on quality of life and is an important risk factor for life-threatening aspiration pneumonia. The randomized controlled trial aims to determine the efficacy of a novel, minimally invasive endoscopic myotomy of the cricopharyngeus muscle (C-POEM) as a treatment for swallowing dysfunction attributable to failed upper oesophageal sphincter relaxation.

Participants will undergo baseline assessments including pharyngeal high-resolution impedance-manometry (P-HRM-I) to confirm a diagnosis of UOS dysfunction. Participants with P-HRM-I features of failed UOS relaxation will be randomised to receive either:
1) C-POEM (treatment group) or
2) swallow therapy guided by a speech pathologist (control group).

At 12 weeks following randomisation and completion of assigned treatment, participants from both arms will undergo repeat evaluation, including: patient-reported outcome questionnaires, P-HRM-I, videofluoroscopic swallow study and clinical outcomes.

Participants in the control group who do not respond to dysphagia therapy will be offered a cross-over to undergo C-POEM.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 133862 0
Dr Peter Wu
Address 133862 0
Department of Gastroenetrology, St George Hospital, Gray St, Kogarah, NSW, 2217
Country 133862 0
Australia
Phone 133862 0
+61 02 91132490
Fax 133862 0
Email 133862 0
p.wu@unsw.edu.au
Contact person for public queries
Name 133863 0
Peter Wu
Address 133863 0
Department of Gastroenetrology, St George Hospital, Gray St, Kogarah, NSW, 2217
Country 133863 0
Australia
Phone 133863 0
+61 02 91132490
Fax 133863 0
Email 133863 0
p.wu@unsw.edu.au
Contact person for scientific queries
Name 133864 0
Michal Szczesniak
Address 133864 0
Department of Gastroenetrology, St George Hospital, Gray St, Kogarah, NSW, 2217
Country 133864 0
Australia
Phone 133864 0
+61 02 91133878
Fax 133864 0
Email 133864 0
m.szczesniak@unsw.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?

No Supporting Document Provided



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.