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Trial registered on ANZCTR


Registration number
ACTRN12619000927123
Ethics application status
Approved
Date submitted
17/06/2019
Date registered
3/07/2019
Date last updated
3/07/2019
Date data sharing statement initially provided
3/07/2019
Type of registration
Prospectively registered

Titles & IDs
Public title
Cricopharyngeal peroral endoscopic myotomy for cricopharyngeal achalasia: a pilot study on feasibility, efficacy, and safety
Scientific title
Cricopharyngeal peroral endoscopic myotomy for cricopharyngeal achalasia: a pilot study on feasibility, efficacy, and safety
Secondary ID [1] 298515 0
none
Universal Trial Number (UTN)
U1111-1235-5211
Trial acronym
C-POEM Study
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pharyngeal dysphagia 313323 0
Upper oesophageal sphincter dysfunction 313324 0
Condition category
Condition code
Oral and Gastrointestinal 311762 311762 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Surgery 311828 311828 0 0
Surgical techniques
Neurological 311829 311829 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Dysphagic patients with manometric evidence of inadequate upper oesophageal sphincter relaxation will undergo cricopharyngeal per-oral endoscopic myotomy (C-POEM). C-POEM is a minimally invasive endoscopic technique designed to perform oesophageal sphincterotomy; its safety and efficacy are well-established in the treatment of achalasia cardia. C-POEM is performed under general anaesthesia, and the anticipated average duration of the procedure will be 60 minutes. Clinical efficacy is objectively determined by 1) patient-reported outcome - dysphagia questionnaire; 2) intra-procedural upper oesophageal sphincter distensibility measurement; and 3) upper oesophageal sphincter relaxation pressure by high-resolution manometry.

C-POEM will be performed by experienced therapeutic gastrointestinal endoscopists (Principal Investigator Dr Peter Wu; co-investigator Prof Philip Chiu). All standard of care monitoring for endoscopic procedures will be applied, including nursing and anaesthetic documentation. Endoscopy reports will also be generated as part of medical record.
Intervention code [1] 314766 0
Treatment: Surgery
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 320442 0
Dysphagia symptoms measured by Swallow Quality-of-Life questionnaire (Swal-QOL)
Timepoint [1] 320442 0
1-month post-endoscopic myotomy
Secondary outcome [1] 371617 0
Upper oesophageal sphincter distensibility measured by the endoluminal functional lumen imaging probe
Timepoint [1] 371617 0
distensibility is measure intra-procedurally immediately before and after per-oral endoscopic myotomy
Secondary outcome [2] 371827 0
Patients will be observed in the hospital for a minimum of 24 hours after the procedure for major procedure-related complications, including (but not exclusive of) 1) Lumen perforation; 2) Would infection; 3) Bleeding requiring transfusion and/or intervention. Following discharge, weekly phone calls and 1-month clinic follow-up will be conducted by investigators to monitor for delayed complications such as bleeding and symptoms of infection.
Timepoint [2] 371827 0
Up to 30-day from the procedure

Eligibility
Key inclusion criteria
1) Known underlying neurological disorders that would result in upper oesophageal sphincter dysfunction, and
2) Self-reported pharyngeal dysphagia symptoms, and
3) Manometric evidence of upper oesophageal sphincter dysfunction
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Oesophageal disorders known to cause dysphagia (e.g. achalasia cardia, peptic oesopahgeal stricture)
2. Known hypopharyngeal structural disorders (e.g. radiotherapy-induced stricture)
3. Unwilling or unable to provide informed consent

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)
not applicable
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
not applicable
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Not Applicable
Type of endpoint(s)
Efficacy
Statistical methods / analysis
This is an open-label proof of principle study; there is no data to support a sample size calculation and the investigators believe n=30 will be sufficient to calculate variance estimates to derive a sample size for an adequately powered subsequent trial.
Hypothesis 1: C-POEM increase (improves) dysphagia severity score (SWAL-QOL)
Therapeutic response is defined as an increase (improvement) in SWAL-QOL score by at least 12 points after the C-POEM; this will be expressed as an percentage of response rate. To test the changes in patient-reported symptoms (SWAL-QOL score) with C-POEM, paired t-test will be used compare the two groups: 1) SWAL-POEM score at time of recruitment; 2) SWAL-POEM score at 3 months after the C-POEM. An equivalent non-parametric analysis will be performed if parametric assumptions are not met. Kaplan-Meier analysis of the follow-up (1-year) SWAL-QOL will be used to determine the rate and time to relapse of dysphagia.

Hypothesis 2: C-POEM increases (improves) CP distensibility.
Objectively measured CP distensibility is expressed as an index calculated by narrowest cross-sectional area (mm2) at maximal distension divided by corresponding intra-bag pressure (mm2/Hg). To test the changes of CP distensibility with C-POEM, paired t-test will be used compare: 1) CP distensibility index immediately before C-POEM; 2) CP distensibility index immediately after C-POEM. An equivalent non-parametric analysis will be performed if parametric assumptions are not met.

Hypothesis 3: C-POEM reduces (improves) CP deglutitive nadir pressure.
To test the changes in the CP deglutitive nadir pressure (mmHg) with C-POEM, paired t-test will be used compare the two groups: 1) CP pressure at time of recruitment; 2) CP pressure at 1 months after the C-POEM. An equivalent non-parametric analysis will be performed if parametric assumptions are not met.

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment outside Australia
Country [1] 21613 0
Hong Kong
State/province [1] 21613 0
HKSAR

Funding & Sponsors
Funding source category [1] 303061 0
Self funded/Unfunded
Name [1] 303061 0
not applicable - unfunded
Address [1] 303061 0
not application - unfunded
Country [1] 303061 0
Primary sponsor type
University
Name
The Chinese University of Hong Kong
Address
4M, S. H. Ho Centre for Digestive Health, Day Treatment Block and Children Wards, Prince of Wales Hospital, 30-32 Ngan Shing Street, Shatin, New Territories.
Country
Hong Kong
Secondary sponsor category [1] 303043 0
None
Name [1] 303043 0
Address [1] 303043 0
Country [1] 303043 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 303611 0
The Joint Chinese University of Hong Kong – New Territories East Cluster Clinical Research Ethics Committee
Ethics committee address [1] 303611 0
8/F, Lui Che Woo Clinical Sciences Building,
Prince of Wales Hospital
Shatin, Hong Kong
Ethics committee country [1] 303611 0
Hong Kong
Date submitted for ethics approval [1] 303611 0
15/03/2019
Approval date [1] 303611 0
09/04/2019
Ethics approval number [1] 303611 0
IRB 2019.183

Summary
Brief summary
Cricopharyngeal (CP) achalasia is a motility disorder where the upper oesophageal sphincter does not relax completely to allow passage of swallowed food and water from the pharynx into the oesophagus. It is associated with several neurological disorders including Parkinson's Disease, Lateral Medullary Stroke, and brain stem tumour. Patients with this condition suffer from troublesome swallowing, often resulting in both reduced quality of life and increase risk for malnourishment and chest infection.
Conventional treatment (cricopharyngeal myotomy) is a morbid open surgery that carries significant risk, thus often not offered to patients. Per-oral endoscopic myotomy (POEM) is a minimally invasive endoscopic technique initially designed to treat lower oesophageal sphincter dysfunction (achalasia cardia). This technique has potentially to be adapted in the treatment of CP achalasia while mimimising the risk associated with conventional open surgery.. In this pilot study, we aim to gather preiliminary clinical efficacy and safety of cricopharyngeal POEM in the treatment of swallowing difficulties related to upper oesophageal sphincter dysfunction.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 94254 0
Dr Peter Wu
Address 94254 0
4M, S. H. Ho Centre for Digestive Health, Day Treatment Block and Children Wards, Prince of Wales Hospital, 30-32 Ngan Shing Street, Shatin, New Territories.
Country 94254 0
Hong Kong
Phone 94254 0
+852 5239 2518
Fax 94254 0
Email 94254 0
p.wu@unsw.edu.au
Contact person for public queries
Name 94255 0
Mr Thomas Lam
Address 94255 0
4M, S. H. Ho Centre for Digestive Health, Day Treatment Block and Children Wards, Prince of Wales Hospital, 30-32 Ngan Shing Street, Shatin, New Territories.
Country 94255 0
Hong Kong
Phone 94255 0
+852 2637 0355
Fax 94255 0
Email 94255 0
thomaslam@cuhk.edu.hk
Contact person for scientific queries
Name 94256 0
Mr Thomas Lam
Address 94256 0
4M, S. H. Ho Centre for Digestive Health, Day Treatment Block and Children Wards, Prince of Wales Hospital, 30-32 Ngan Shing Street, Shatin, New Territories.
Country 94256 0
Hong Kong
Phone 94256 0
+85226370355
Fax 94256 0
Email 94256 0
thomaslam@cuhk.edu.hk

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 other documents available
Summary results
No Results